3 unchanged sentences
We expect to incur losses for the foreseeable future and may never achieve or maintain profitability.
−Removed: Since inception, we have incurred recurring losses and negative cash flows from operations and, at March 31, 2021, we had an accumulated deficit of $87.0 million.
+Added: Since inception, we have incurred recurring losses and negative cash flows from operations and, at June 30, 2021, we had an accumulated deficit of $103.4 million.
Our ability to achieve profitability depends on our ability to successfully complete the development of, and obtain the regulatory approvals necessary to commercialize our products candidates.
1 unchanged sentence
We have devoted substantially all our efforts to date to research and development of our gene therapy product candidates as well as to building out our infrastructure.
−Removed: We expect that it could be several years, if ever, before we have a commercialized product candidate.
+Added: We expect that it could be at least a year, if ever, before we have a commercialized product candidate.
We expect to continue to incur significant expenses and increasing operating losses for the foreseeable future.
3 unchanged sentences
• initiate clinical trials for KB104, KB407, KB408 and preclinical studies for any additional product candidates that we may pursue in the future;
−Removed: • prepare our BLA, MAA and approvals in certain other countries for B-VEC;
−Removed: • continue to operate our in-house commercial-scale cGMP manufacturing facility ANCORIS and complete build out of our second cGMP manufacturing facility ASTRA;
+Added: • prepare our Biologics License Application ("BLA"), Marketing Authorisation Application and approvals in certain other countries for B-VEC;
+Added: • continue to operate our in-house commercial-scale cGMP manufacturing facility, ANCORIS, and complete the build out of our second cGMP manufacturing facility, ASTRA;
• manufacture material for clinical trials or potential commercial sales;
5 unchanged sentences
To become and remain profitable, we must develop and eventually commercialize one or more product candidates with significant market potential.
−Removed: This will require us to be successful in a range of challenging activities, including completing the clinical trials for our product candidates, developing and validating commercial scale manufacturing processes, obtaining marketing approval for this product candidate, manufacturing, marketing and selling any future product candidates for which we may obtain marketing approval and satisfying any post-marketing requirements.
−Removed: If we were required to discontinue development of any of our product candidates, if any of our product candidates do not receive regulatory approval, if we do not obtain our targeted indications for our product candidates or if any of our product candidates fails to achieve sufficient market acceptance for any indication, we could be delayed by many years in our ability to achieve profitability, if ever, and would materially adversely affect our business prospects and financial condition.
−Removed: Moreover, if we decide to leverage any success with our B-VEC, KB105, KB301, KB104 or KB407 product candidates to develop other product opportunities, we may not be successful in such efforts.
−Removed: In any such event, our business will be materially adversely affected.
−Removed: We currently only have three product candidates, B-VEC, KB105, and KB301 in clinical trials and we may never develop, acquire or in-license additional product candidates.
−Removed: We may never succeed in any or all these activities and, even if we do, we
−Removed: may never generate revenues that are significant or large enough to achieve profitability.
+Added: This will require us to be successful in a range of challenging activities, including completing the clinical trials for our product candidates, developing and validating commercial scale manufacturing processes, obtaining marketing approval for our product candidates, manufacturing, marketing and selling any product candidates for which we may obtain marketing approval and satisfying any post-marketing requirements.
+Added: If we were required to discontinue development of any of our product candidates, if any of our product candidates do not receive regulatory approval, if we do not obtain our targeted indications for our product candidates or if any of our product candidates fails to achieve sufficient market acceptance for any indication, we could be delayed by many years in our ability to achieve profitability, if ever, and this would materially adversely affect our business prospects and financial condition.
+Added: Moreover, if we decide to leverage any success with our B-VEC, KB105, KB301, KB104 or KB407 product candidates to develop other product opportunities, we may not be successful in such efforts, and our business could be materially adversely affected.
+Added: We currently have three product candidates, B-VEC, KB105, and KB301 in clinical trials and we may never develop, acquire or in-license additional product candidates.
+Added: We may never succeed in any or all these activities and, even if we do, we may never generate revenues that are significant or large enough to achieve profitability.
If we do achieve profitability, we may not be able to sustain or increase profitability on a quarterly or annual basis.
Our failure to become and remain profitable would decrease the value of our company and could impair our ability to raise capital, maintain our research and development efforts, expand our business or continue our operations.
−Removed: A decline in the value of our company also could cause you to lose all or part of your investment.
+Added: A decline in the value of our company also could cause investors to lose all or part of their investment.
Because of the numerous risks and uncertainties associated with pharmaceutical product and biological development, we are unable to accurately predict the timing or amount of increased expenses or when, or if, we will be able to achieve profitability.
−Removed: If we are required by the FDA, the EMA, or other regulatory authorities to perform studies in addition to those currently expected, or if there are any delays in completing our clinical trials or the development of B-VEC, KB105, and KB301 our expenses could increase and revenue could be further delayed.
+Added: If we are required by the FDA, the European Medicines Agency ("EMA"), or other regulatory authorities to perform studies in addition to those currently expected, or if there are any delays in completing our clinical trials or the development of B-VEC, KB105, and KB301 our expenses could increase and revenue could be further delayed.
We may need to raise additional funding in order to receive approval for our other product candidate.
3 unchanged sentences
In addition, if we obtain marketing approval for our product candidates, we expect to incur significant expenses related to product sales, medical affairs, marketing, manufacturing and distribution.
−Removed: Furthermore, we expect to continue to incur significant costs associated with operating as a public company.
We anticipate that we may need additional funding to complete the development of B-VEC, KB105, KB301 and any future product candidates and to commercialize any such approved products.
8 unchanged sentences
• the costs associated with the manufacturing process development and evaluation of third-party manufacturers, if necessary;
−Removed: • the costs of future activities, including product sales, medical affairs, marketing, manufacturing and distribution, in the event we receive marketing approval for any of our current and future product candidates;
• the extent to which the costs of our product candidates, if approved, will be paid by health maintenance, managed care, pharmacy benefit and similar healthcare management organizations, or will be reimbursed by government authorities, private health coverage insurers and other third-party payors;
4 unchanged sentences
• our current license agreements, if any, remaining in effect and our achievement of milestones under those agreements;
−Removed: • our ability to establish and maintain collaborations and licenses on favorable terms, if at all;
• the extent to which we acquire or in-license other product candidates and technologies.
16 unchanged sentences
We expect our financial condition and operating results to continue to fluctuate from quarter to quarter and year to year due to a variety of factors, many of which are beyond our control.
−Removed: We will need to transition at some point from a company with a research and development focus to a company capable of undertaking commercial activities.
+Added: We will need to transition from a company with a research and development focus to a company capable of undertaking commercial activities.
We may encounter unforeseen expenses, difficulties, complications and delays and may not be successful in such a transition.
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We plan to conduct clinical trials in geographies that are currently being affected by COVID-19.
−Removed: Further, in response to the pandemic and in accordance with direction from national, state and local government authorities, we have restricted access to our office, manufacturing and laboratory facilities to personnel and third parties who must perform critical activities that must be completed on-site, limited the number of such personnel that can be present at our facilities at any one time, and requested that many of our personnel work remotely.
−Removed: In the event that governmental authorities were to further modify current restrictions, our employees conducting research and development or manufacturing activities may not be able to access our laboratory or manufacturing spaces, and our core activities may be significantly limited or curtailed, possibly for an extended period of time.
+Added: In the event that we or governmental authorities were to modify or implement restrictions, our employees conducting research and development or manufacturing activities may not be able to access our laboratory or manufacturing spaces, and our core activities may be significantly limited or curtailed, possibly for an extended period of time.
Some factors from the COVID-19 pandemic that could delay or otherwise adversely affect the completion of our preclinical activities and the planned initiation of our clinical trials for our investigational drug product candidates, as well as our business operations generally, include:
7 unchanged sentences
• business disruptions caused by potential office, manufacturing and laboratory closures and an increased reliance on employees working from home, disruptions to or delays in ongoing laboratory experiments and operations, staffing shortages, travel limitations, cyber security and data accessibility, or communication or mass transit disruptions, any of which could adversely impact our business operations or delay necessary interactions with local regulators, ethics committees, manufacturing sites, research sites and other important agencies and contractors.
−Removed: These and other factors arising from COVID-19 could worsen in countries that are already afflicted with the coronavirus or could continue to spread to additional countries, each of which could further adversely impact our ability to conduct clinical trials and our business generally and could have a material adverse impact on our operations and financial condition and results.
+Added: These and other factors arising from COVID-19 could worsen in countries that are already afflicted with the coronavirus which could further adversely impact our ability to conduct clinical trials and our business generally and could have a material adverse impact on our operations and financial condition and results.
In addition, the trading prices for our common stock and other biopharmaceutical companies have been highly volatile as a result of the COVID-19 pandemic.
1 unchanged sentence
Further, conditions in the bank lending, capital and other financial markets may continue to deteriorate as a result of the pandemic such that our access to capital and other sources of funding may be constrained.
−Removed: The COVID-19 outbreak continues to evolve.
−Removed: The extent to which the outbreak may impact our business, preclinical studies and planned clinical trials will depend on future developments, which are highly uncertain and cannot be predicted with confidence, such as the ultimate geographic spread of the disease, the duration of the outbreak, travel restrictions and other actions to contain the outbreak or address its impact, such as social distancing and quarantines or lock-downs in the United States and other countries, business closures or business disruptions and the effectiveness of actions taken in the United States and other countries to contain and address the disease.
+Added: The COVID-19 pandemic continues to evolve.
+Added: The extent to which the outbreak may impact our business, preclinical studies and planned clinical trials will depend on future developments, which are highly uncertain and cannot be predicted with confidence, such as the duration of the outbreak, travel restrictions and other actions to contain the outbreak or address its impact, such as social distancing and quarantines or lock-downs in the United States and other countries, business closures or business disruptions and the effectiveness of actions taken in the United States and other countries to contain and address the disease.
We are a development-stage company.
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If we fail in one or more of these factors in a timely manner or at all, we could experience significant delays or an inability to successfully commercialize our product candidates, which would materially harm our business.
−Removed: If we do not receive regulatory approvals for our product candidates, our business, financial condition, results of operations and prospects could be materially and adversely affected.
Our lead candidate, B-VEC, is still in clinical development, and there is no guarantee that the results from preclinical studies will be indicative of our ability to complete or the results to be obtained in the current Phase 3 clinical trials.
4 unchanged sentences
Furthermore, research and discoveries by us or others may identify serious adverse events, undesirable side effects or other unexpected properties of our current and future product candidates, including B-VEC, that could delay, prevent or cause the withdrawal of regulatory approval, limit the commercial potential, or result in significant negative consequences following marketing approval.
−Removed: The regulatory authorities may not complete their review processes in a timely manner, or we may not be able to obtain regulatory approval.
−Removed: Additional delays may result if an FDA Advisory Committee or other regulatory authority recommends non-approval or restrictions on approval.
−Removed: In addition, we may experience delays or rejections based upon additional government regulation from future legislation or administrative action, or changes in regulatory authority policy during the period of product development, clinical trials and the review process.
−Removed: Regulatory authorities also may approve a product candidate for more limited indications than requested or they may impose significant limitations in the form of narrow indications, warnings or post-approval safety monitoring program.
−Removed: These regulatory authorities may require precautions or contra-indications with respect to conditions of use or they may grant approval subject to the performance of costly post-marketing clinical trials.
−Removed: In addition, regulatory authorities may not approve the labeling claims that are necessary or desirable for the successful commercialization of B-VEC.
−Removed: Any of the foregoing scenarios could materially harm the commercial prospects for B-VEC and materially and adversely affect our business, financial condition, results of operations and prospects.
Even if we complete the necessary clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize B-VEC and the approval may be for a narrower indication than we seek.
2 unchanged sentences
Additional delays may result if an FDA Advisory Committee or other regulatory authority recommends non-approval or restrictions on approval.
−Removed: In addition, we may experience delays or rejections based upon additional government regulation from future legislation or administrative action, or changes in regulatory authority policy during the period of product development, clinical trials and the review process.
+Added: In addition, we may experience delays or rejections based upon additional government regulation from future legislation or administrative action, or changes in regulatory authority or policy during the period of product development, clinical trials and the review process.
Regulatory authorities also may approve a product candidate for more limited indications than requested or they may impose significant limitations in the form of narrow indications, warnings or a post-approval safety monitoring program.
−Removed: These regulatory authorities may require precautions or contra-indications with respect to conditions of use or they may grant approval subject to the performance of costly post-marketing clinical trials.
+Added: Regulatory authorities may require precautions or contra-indications with respect to conditions of use or they may grant approval subject to the performance of costly post-marketing clinical trials.
In addition, regulatory authorities may not approve the labeling claims that are necessary or desirable for the successful commercialization of B-VEC.
3 unchanged sentences
The regulatory approval process for novel product candidates such as ours can be more expensive and take longer than for other, better known or more extensively studied product candidates.
−Removed: It is difficult to determine how long it will take or how much it will cost to obtain regulatory approvals for our product candidates in either the United States or the European Union or how long it will take to commercialize our product candidates.
−Removed: Approvals by the European Commission may not be indicative of what FDA may require for approval.
−Removed: Regulatory requirements governing gene and cell therapy products have changed frequently and may continue to change in the future.
+Added: It is difficult to determine how long it will take or how much it will cost to obtain regulatory approvals for our product candidates in
+Added: either the United States or the EU or how long it will take to commercialize our product candidates.
+Added: Approvals by the European Commission may not be indicative of what the FDA may require for approval, and approval by the FDA may not be indicative of what the European Commission would require for approval.
+Added: Regulatory requirements and policy governing gene and cell therapy products have changed frequently and may continue to change in the future.
The FDA has established the Office of Tissues and Advanced Therapies within its Center for Biologics Evaluation and Research (“CBER”) to consolidate the review of gene therapy and related products, and has established the Cellular, Tissue and Gene Therapies Advisory Committee to advise CBER in its review.
−Removed: If we were to engage a National Institutes of Health funded institution to conduct a clinical trial, that institution’s Institutional Biosafety Committee (“IBC”) as well as its Institutional Review Board (“IRB”), would need to review the proposed clinical trial to assess the safety of the trial.
+Added: If we were to engage a National Institutes of Health funded institution to conduct a clinical trial, that institution’s Institutional Biosafety Committee as well as its Institutional Review Board (“IRB”), would need to review the proposed clinical trial to assess the safety of the trial.
Similarly, the EMA may issue new guidelines concerning the development and marketing authorization for gene therapy medicinal products and require that we comply with these new guidelines.
These regulatory review committees and advisory groups and the new guidelines they promulgate may lengthen the regulatory review process, require us to perform additional studies, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of B-VEC or future product candidates or lead to significant post-approval limitations or restrictions.
−Removed: As we advance B-VEC, we will be required to consult with these regulatory and advisory groups and comply with applicable requirements and guidelines.
+Added: As we advance B-VEC, we may be required to consult with these regulatory and advisory groups and will need to comply with applicable requirements and guidelines.
If we fail to do so, we may be required to delay or discontinue development of B-VEC.
4 unchanged sentences
Gene therapy is still a relatively new approach to disease treatment and additional adverse side effects could develop.
+Added: The risk of cancer remains a concern for gene therapy, and we cannot assure that it will not occur in any of our planned or future clinical studies.
There also is the potential risk of delayed adverse events following exposure to gene therapy products due to persistent biologic activity of the genetic material or other components of products used to carry the genetic material.
In addition to side effects caused by the product candidate, the administration process or related procedures also can cause adverse side effects.
−Removed: If any such adverse events occur, our clinical trials could be suspended or terminated.
−Removed: If in the future we are unable to demonstrate that such adverse events were caused by the administration process or related procedures, the FDA, the European Commission, the EMA or other regulatory authorities could order us to cease further development of, or deny approval of, B-VEC for any or all targeted indications.
+Added: If any such adverse events occur with B-VEC, our clinical trials could be suspended or terminated.
+Added: If we are unable to demonstrate that such adverse events were caused by the administration process or related procedures, the FDA, the European Commission, the EMA or other regulatory authorities could order us to cease further development of, or deny approval of, B-VEC for any or all targeted indications.
Even if we can demonstrate that any serious adverse events are not product-related, such occurrences could affect patient recruitment or the ability of enrolled patients to complete the trial.
−Removed: Moreover, if we elect, or are required, to delay, suspend or terminate any clinical trial of B-VEC, the commercial prospects of such product candidate may be harmed and our ability to generate product revenues from this product candidate may be delayed
−Removed: or eliminated.
+Added: Moreover, if we elect, or are required, to delay, suspend or terminate any clinical trial of B-VEC, the commercial prospects may be harmed and our ability to generate product revenues may be delayed or eliminated.
Any of these occurrences may harm our ability to develop other product candidates, and may harm our business, financial condition and prospects significantly.
1 unchanged sentence
Furthermore, if we or others later identify undesirable side effects caused by B-VEC, several potentially significant negative consequences could result, including:
−Removed: • regulatory authorities may suspend or withdraw approvals of such product candidate;
+Added: • regulatory authorities may suspend or withdraw approvals of B-VEC;
• regulatory authorities may require additional warnings on the label;
−Removed: • we may be required to change the way a product candidate is administered or conduct additional clinical trials;
+Added: • we may be required to change the way B-VEC is administered or conduct additional clinical trials;
• we could be sued and held liable for harm caused to patients;
13 unchanged sentences
• changes in regulatory requirements and guidance that require amending or submitting new clinical protocols.
−Removed: In addition, if we make manufacturing or formulation changes to B-VEC, we may need to conduct additional studies to bridge our modified product candidate to earlier versions.
−Removed: Clinical trial delays could also shorten any periods during which we may have the exclusive right to commercialize B-VEC or allow our competitors to bring products to market before we do, which could limit our potential revenue or impair our ability to successfully commercialize B-VEC and may harm our business, financial condition, results of operations and prospects.
+Added: In addition, if we make manufacturing or formulation changes to a product candidate, we may need to conduct additional studies to bridge our modified product candidate to earlier versions.
+Added: Clinical trial delays could also shorten any periods during which we may have the exclusive right to commercialize our product candidates or allow our competitors to bring products to market before we do, which could limit our potential revenue or impair our ability to successfully commercialize our product candidates and may harm our business, financial condition, results of operations and prospects.
Any delays, setbacks or failures in our clinical trials could materially and adversely affect our business, financial condition, results of operations and prospects.
4 unchanged sentences
• be subject to additional post-marketing testing requirements;
−Removed: • be required to perform additional clinical trials to support approval or be subject to additional post-marketing testing requirements;
+Added: • be required to perform additional clinical trials to support approval;
• have regulatory authorities withdraw, or suspend, their approval of the drug or impose restrictions on its distribution;
−Removed: • be subject to the addition of labeling statements, such as warnings or contraindications;
• experience damage to our reputation.
1 unchanged sentence
We do not know whether any of our preclinical studies or clinical trials will begin as planned, need to be restructured or be completed on schedule, if at all.
−Removed: Further, we, the FDA or an IRB, may suspend our clinical trials at any time if it appears that we or our collaborators are failing to conduct a trial in accordance with regulatory requirements, including the FDA’s current Good Clinical Practice (“GCP”) regulations, that we are exposing participants to unacceptable health risks, or if the FDA finds deficiencies in our IND applications or the conduct of these trials.
+Added: Further, we, the FDA or an IRB, may suspend our clinical trials at any time if it appears that we or our collaborators are failing to conduct a trial in accordance with regulatory requirements, including the FDA’s current Good Clinical Practice regulations, that we are exposing participants to unacceptable health risks, or if the FDA finds deficiencies in our IND applications or the conduct of these trials.
Therefore, we cannot predict with any certainty the schedule for commencement and completion of future clinical trials.
2 unchanged sentences
We are a small company with a limited number of employees and corporate infrastructure.
−Removed: We have experienced a period of significant expansion in headcount and expect to experience significant expansion of our facilities, infrastructure and overhead as we develop our own manufacturing facility and increase our research and development efforts.
+Added: We have experienced a period of significant expansion in headcount and expect to experience significant expansion of our facilities, infrastructure and overhead as we develop our own manufacturing facilities and increase our research and development efforts.
Future growth will impose significant added capital requirements, as well as added responsibilities on members of management, including the need to identify, recruit, maintain and integrate new personnel.
8 unchanged sentences
In addition, product manufacturers and their facilities are subject to payment of user fees and continual review and periodic inspections by the FDA and other regulatory authorities for compliance with cGMP requirements and adherence to commitments made in the BLA or foreign marketing application.
−Removed: If we, or a regulatory authority, discover previously unknown problems with a product, such as adverse events of unanticipated severity or frequency, or problems with the facility where the product is manufactured or disagrees with the promotion, marketing or labeling of that product, a regulatory authority may impose restrictions relative to that product, the manufacturing facility or us, including requiring recall or withdrawal of the product from the market or suspension of manufacturing.
+Added: If we, or a regulatory authority, discover previously unknown problems with a product, such as adverse events of unanticipated severity or frequency, or problems with the facility where the product is manufactured or a regulatory authority disagrees with the promotion, marketing or labeling of that product, a regulatory authority may impose restrictions relative to that product, the manufacturing facility or us, including requiring recall or withdrawal of the product from the market or suspension of manufacturing.
If we fail to comply with applicable regulatory requirements following approval of B-VEC or any future product candidate, a regulatory authority may:
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We cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative action, either in the United States or abroad.
−Removed: If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any marketing approval that we may have obtained and we may not achieve or sustain profitability, which would materially and adversely affect our business, financial condition, results of operations and prospects.
+Added: If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any marketing approval that
+Added: we may have obtained and we may not achieve or sustain profitability, which would materially and adversely affect our business, financial condition, results of operations and prospects.
While we have obtained orphan drug designation for B-VEC, KB105 and KB407, it may not effectively protect us from competition, and we may be unable to obtain orphan drug designation for our future product candidates.
If our competitors are able to obtain orphan drug exclusivity for products that constitute the same drug and treat the same indications as our product candidates before us, we may not be able to have competing products approved by the applicable regulatory authority for a significant period of time.
−Removed: On November 2, 2017, the FDA granted orphan drug designation to our lead product candidate, B-VEC, for the treatment of DEB and we may seek orphan drug designation from the FDA for our future product candidates.
−Removed: On April 16, 2018, the European Commission granted the Orphan Medicinal Product Designation (“OMPD”) for B-VEC.
+Added: On November 2, 2017, the FDA granted orphan drug designation to our lead product candidate, B-VEC, for the treatment of DEB.
+Added: On April 16, 2018, the European Commission granted the Orphan Medicinal Product Designation for B-VEC.
On August 7, 2018, the FDA granted orphan drug designation to our second product candidate, KB105, currently in clinical development for treatment of patients with TGM1 deficient ARCI, and on October 10, 2019, the European Commission granted the Orphan Medicinal Product Designation for KB105.
15 unchanged sentences
• the holder of the marketing authorization for the original orphan medicinal product cannot supply enough quantities of orphan medicinal product.
−Removed: Breakthrough therapy designation, Regenerative Medicine Advanced Therapy designation, Fast Track designation or Rare Pediatric Disease designation by the FDA, even if granted for any of our product candidates, may not lead to a faster development, regulatory review or approval process, and it does not increase the likelihood that any of our product candidates will receive marketing approval in the United States.
+Added: Breakthrough therapy designation, Regenerative Medicine Advanced Therapy ("RMAT") designation, Fast Track designation or Rare Pediatric Disease designation by the FDA, even if granted for any of our product candidates, may not lead to a faster development, regulatory review or approval process, and it does not increase the likelihood that any of our product candidates will receive marketing approval in the United States.
The FDA granted Fast Track designation in the United States for B-VEC on May 23, 2018 and for KB105 on October 24, 2019.
27 unchanged sentences
• a product candidate may not be accepted as safe and effective by patients, the medical community or third-party payors.
−Removed: If we are unsuccessful in identifying and developing additional product candidates, our potential for growth may be impaired.
+Added: If we are unsuccessful in identifying and developing additional product candidates, our potential for growth will be impaired.
We face significant competition in an environment of rapid technological change and the possibility that our competitors may achieve regulatory approval before us or develop therapies that are more advanced or effective than ours, which may adversely affect our financial condition and our ability to successfully market or commercialize our lead product candidate, B-VEC or any future product candidate.
5 unchanged sentences
Additionally, technologies developed by our competitors may render B-VEC or any future product candidate uneconomical or obsolete, and we may not be successful in marketing B-VEC or any future product candidate against competitors.
−Removed: In addition, as a result of the expiration or successful challenge of our patent rights, we could face more litigation with respect to the validity and/or scope of patents relating to our competitors’ products.
+Added: In the future, even if we commercialize a product candidate faster than our competitors, we could also face competition from lower cost biosimilars in the United States or in Europe.
+Added: In addition, as a result of the expiration or successful challenge of our patent rights, we could face litigation with respect to the validity and/or scope of patents relating to our competitors’ products.
The availability of our competitors’ products could limit the demand, and the price we are able to charge, for any product candidate that we may develop and commercialize.
1 unchanged sentence
Delays in obtaining regulatory approvals of the process and facilities needed to manufacture our product candidates or disruptions in our manufacturing process may delay or disrupt our product development and commercialization efforts.
−Removed: Before we can begin to commercially manufacture our product candidates, whether in a third-party facility or in our own facilities, once established, we must pass a pre-approval inspection of our manufacturing facilities by the FDA before our product candidates can obtain marketing approval.
+Added: Before we can begin to commercially manufacture our product candidates, whether in a third-party facility or in our own facilities, we must pass a pre-approval inspection of our manufacturing facilities by the FDA before our product candidates can obtain marketing approval.
A manufacturing authorization must also be obtained from the appropriate EU regulatory authorities.
3 unchanged sentences
The cGMP requirements govern quality control of the manufacturing process and documentation policies and procedures.
−Removed: In complying with cGMP, we will be obligated to expend time, money and effort in production, record keeping and quality control to assure that the product meets applicable
−Removed: specifications and other requirements.
+Added: In complying with cGMP, we will be obligated to expend time, money and effort in production, record keeping and quality control to assure that the product meets applicable specifications and other requirements.
If we fail to comply with these requirements, we would be subject to possible regulatory action and may not be permitted to sell any product candidate that we may develop.
11 unchanged sentences
Even if we obtain the validation from the FDA of our cGMP manufacturing facility, we intend to maintain third-party manufacturing capabilities in order to provide multiple sources of supply.
−Removed: In the event that these third-party manufacturers do not successfully carry out their contractual duties, meet expected deadlines or manufacture our product candidates in accordance with regulatory requirements or if there are disagreements between us and these third-party manufacturers, we will not be able to complete, or may be delayed in completing, the preclinical studies required to support future IND submissions of other product candidates or the clinical trials required for approval of our product candidates.
−Removed: In such instances, we may need to locate an appropriate replacement third-party relationship, which may not be readily available or on acceptable terms, which would cause additional delay or increased expense prior to the approval of our product candidates and would thereby have a material adverse effect on our business, financial condition, results of operations and prospects.
+Added: In the event that these third-party manufacturers do not successfully carry out their contractual duties, meet expected deadlines or manufacture our product candidates in accordance with regulatory requirements or if there are disagreements between us and these third-party manufacturers, we will not be able to complete, or may be delayed in completing, the preclinical studies required to support future IND submissions of product candidates or the clinical trials required for approval of our product candidates.
+Added: In such instances, we may need to locate an appropriate replacement third-party manufacturer, which may not be readily available or on acceptable terms, which would cause additional delay or increased expense prior to the approval of our product candidates and would thereby have a material adverse effect on our business, financial condition, results of operations and prospects.
If we or our third-party manufacturer fails to comply with applicable cGMP regulations, the FDA and foreign regulatory authorities can impose regulatory sanctions including, among other things, refusal to approve a pending application for a new product candidate or suspension or revocation of a pre-existing approval.
10 unchanged sentences
To successfully commercialize our product candidates, if approved, we plan to expand our capabilities to promote market access and build awareness.
−Removed: To successfully commercialize any other products that may result from our development programs, we will need to further expand our market development organization, either on our own or with a third party.
+Added: To successfully commercialize any products that may result from our development programs, we will need to further expand our market development organization, either on our own or with a third party.
The development of our own market development team will be expensive and time-consuming and could delay any product launch.
−Removed: Moreover, we cannot be certain that we will be able to successfully
−Removed: develop this capability.
+Added: Moreover, we cannot be certain that we will be able to successfully develop this capability.
We may enter into collaboration agreements regarding any of our product candidates with third parties to utilize their established marketing and distribution capabilities, but we may be unable to enter into such agreements on favorable terms, if at all.
13 unchanged sentences
Serious adverse events in our clinical trials, or other clinical trials involving gene therapy products or our competitors’ products, even if not ultimately attributable to the relevant product candidates, and the resulting publicity, could result in increased government regulation, unfavorable public perception, potential regulatory delays in the testing or approval of our product candidates, stricter labeling requirements for those product candidates that are approved and a decrease in demand for any such product candidates.
−Removed: Our success also will depend upon physicians who specialize in the treatment of prescribing treatments that involve the use of our product candidates, respectively, in lieu of, or in addition to, other treatments with which they are more familiar and for which greater clinical data may be available.
−Removed: More restrictive government regulations or negative public opinion would have an adverse effect on our business, financial condition, results of operations and prospects and may delay or impair the development and commercialization of any product candidate we may develop.
−Removed: Serious adverse events in our clinical trials, or other clinical trials involving gene therapy products or our competitors’ products, even if not ultimately attributable to the relevant product candidates, and the resulting publicity, could result in increased government regulation, unfavorable public perception, potential regulatory delays in the testing or approval of our product candidates, stricter labeling requirements for our product candidates if approved and a decrease in demand for our product candidates.
If the market opportunities for our product candidates are smaller than we believe they are, our product revenues may be adversely impacted, and our business may suffer.
5 unchanged sentences
These include the lack of widespread availability of, and limited reimbursement for, new therapies in many underdeveloped markets.
−Removed: Further, the severity of the progression of a disease up to the time of treatment will
−Removed: likely diminish the therapeutic benefit conferred by a gene therapy due to irreversible cell damage.
+Added: Further, the severity of the progression of a disease up to the time of treatment will likely diminish the therapeutic benefit conferred by a gene therapy due to irreversible cell damage.
Lastly, certain patients’ immune systems might prohibit the successful delivery of certain gene therapy products to the target tissue, thereby limiting the treatment outcomes.
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• the efficacy and safety of our product candidates as demonstrated in clinical trials;
−Removed: • the efficacy, potential and perceived advantages of our product candidates over alternative treatments, if available;
+Added: • the potential and perceived advantages of our product candidates over alternative treatments, if available;
• the cost of our product candidates relative to alternative treatments, if any are available;
12 unchanged sentences
Even if a potential product displays a favorable efficacy and safety profile in preclinical studies and clinical trials, market acceptance of the product will not be fully known until after it is launched.
−Removed: Government price controls or other changes in pricing regulation could restrict the amount that we are able to charge for our product candidates, if approved, or any of our other product candidates that may be approved in the future, which would adversely affect our revenue and results of operations.
−Removed: We expect that coverage and reimbursement of pharmaceutical may be increasingly restricted both in the U.S.
+Added: Government price controls or other changes in pricing regulation could restrict the amount that we are able to charge for our product candidates, if approved, which would adversely affect our revenue and results of operations.
+Added: We expect that coverage and reimbursement of pharmaceuticals may be increasingly restricted both in the U.S.
and internationally.
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We cannot predict the extent to which our business may be affected by these or other potential future legislative or regulatory developments.
−Removed: However, future price controls or other changes in pricing regulation or negative publicity related to the pricing of pharmaceutical drugs generally could restrict the amount that we are able to charge for our future products, which would adversely affect our anticipated revenue and results of operations.
+Added: However, future price controls or other changes in pricing regulation or negative publicity related to the pricing of pharmaceutical drugs generally could restrict the amount that we are able to charge for our products, which would adversely affect our anticipated revenue and results of operations.
The insurance coverage and reimbursement status of newly approved products is uncertain.
Failure to obtain or maintain adequate coverage and reimbursement for our products, if approved, could limit our ability to market those products and decrease our ability to generate product revenue.
−Removed: We expect that coverage and reimbursement by government and private payors will be essential for most patients to be able to afford these treatments.
+Added: We expect that coverage and reimbursement by government and private payors will be essential for most patients to be able to afford our product candidates.
Accordingly, sales of our product candidates will depend substantially, both domestically and abroad, on the extent to which the costs of our product candidates will be paid by health maintenance, managed care, pharmacy benefit and similar healthcare management organizations, or will be reimbursed by government authorities, private health coverage insurers and other third-party payors.
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We may not be able to provide data sufficient to gain acceptance with respect to coverage and reimbursement.
−Removed: If coverage and reimbursement are not available, or are available only at limited levels, we may not be able to successfully commercialize our product candidates.
+Added: If coverage and reimbursement are not available, or
+Added: are available only at limited levels, we may not be able to successfully commercialize our product candidates.
Even if coverage is provided, the approved reimbursement amount may not be adequate to realize a sufficient return on our investment.
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Moreover, increasing efforts by government and third-party payors in the United States and abroad to cap or reduce healthcare costs may cause such organizations to limit both coverage and the level of reimbursement for new products approved and, as a result, they may not cover or provide adequate payment for our product candidates.
−Removed: Payors increasingly are considering new metrics as the basis for reimbursement rates, such as average sales price (“ASP”), average manufacturer price (“AMP”), and Actual Acquisition Cost.
−Removed: The existing data for reimbursement based on some of these metrics is relatively limited, although
−Removed: certain states have begun to survey acquisition cost data for the purpose of setting Medicaid reimbursement rates, and CMS has begun making pharmacy National Average Drug Acquisition Cost and National Average Retail Price data publicly available on at least a monthly basis.
+Added: Payors increasingly are considering new metrics as the basis for reimbursement rates, such as average sales price, average manufacturer price, and actual acquisition cost.
+Added: The existing data for reimbursement based on some of these metrics is relatively limited, although certain states have begun to survey acquisition cost data for the purpose of setting Medicaid reimbursement rates, and CMS has begun making pharmacy National Average Drug Acquisition Cost and National Average Retail Price data publicly available on at least a monthly basis.
Therefore, it may be difficult to project the impact of these evolving reimbursement metrics on the willingness of payors to cover candidate products that we or our partners are able to commercialize.
−Removed: We expect to experience pricing pressures in connection with the sale of any of our product candidates due to the trend toward managed healthcare, the increasing influence of health maintenance organizations and additional legislative changes.
+Added: We expect to experience pricing pressures in connection with the sale of any of our product candidates due to the trend toward managed healthcare, the increasing influence of health maintenance organizations and additional potential legislative and administrative changes.
The downward pressure on healthcare costs in general, particularly prescription drugs and surgical procedures and other treatments, has become intense.
As a result, increasingly high barriers are being erected to the entry of new products such as ours.
−Removed: Ethical, legal and social issues related to genetic testing may reduce demand for our products candidate, if approved .
+Added: Ethical, legal and social issues related to genetic testing may reduce demand for our product candidates, if approved .
We anticipate that prior to receiving certain gene therapies, patients may be required to undergo genetic testing.
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For example, concerns have been expressed that insurance carriers and employers may use these tests to discriminate based on genetic information, resulting in barriers to the acceptance of genetic tests by consumers.
−Removed: This could lead to governmental authorities prohibiting genetic testing or calling for limits on or regulating the use of genetic testing, particularly for diseases for which there is no known cure.
+Added: Concerns have also been raised about the accuracy of genetic testing.
+Added: This could lead to governmental authorities restricting genetic testing or calling for additional regulation of genetic testing, particularly for diseases for which there is no known cure.
Any of these scenarios could decrease demand for our product candidates, if approved.
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Further, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries.
−Removed: regulatory approval for any of our product candidates may be withdrawn.
+Added: Also, regulatory approval for any of our product candidates may be withdrawn.
If we fail to comply with the regulatory requirements, our target market will be reduced and our ability to realize the full market potential of our product candidates will be harmed and our business, financial condition, results of operations and prospects will be adversely affected.
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Although certain of our product candidates are currently in clinical or preclinical development, we may fail to identify other potential product candidates for clinical development for several reasons.
−Removed: For example, our research may be
−Removed: unsuccessful in identifying potential product candidates or our potential product candidates may be shown to have harmful side effects, may be commercially impracticable to manufacture or may have other characteristics that may make the products unmarketable or unlikely to receive marketing approval.
+Added: For example, our potential product candidates may be shown to have harmful side effects, may be commercially impracticable to manufacture or may have other characteristics that may make the products unmarketable or unlikely to receive marketing approval.
Additionally, because we have limited resources, we may forego or delay pursuit of opportunities with certain programs or product candidates or for indications that later prove to have greater commercial potential.
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We may be unable to successfully implement these tasks on a larger scale and, accordingly, may not achieve our research, development and growth goals.
−Removed: Our future success depends on our ability to retain key employees and scientific advisors and to attract, retain and motivate qualified personnel.
+Added: Our future success depends on our ability to retain key employees, including scientific and technical personnel, and to attract, retain and motivate qualified personnel.
We are highly dependent on members of our management team, the loss of whose services may adversely impact the achievement of our objectives.
−Removed: Our employees and scientific advisors are at-will employees and consultants, and the loss of one or more of them might impede the achievement of our research, development and commercialization objectives.
−Removed: Recruiting and retaining other qualified employees and scientific advisors for our business, including scientific and technical personnel, also will be critical to our success.
+Added: Our employees are at-will employees, and the loss of one or more of them might impede the achievement of our research, development and commercialization objectives.
+Added: Recruiting and retaining other qualified employees for our business, including scientific and technical personnel, also will be critical to our success.
There currently is a shortage of skilled individuals with substantial gene therapy experience, which is likely to continue.
3 unchanged sentences
The inability to recruit, or loss of services of certain executives, key employees or advisors, may impede the progress of our research, development and commercialization objectives and have a material adverse effect on our business, financial condition, results of operations and prospects.
−Removed: Our employees, principal investigators and advisors may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading.
+Added: Our employees, principal investigators and advisors may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements.
We are exposed to the risk of fraud or other misconduct by our employees, principal investigators and advisors.
3 unchanged sentences
Such misconduct also could involve the improper use of information obtained in the course of clinical trials or interactions with the FDA or other regulatory authorities, which could result in criminal and civil penalties or sanctions and cause serious harm to our reputation.
−Removed: It is not always possible to identify and deter
−Removed: employee misconduct, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from government investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations.
+Added: It is not always possible to identify and deter employee misconduct, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from government investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations.
If any such actions are instituted against us and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business, financial condition, results of operations and prospects, including the imposition of significant fines, criminal penalties, or other sanctions.
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In the United States and some foreign jurisdictions, there have been, and continue to be, several legislative and regulatory changes and proposed changes regarding the healthcare system that could prevent or delay marketing approval of our product candidates, restrict or regulate post-approval activities, and affect our ability to profitably sell any product candidates for which we obtain marketing approval.
+Added: In the U.S., there have been and continue to be a number of legislative efforts to contain healthcare costs.
For example, in March 2010, the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act (“PPACA”), was passed, which substantially changes the way healthcare is financed by both the government and private insurers, and significantly impacts the U.S.
2 unchanged sentences
(i) addresses a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected;
−Removed: (ii) increases the minimum Medicaid rebates owed by manufacturers under the Medicaid Drug Rebate Program and extends the rebate program to individuals enrolled in Medicaid managed care organizations;
+Added: (ii) increases the minimum Medicaid rebates owed by manufacturers under the Medicaid Drug Rebate Program and extends the rebate program to individuals enrolled in Medicaid managed care
+Added: organizations;
(iii) establishes annual fees and taxes on manufacturers of certain branded prescription drugs;
1 unchanged sentence
and (v) establishes a new Medicare Part D coverage gap discount program, in which manufacturers must agree to offer 50% point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D.
−Removed: Since its enactment, there have been judicial and Congressional challenges to certain aspects of the PPACA.
−Removed: As a result, there have been delays in the implementation of, and action taken to repeal or replace, certain aspects of the PPACA.
−Removed: In January 2017, President Trump signed an Executive Order directing federal agencies with authorities and responsibilities under the PPACA to waive, defer, grant exemptions from, or delay the implementation of any provision of the Affordable Care Act (“ACA”) that would impose a fiscal or regulatory burden on states, individuals, healthcare providers, health insurers, or manufacturers of pharmaceuticals or medical devices.
−Removed: On October 13, 2017, President Trump signed an Executive Order terminating the cost-sharing subsidies that reimburse insurers under the ACA.
−Removed: Further, on December 14, 2018, U.S.
−Removed: Court of Appeals for the Federal Circuit ruled that the federal government was not required to pay more than $12 billion in ACA risk corridor payments to third-party payors who argued were owed to them.
−Removed: In addition, CMS has recently proposed regulations that would give states greater flexibility in setting benchmarks for insurers in the individual and small group marketplaces, which may have the effect of relaxing the essential health benefits required under the ACA for plans sold through such marketplaces.
−Removed: Further, each chamber of Congress has put forth multiple bills this year designed to repeal or repeal and replace portions of the ACA.
−Removed: While Congress has not passed repeal legislation, the Tax Reform Act includes a provision repealing,
−Removed: effective January 1, 2019, the tax-based shared responsibility payment imposed by the ACA on certain individuals who fail to maintain qualifying health coverage for all or part of a year that is commonly referred to as the “individual mandate.” Further, the Bipartisan Budget Act of 2018 (“BBA”), among other things, amends the ACA, effective January 1, 2019, to increase from 50 percent to 70 percent the point-of-sale discount that is owed by pharmaceutical manufacturers who participate in Medicare Part D and to close the coverage gap in most Medicare drug plans, commonly referred to as the “donut hole.” Congress may consider other legislation to repeal and replace elements of the ACA.
−Removed: Litigation and legislation over the ACA are likely to continue, with unpredictable and uncertain results.
−Removed: Additionally, in the United States, the Biologics Price Competition and Innovation Act of 2009 created an abbreviated approval pathway for biologic products that are demonstrated to be “highly similar” or “biosimilar or interchangeable” with an FDA-approved biologic product.
−Removed: This new pathway could allow competitors to reference data from biologic products already
−Removed: approved after 12 years from the time of approval.
−Removed: This could expose us to potential competition by lower cost biosimilars even if we commercialize a product candidate faster than our competitors.
−Removed: Moreover, the creation of this abbreviated approval pathway does not preclude or delay a third party from pursuing approval of a competitive product candidate via the traditional approval pathway based on their own clinical trial data.
+Added: Since its enactment, there have been numerous judicial, administrative, executive, and legislative challenges to certain aspects of the PPACA, and we expect there will be additional challenges in the future.
+Added: As a result, there have been delays in the implementation of, and action taken to attempt to repeal or replace, certain aspects of the PPACA.
+Added: Litigation and legislation over the ACA may continue in the future, with unpredictable and uncertain results.
+Added: We cannot predict the impact that such actions against the ACA or other health care reform efforts in the future will have on our business, and there is uncertainty as to what healthcare programs and regulations may be implemented or changed at the federal and/or state level in the United States, or the effect of any future legislation or regulation.
+Added: However, it is possible that such initiatives could have an adverse effect on our ability to obtain approval and/or successfully commercialize products in the United States in the future.
+Added: For example, any changes that reduce, or impede the ability to obtain, reimbursement for the type of products we intend to commercialize in the United States (or our products more specifically, if approved) could adversely affect our business plan to introduce our products in the United States.
Other legislative changes have been proposed and adopted in the United States since the PPACA was enacted.
3 unchanged sentences
In January 2013, the American Taxpayer Relief Act of 2012, among other things, further reduced Medicare payments to certain providers, and increased the time for Medicare contractors to recoup Medicare overpayments to providers from three to five years.
−Removed: Additionally, there have been several recent U.S.
−Removed: Congressional inquiries and proposed bills designed to, among other things, bring more transparency to drug pricing, reduce the cost of prescription drugs under Medicare, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drugs.
−Removed: Further, there has been heightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which have resulted in several recent Congressional inquiries and proposed and enacted bills designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for products.
+Added: Further, there has been heightened governmental scrutiny in recent years over the manner in which manufacturers set prices for their marketed products and the cost of prescription drugs to consumers and government healthcare programs, which have resulted in several recent Congressional inquiries and proposed and enacted bills designed to, among other things, reduce the cost of prescription drugs, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for products.
In addition, the United States government, state legislatures, and foreign governments have shown significant interest in implementing cost containment programs, including price-controls, restrictions on reimbursement and requirements for substitution of generic products for branded prescription drugs to limit the growth of government paid health care costs.
4 unchanged sentences
Additional changes may affect our business, including those governing enrollment in federal healthcare programs, reimbursement changes, fraud and abuse enforcement, and expansion of new programs, such as Medicare payment for performance initiatives.
−Removed: We expect that these initiatives, as well as other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and in additional downward pressure on the price that we receive for any approved product.
+Added: These initiatives, as well as other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and in additional downward pressure on the price that we receive for any approved product.
Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payors.
2 unchanged sentences
If we are unable to comply, or have not fully complied, with such laws, we could face substantial penalties.
−Removed: If we obtain FDA approval for our product candidates and begin commercializing it in the United States, our operations will be directly, or indirectly through our prescribers, customers and purchasers, subject to various federal and state fraud and abuse laws and regulations, including, without limitation, the federal Anti-Kickback Statute, federal civil and criminal false claims laws and the Physician Payments Sunshine Act and regulations.
+Added: If we obtain FDA approval for our product candidates and begin commercializing in the United States, our operations will be directly, or indirectly through our prescribers, customers and purchasers, subject to various federal and state fraud and abuse laws and regulations, including, without limitation, the federal Anti-Kickback Statute, federal civil and criminal false claims laws and the Physician Payments Sunshine Act and regulations.
These laws will impact, among other things, our proposed sales, marketing and educational programs.
1 unchanged sentence
The laws that will affect our operations include, but are not limited to:
−Removed: • the federal Anti-Kickback Statute, which prohibits, among other things, persons or entities from knowingly and willfully soliciting, receiving, offering or paying any remuneration (including any kickback, bribe or rebate), directly or indirectly, overtly or covertly, in cash or in kind, in return for the purchase, recommendation, leasing or furnishing of an
−Removed: item or service reimbursable under a federal healthcare program, such as the Medicare and Medicaid programs.
+Added: • the federal Anti-Kickback Statute, which prohibits, among other things, persons or entities from knowingly and willfully soliciting, receiving, offering or paying any remuneration (including any kickback, bribe or rebate), directly or indirectly, overtly or covertly, in cash or in kind, in return for the purchase, recommendation, leasing or furnishing of an item or service reimbursable under a federal healthcare program, such as the Medicare and Medicaid programs.
This statute has been interpreted to apply to arrangements between pharmaceutical manufacturers on the one hand, and prescribers, purchasers and formulary managers on the other.
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If our operations are found to be in violation of any of the laws described above or any other government regulations that apply to us, we may be subject to penalties, including civil and criminal penalties, damages, fines, exclusion from participation in government health care programs, such as Medicare and Medicaid, imprisonment and the curtailment or restructuring of our operations, any of which could adversely affect our ability to operate our business and our results of operations.
+Added: Often, to avoid the threat of treble
+Added: damages and penalties under the False Claims Act, health care providers will resolve allegations in a settlement without admitting liability to avoid the potential of treble damages.
+Added: Any such settlement could materially affect our business, financial operations, and reputation.
Efforts to ensure that our business arrangements with third parties will comply with applicable healthcare laws and regulations will involve substantial costs.
It is possible that governmental authorities will conclude that our business practices may not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations.
−Removed: If our operations are found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal and administrative penalties, damages, fines, imprisonment, exclusion of products from government funded healthcare programs, such as Medicare and Medicaid, and the curtailment or restructuring of our operations.
The risk of our being found in violation of these laws is increased by the fact that many of them have not been fully interpreted by the regulatory authorities or the courts, and their provisions are open to a variety of interpretations.
Any action against us for violation of these laws, even if we successfully defend against it, could cause us to incur significant legal expenses and divert our management’s attention from the operation of our business.
−Removed: The shifting compliance environment and the need to build and
−Removed: maintain a robust and expandable systems to comply with multiple jurisdictions with different compliance and/or reporting requirements increases the possibility that a healthcare company may run afoul of one or more of the requirements.
+Added: The shifting compliance environment and the need to build and maintain a robust and expandable systems to comply with multiple jurisdictions with different compliance and/or reporting requirements increases the possibility that a healthcare company may run afoul of one or more of the requirements.
If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.
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Substantially all our current supply of our product candidates is located at our manufacturing facility in Pittsburgh, Pennsylvania.
−Removed: We were are in the early stages of constructing an additional manufacturing facility and establishing a relationship with a third-party contract manufacturer as a back-up supplier for the commercial supply of our products, if necessary.
+Added: We are constructing an additional manufacturing facility and establishing a relationship with a third-party contract manufacturer as a back-up supplier for the commercial supply of our products, if necessary.
We may incur substantial expenses as a result of the limited nature of our disaster recovery and business continuity plans, which could have a material adverse effect on our business, financial condition, results of operations and prospects.
9 unchanged sentences
10,441,614 covering aspects of our vector platform technology, and its uses in delivering any gene of interest to the skin;
−Removed: 10,525,090, covering pharmaceutical compositions comprising our second clinical product candidate, KB105, and methods of its use for treating TGM1-deficient autosomal recessive congenital ichthyosis;
+Added: 10,525,090, covering pharmaceutical compositions comprising our second clinical product candidate, KB105, and methods of its use for treating TGM1-deficient autosomal recessive
+Added: congenital ichthyosis;
10,786,438 covering pharmaceutical compositions comprising vectors encoding cosmetic proteins, including our third product candidate, KB301, and methods for their use for improving skin condition, quality, and/or appearance;
10,829,529 covering the methods of using KB407 for the treatment of cystic fibrosis and other diseases causing progressive lung destruction.
−Removed: Furthermore, we have nine international patent applications filed in accordance with the Paris Cooperation treaty directed to multiple discovery, preclinical, and clinical programs, including both B-VEC, KB105, KB301, KB104 and KB407, as well as multiple patent applications filed in foreign jurisdictions stemming from these international applications.
−Removed: B-VEC is also the subject of patents granted in both Australia and Europe, including European Patent No.
+Added: Furthermore, we have international patent applications filed in accordance with the Paris Cooperation treaty directed to multiple discovery, preclinical, and clinical programs, including B-VEC, KB105, KB301, KB104, KB407, and KB-408, as well as multiple patent applications filed in foreign jurisdictions stemming from these international applications.
+Added: B-VEC is also the subject of multiple patents granted in foreign jurisdictions, including European Patent No.
3 377 637 B1, covering pharmaceutical compositions containing B-VEC as well as uses thereof.
1 unchanged sentence
Our competitors, or other third parties, may be able to circumvent our patents by developing similar or alternative technologies or products in a non-infringing manner.
−Removed: For example, there is no assurance that the ’990 patent, or any other patent we are granted, will prevent third parties from developing competing
−Removed: technologies.
+Added: For example, there is no assurance that the ’990 patent, or any other patent we are granted, will prevent third parties from developing competing technologies.
Moreover, our patent estate, including the ’990 patent, does not preclude third parties from having intellectual property rights that could interfere with our freedom to use our platform, including for dermatological indications.
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Our commercial success depends upon our ability (and the ability of any potential future collaborators) to develop, manufacture, market and sell our product candidates, and to freely use our proprietary technologies ( e.g.
−Removed: , without infringing the rights and intellectual property of others).
+Added: , without infringing the
+Added: rights and intellectual property of others).
Many companies and institutions have filed, and continue to file, patent applications related to various aspects of gene therapy.
1 unchanged sentence
The biotechnology and pharmaceutical industries are characterized by extensive and complex litigation regarding patents and other intellectual property rights.
−Removed: We may in the future become party to, or be threatened with, adversarial proceedings or litigation
−Removed: regarding intellectual property rights with respect to our product candidates or related technologies, including, for example, interference proceedings, post grant review challenges, and inter partes review before the USPTO.
+Added: On May 1, 2020, a complaint was filed against us in the United States District Court for the Western District of Pennsylvania by PeriphaGen Inc., which also named our Chief Executive Officer and Chief Operating Officer, Krish Krishnan and Suma Krishnan, respectively.
+Added: The complaint alleges breach of contract and misappropriation of trade secrets, which secrets the plaintiff asserts were used to develop our product candidates, including the vector backbones, and our STAR-D platform.
+Added: For more information, see "Legal Proceedings" in Note 6 of the Notes to Condensed Consolidated Financial Statements included in Item 1 of Part I of this Form 10-Q.
+Added: We may in the future become party to, or be threatened with, other adversarial proceedings or litigation regarding intellectual property rights with respect to our product candidates or related technologies, including, for example, interference proceedings, post grant review challenges, and inter partes review before the United States Patent and Trademark Office ("USPTO").
For example, a third party may bring an inter partes review challenging our patents and any future patent that may be granted to us.
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We may not have sufficient financial or other resources to adequately conduct such litigation or proceedings.
−Removed: Some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their greater financial resources.
+Added: Some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their
+Added: greater financial resources.
Accordingly, despite our efforts, we may not be able to prevent third parties from infringing, misappropriating, or successfully challenging our intellectual property rights.
Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could have a material adverse effect on our ability to compete in the marketplace.
−Removed: We are be subject to claims asserting that we, our employees or our advisors have wrongfully used or disclosed alleged trade secrets of other parties, including current or former employers, or claims asserting ownership of what we regard as our own intellectual property and we may face other such claims in the future.
−Removed: Certain of our employees or advisors are currently, or were previously, employed at universities or other biotechnology or pharmaceutical companies, including potential competitors, and we have and may in the future enter into agreements providing us with rights to intellectual property of third parties for limited purposes.
+Added: We are and may be subject to claims asserting that we, our employees or our advisors have wrongfully used or disclosed alleged trade secrets of other parties, including current or former employers, or claims asserting ownership of what we regard as our own intellectual property.
+Added: Certain of our employees or advisors are currently, or were previously, employed at universities or other biotechnology or pharmaceutical companies, including potential competitors, and we have and may in the future entered into agreements providing us with rights to intellectual property of third parties for limited purposes.
Although we try to observe the terms of agreements under which we obtain access to third party intellectual property and to ensure that our employees and advisors do not use the proprietary information or know-how of others in their work for us, we may be subject to claims that these individuals, or we, have used or disclosed intellectual property, including trade secrets or other proprietary information, of third parties or the current or former employers of employees or advisors.
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We cannot assure you that our efforts to seek patent protection for our technology and product candidates will not be negatively impacted by the future court decisions or changes in guidance or procedures issued by the USPTO.
−Removed: These decisions, and any guidance issued by the USPTO (or changes thereto), could have a material adverse effect on our existing patent portfolio and our ability to protect and enforce our intellectual property rights in the future.
+Added: These decisions, and any guidance issued by the USPTO
+Added: (or changes thereto), could have a material adverse effect on our existing patent portfolio and our ability to protect and enforce our intellectual property rights in the future.
If our trademarks and trade names are not adequately protected, then we may not be able to build name recognition in our markets of interest and our business may be adversely affected.
−Removed: We currently have not registered our trademarks and trade names.
+Added: We are in the process of registering certain of our trademarks and trade names.
Once trademarks or trade names have been registered, they may be challenged, infringed, circumvented or declared generic or determined to be infringing on other marks.
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Our Chief Executive Officer and Chairman of the Board of Directors and our founder, Chief Operating Officer and director will have the ability to substantially influence all matters submitted to stockholders for approval.
−Removed: As of March 31, 2021, Krish S.
+Added: As of June 30, 2021, Krish S.
Krishnan and Suma M.
Krishnan, our Chief Executive Officer and Chairman of the Board and our founder, Chief Operating Officer and director, respectively, in the aggregate, beneficially owned shares representing approximately 17% of our capital stock.
−Removed: As a result, they will be able to substantially influence all matters submitted to our stockholders for approval, as well as our management and affairs.
+Added: As a result, they will be able to substantially influence all matters submitted to our
+Added: stockholders for approval, as well as our management and affairs.
For example, these persons would substantially influence the election of directors and approval of any merger, consolidation or sale of all or substantially all our assets.
21 unchanged sentences
• disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain patent protection for our technologies;
−Removed: • significant lawsuits, including patent or stockholder litigation;
+Added: • significant lawsuits, including intellectual property or stockholder litigation;
• variations in our financial results or those of companies that are perceived to be similar to us;
4 unchanged sentences
We are an “emerging growth company” and the reduced disclosure requirements applicable to emerging growth companies may make our common stock less attractive to investors.
−Removed: We are an “emerging growth company,” as defined in the JOBS Act, and we may take advantage of certain exemptions and relief from various reporting requirements that are applicable to other public companies that are not “emerging growth companies.” In particular, while we are an “emerging growth company:
+Added: We are an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act ("JOBS Act"), and we may take advantage of certain exemptions and relief from various reporting requirements that are applicable to other public companies that are not “emerging growth companies.” In particular, while we are an “emerging growth company:
(i) we will not be required to comply with the auditor attestation requirements of Section 404(b) of the Sarbanes-Oxley Act;
(ii) we will be exempt from any rules that may be adopted by the Public Company Accounting Oversight Board requiring mandatory audit firm rotations or a supplement to the auditor’s report on financial statements;
−Removed: (iii) we will be subject to reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements;
+Added: (iii) we will be subject to reduced disclosure
+Added: obligations regarding executive compensation in our periodic reports and proxy statements;
and (iv) we will not be required to hold nonbinding advisory votes on executive compensation or stockholder approval of any golden parachute payments not previously approved.
4 unchanged sentences
We have irrevocably elected not to avail ourselves of this exemption from new or revised accounting standards and, therefore, we will be subject to the same new or revised accounting standards as other public companies that are not emerging growth companies.
−Removed: We will continue to incur costs as a result of becoming a public company, and such costs may increase if and when we cease to be an “emerging growth company.”
+Added: We will continue to incur costs as a result of becoming a public company, and such costs will increase when we cease to be an “emerging growth company.”
As a public company, we expect to continue to incur significant legal, accounting, insurance and other expenses, including costs associated with public company reporting requirements.
The expenses incurred by public companies generally for reporting and corporate governance purposes have been increasing.
−Removed: We expect compliance with these public reporting requirements and associated rules and regulations to increase expenses, particularly after we are no longer an emerging growth company, although we are currently unable to estimate these costs with any degree of certainty.
−Removed: We could be an emerging growth
−Removed: company until the end of 2021, after which, we will incur additional costs applicable to public companies that are not emerging growth companies.
+Added: We expect compliance with these public reporting requirements and associated rules and regulations to increase expenses, particularly after we are no longer an emerging growth company beginning in 2022, although we are currently unable to estimate these costs with any degree of certainty.
+Added: We will be an emerging growth company until the end of 2021, after which, we will incur additional costs applicable to public companies that are not emerging growth companies.
If we fail to maintain effective internal control over financial reporting, we may not be able to accurately report our financial results, which may adversely affect investor confidence in our company and, as a result, the value of our common stock.
Our management is responsible for establishing and maintaining adequate internal control over financial reporting.
−Removed: After we are no longer an emerging growth company under the JOBS Act, beginning no later than our year ending December 31, 2023, Section 404 of the Sarbanes-Oxley Act requires our auditors to deliver an attestation report on the effectiveness of our internal control over financial reporting in conjunction with their opinion on our audited financial statements.
+Added: Beginning with our fiscal year ending December 31, 2022, we will cease to be an "emerging growth company" as defined by the JOBs Act.
+Added: After we are no longer an emerging growth company under the JOBS Act, Section 404 of the Sarbanes-Oxley Act requires our auditors to deliver an attestation report on the effectiveness of our internal control over financial reporting in conjunction with their opinion on our audited financial statements.
Substantial work on our part is required to implement appropriate processes, document the system of internal control over key processes, assess their design, remediate any deficiencies identified and test their operation.
5 unchanged sentences
Provisions in our corporate charter documents and under Delaware law could make an acquisition of us, which may be beneficial to our stockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current management.
−Removed: Provisions in our corporate charter and our bylaws may discourage, delay or prevent a merger, acquisition or other change in control of us that stockholders may consider favorable, including transactions in which you might otherwise receive a premium for your shares.
+Added: Provisions in our corporate charter and our bylaws may discourage, delay or prevent a merger, acquisition or other change in control of us that stockholders may consider favorable, including transactions in which stakeholders might otherwise receive a premium for their shares.
These provisions also could limit the price that investors might be willing to pay in the future for shares of our common stock, thereby depressing the market price of our common stock.
29 unchanged sentences
If we fail to satisfy the expectations of investors and other stakeholders or our initiatives are not executed as planned, our reputation and financial results could be adversely affected
−Removed: Cyber-security incidents, including data security breaches or computer viruses, could harm our business by disrupting our delivery of services, damaging our reputation or exposing us to liability.
−Removed: We receive, process, store, and transmit, often electronically, confidential data of others.
−Removed: Unauthorized access to our computer systems or stored data could result in the theft or improper disclosure of confidential information, the deletion or modification of records, or could cause interruptions in our operations.
−Removed: These cyber-security risks increase when we transmit information from one location to another, including transmissions over the Internet or other electronic networks.
−Removed: Despite implemented security measures, our facilities, systems, and procedures, and those of our third-party service providers, may be vulnerable to security breaches, acts of vandalism, software viruses, misplaced or lost data, programming and/or human errors, or other similar events which may disrupt our delivery of services or expose the confidential information of our customers and others.
−Removed: Any security breach involving the misappropriation, loss or other unauthorized disclosure or use of confidential information of others, whether by us or a third party, could:
−Removed: (i) subject us to civil and criminal penalties;
−Removed: (ii) have a negative impact on our reputation;
−Removed: or (iii) expose us to liability to our customers, third parties or government authorities.
−Removed: Any of these developments could have a material adverse effect on our business, financial condition, and results of operations.
UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS
4 unchanged sentences
Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.