25 unchanged sentences
We have devoted most of our financial resources to research and development, including our clinical and preclinical development activities.
−Removed: To date, we and our predecessor have financed our operations primarily through the issuance of equity securities, research and development grants from the Australian government and payments from our collaboration partners.
−Removed: not expect to generate any significant revenue for the foreseeable future, and we expect to incur significant operating losses for the foreseeable future due to the cost of research and development, preclinical studies and clinical trials and the regulatory approval process for product candidates.
+Added: To date, we and our predecessor have financed our operations primarily through the issuance of equity securities, research
+Added: and development grants from the Australian government and payments from our collaboration partners.
+Added: We do not expect to generate any significant revenue for the foreseeable future, and we expect to incur significant operating losses for the foreseeable future due to the cost of research and development, preclinical studies and clinical trials and the regulatory approval process for product candidates.
The amount of our future net losses is uncertain and will depend, in part, on the rate of our future expenditures.
3 unchanged sentences
continue our research and preclinical development of our product candidates;
−Removed: expand the scope of our current preclinical studies for our product candidates or initiate clinical, additional preclinical or other studies for product candidates;
+Added: expand the scope of our current preclinical and clinical studies for our product candidates or initiate clinical, additional preclinical or other studies for product candidates;
seek regulatory and marketing approvals for any of our product candidates that successfully complete clinical trials;
10 unchanged sentences
Our ability to generate significant revenue and achieve profitability depends on our ability to, alone or with strategic collaboration partners, successfully complete the development of and obtain the regulatory approvals for our product candidates, to manufacture sufficient supply of our product candidates, to establish a sales and marketing organization or suitable third-party alternative for the marketing of any approved products and to successfully commercialize any approved products on commercially reasonable terms.
−Removed: All of these activities will require us to raise sufficient funds to finance business activities.
−Removed: In the past we received milestone payments from our collaborative partners.
−Removed: However, we do not anticipate generating revenue from commercializing product candidates for the foreseeable future, if ever.
Our ability to generate future revenues from commercializing product candidates depends heavily on our success in:
22 unchanged sentences
Failure to obtain capital when needed may negatively impact our ability to continue as a going concern.
−Removed: Developing ddRNAi products is expensive, and we expect our research and development expenses to increase substantially in connection with our ongoing activities, particularly as we advance our product candidates in preclinical studies and clinical trials and as we undertake preclinical studies of new product candidates.
−Removed: We estimate that our cash and cash equivalents will be sufficient to fund the Company’s operations for at least the next twelve months after the date that this Annual Report is filed.
Because the length of time and activities associated with successful development of our product candidates is highly uncertain, we are unable to estimate the actual funds we will require for development and any approved marketing and commercialization activities.
In any event, we will require additional capital to obtain regulatory approval for our product candidates and to commercialize any product candidates that receive regulatory approval.
+Added: We estimate that our cash and cash equivalents will be sufficient to fund the Company’s operations for at least the next twelve months after the date that this Annual Report is filed.
Any additional fundraising efforts may divert our management from their day-to-day activities, which may compromise our ability to develop and commercialize our product candidates.
In addition, we cannot guarantee that future financing will be available in sufficient amounts or on terms acceptable to us, if at all.
−Removed: Moreover, the terms of any financing may adversely affect the holdings or the rights of our stockholders, and the issuance of additional securities, whether equity or debt, by us, or the possibility of such issuance, may cause the market price of our common stock to decline.
+Added: Moreover, the
+Added: terms of any financing may adversely affect the holdings or the rights of our stockholders, and the issuance of additional securities, whether equity or debt, by us, or the possibility of such issuance, may cause the market price of our common stock to decline.
If we incur indebtedness we may be required to agree to restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell or license intellectual property rights and other operating restrictions that could compromise our ability to conduct our business.
5 unchanged sentences
If we seek additional financing to fund our business activities in the future and there remains substantial doubt about our ability to continue as a going concern, investors or other financing sources may be unwilling to provide additional funding to us on commercially reasonable terms, if at all.
−Removed: We may be unable to conduct and conclude clinical trials for our product candidate for OPMD if we are unable to raise additional financing.
−Removed: If we are unable to raise additional funds, and thus unable to continue our studies, our business operations may be adversely affected.
Risks Related to the Product Development and Regulatory Approval of Our Product Candidates
26 unchanged sentences
The numbers, types and sizes of preclinical studies and clinical trials that will be required for regulatory approval varies depending on the product candidate, the disease or condition that the product candidate is designed to address and the regulations applicable to any particular product candidate.
−Removed: Approval policies, regulations or the type and amount of clinical data necessary to gain marketing approval may change during the course of a product candidate’s clinical development and may vary among jurisdictions, and there may be varying interpretations of data obtained from preclinical studies or clinical trials, any of which may cause
−Removed: delays or limitations in the marketing approval or the decision not to approve an application.
+Added: Approval policies, regulations or the type and amount of clinical data necessary to gain marketing approval may change during the course of a product candidate’s clinical development and may vary among jurisdictions, and there may be varying interpretations of data obtained from preclinical studies or clinical trials, any of which may cause delays or limitations in the marketing approval or the decision not to approve an application.
Regulatory agencies can delay, limit or deny marketing approval of a product candidate for many reasons, including:
1 unchanged sentence
we may be unable to demonstrate to the satisfaction of the FDA or comparable foreign regulatory authorities that a product candidate is safe and effective for its proposed indication;
−Removed: the results of any future clinical trials may not meet the level of statistical or clinical significance required by the FDA or comparable foreign regulatory authorities for approval;
+Added: the results of clinical trials may not meet the level of statistical or clinical significance required by the FDA or comparable foreign regulatory authorities for approval;
the patients recruited for a particular clinical program may not be sufficiently broad or representative to assure safety and effectiveness in the full population for which we seek approval;
−Removed: the results of any future clinical trials may not confirm the positive results from earlier preclinical studies or clinical trials;
+Added: the results of clinical trials may not confirm the positive results from earlier preclinical studies or clinical trials;
we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh its safety risks;
the FDA or comparable foreign regulatory authorities may disagree with our interpretation of data from preclinical studies or clinical trials;
−Removed: the data collected from any future clinical trials of our product candidates may not be sufficient to the satisfaction of FDA or comparable foreign regulatory authorities to support the submission of a biologics license application, or BLA, or other comparable submission in foreign jurisdictions or to obtain regulatory approval in the United States or elsewhere;
+Added: the data collected from clinical trials of our product candidates may not be sufficient to the satisfaction of FDA or comparable foreign regulatory authorities to support the submission of a biologics license application, or BLA, or other comparable submission in foreign jurisdictions or to obtain regulatory approval in the United States or elsewhere;
the FDA or comparable foreign regulatory authorities may only agree to approve a product candidate under conditions that are so restrictive that the product is not commercially viable;
23 unchanged sentences
Because our ddRNAi and silence and replace product candidates are considered gene therapies, it is difficult to predict the time and cost of product candidate development as well as subsequently obtaining regulatory approval.
−Removed: The clinical trial requirements of the FDA and comparable foreign regulatory authorities and the criteria these regulators use to determine the safety and efficacy of a product candidate vary substantially according to the type, complexity, novelty and intended use and market of such product candidates.
+Added: The clinical trial requirements of the FDA and comparable foreign regulatory authorities and the criteria these regulators use to determine the safety and efficacy of a product candidate vary substantially according to the
+Added: type, complexity, novelty and intended use and market of such product candidates.
The regulatory approval process for novel product candidates such as ours can be more expensive and take longer than for other pharmaceutical product candidates.
5 unchanged sentences
In addition, adverse developments in clinical trials of gene therapy products conducted by others may cause the FDA or comparable foreign regulatory bodies to change the requirements for approval of any of our product candidates.
−Removed: These committees and advisory groups and the new guidelines they promulgate and new requirements they may impose may lengthen the clinical development and 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 these product candidates or lead to significant post-approval limitations or restrictions.
−Removed: As we advance our product candidates, we will be required to consult with these regulatory committees and advisory groups, and comply with applicable guidelines and requirements as they may change from time to time.
−Removed: If we fail to do so, we may be required to delay or discontinue development of our product candidates.
−Removed: These additional processes may result in a development, review and approval process that is longer than we otherwise would have expected for our product candidates.
Delay or failure to obtain, or unexpected costs in obtaining, the regulatory approval necessary to bring a potential product to market would delay or prevent us from commercializing our product candidates, generating revenues and achieving and sustaining profitability.
11 unchanged sentences
AAV vectors can also trigger immune responses in some patients, and those patients will not derive clinical benefit from administration of a product candidate unless steps are taken to clinically address the issue.
−Removed: If we or our collaborators are not successful in identifying effective delivery methodologies to achieve a therapeutically relevant concentration for our product candidates in the target tissues, we may not succeed in developing marketable products.
+Added: If we or our collaborators are not successful in identifying effective delivery methodologies to achieve a therapeutically relevant concentration for our product candidates in
+Added: the target tissues, we may not succeed in developing marketable products.
In addition, if we are unable to reach agreement on favorable terms, or at all, with providers of any effective vectors that we do identify, we may not succeed in completing our clinical trials or commercializing our products on a timely or profitable basis, if at all.
−Removed: We use AAV vectors as part of our ddRNAi and silence and replace approaches for several indications.
−Removed: As such, we require licenses and the ability to manufacture large quantities of AAV particles under the FDA’s current good manufacturing practices, or cGMP, requirements and those of comparable foreign regulatory authorities in order to commercialize a product candidate using an AAV vector.
−Removed: We may find it difficult to enroll patients in any future clinical trials, and patients could discontinue their participation in our current and any future clinical trials, which could delay or prevent our current and any future clinical trials of our product candidates and make those trials more expensive to undertake.
−Removed: Identifying and qualifying patients to participate in current and any future clinical trials of our product candidates is critical to our success.
+Added: We may find it difficult to enroll patients in our clinical trials, and patients could discontinue their participation in our current and any future clinical trials, which could delay or prevent our clinical trials of our product candidates and make those trials more expensive to undertake.
+Added: Identifying and qualifying patients to participate in clinical trials of our product candidates is critical to our success.
The timing of our clinical trials depends on the speed at which we can recruit patients to participate in testing our product candidates.
−Removed: Patients may be unwilling to participate in any future clinical trials because of negative publicity from adverse events in the biotechnology, RNAi or gene therapy industries.
+Added: Patients may be unwilling to participate in any clinical trials because of negative publicity from adverse events in the biotechnology, RNAi or gene therapy industries.
Patients may be unavailable for other reasons, including competitive clinical trials for similar patient populations, and the timeline for recruiting patients, conducting trials and obtaining regulatory approval of potential products may be delayed.
1 unchanged sentence
Clinical trial delays could result in increased costs, slower product development, setbacks in testing the safety and effectiveness of our technology or discontinuation of the clinical trials altogether.
−Removed: We may not be able to identify, recruit and enroll a sufficient number of patients, or those with required or desired characteristics to achieve diversity in a trial, to complete any future clinical trials in a timely manner.
+Added: We may not be able to identify, recruit and enroll a sufficient number of patients, or those with required or desired characteristics to achieve diversity in a trial, to complete any clinical trials in a timely manner.
Patient enrollment is affected by factors including:
10 unchanged sentences
ability to monitor patients adequately during and after treatment.
−Removed: We or any potential collaborators plan to seek initial marketing approval for our product candidates in the United States, Canada, Europe and Israel.
−Removed: We may not be able to initiate or continue any future clinical trials in a timely manner if we cannot enroll a sufficient number of eligible patients to participate in the clinical trials required by the FDA or comparable foreign regulatory agencies.
+Added: We or any potential collaborators plan to seek initial marketing approval for our product candidates in the United States and other regions and countries.
+Added: We may not be able to initiate or continue clinical trials in a timely manner if we cannot enroll a sufficient number of eligible patients to participate in the clinical trials required by the FDA or comparable foreign regulatory agencies.
Our ability to successfully initiate, enroll and complete a clinical trial in any foreign country is subject to numerous risks unique to conducting business in foreign countries, including:
3 unchanged sentences
the potential burden of complying with a variety of foreign laws, medical standards and regulatory requirements, including the regulation of biopharmaceutical and biotechnology products and treatments.
−Removed: In addition, patients enrolled in current and any future clinical trials may discontinue their participation at any time during the trial as a result of a number of factors, including experiencing adverse events, which may or may not be judged related to our product candidates under evaluation.
+Added: In addition, patients enrolled in clinical trials may discontinue their participation at any time during the trial as a result of a number of factors, including experiencing adverse events, which may or may not be judged related to our product candidates under evaluation.
The discontinuation of patients in any one of our trials may cause us to delay or discontinue our clinical trial, or cause the results from that trial not to be positive or sufficient to support either partnering with a pharmaceutical or biotechnology company to further develop and commercialize the product candidate or filing for regulatory approval of the product candidate.
−Removed: We may encounter substantial delays in any future clinical trials or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.
+Added: We may encounter substantial delays in our clinical trials or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.
Before obtaining marketing approval from regulatory authorities for the sale of any of our product candidates, we must conduct extensive clinical trials to demonstrate the safety and efficacy of the product candidates in humans.
Clinical trials are expensive and time-consuming, and their outcome is uncertain.
−Removed: We cannot guarantee that any
−Removed: clinical trials will be initiated or conducted as planned or completed on schedule, if at all.
+Added: We cannot guarantee that any particular clinical trials will be initiated or conducted as planned or completed on schedule, if at all.
A failure of one or more clinical trials can occur at any stage of testing.
24 unchanged sentences
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 impair our ability to successfully commercialize our product candidates.
−Removed: If the results of our current or any future clinical trials are inconclusive or if there are safety concerns or adverse events associated with our product candidates, we may:
+Added: If the results of our clinical trials are inconclusive or if there are safety concerns or adverse events associated with our product candidates, we may:
fail to obtain, or be delayed in obtaining, marketing approval for our product candidates;
14 unchanged sentences
Treatment with our product candidates may produce undesirable side effects or adverse reactions or events.
−Removed: For example, the AAV vector and related capsid protein, which we are currently using to deliver many of our ddRNAi and silence and replace product candidates, could cause adverse immunological side effects due to
−Removed: preexisting and/or recall responses to the naturally occurring virus from which the vector is engineered, or to the DNA construct product itself.
+Added: For example, the AAV vector and related capsid protein, which we are currently using to deliver many of our ddRNAi and silence and replace product candidates, could cause adverse immunological side effects due to preexisting and/or recall responses to the naturally occurring virus from which the vector is engineered, or to the DNA construct product itself.
These responses may also interfere with therapeutic efficacy if not identified and managed optimally.
5 unchanged sentences
Additionally, the procedure used to administer the treatment could result in undesirable health effects.
−Removed: If any such adverse events occur, our current and any future clinical trials could be suspended or discontinued and the FDA or comparable foreign regulatory authorities could order us to cease further development or deny approval of our product candidates for any or all targeted indications.
+Added: If any such adverse events occur, our clinical trials could be suspended or discontinued and the FDA or comparable foreign regulatory authorities could order us to cease further development or deny approval of our product candidates for any or all targeted indications.
The product-related side effects could affect patient recruitment or the ability of enrolled patients to complete the trial.
24 unchanged sentences
If any of these events were to occur, our business would be harmed.
−Removed: Even if we complete the necessary preclinical studies and clinical trials, we cannot predict when or if we will obtain regulatory approval to commercialize a product candidate or the approval may be for a narrower indication than we expect.
+Added: Even if we complete the necessary preclinical studies and clinical trials for a product candidate, we cannot predict when or if we will obtain regulatory approval to commercialize a product candidate or the approval may be for a narrower indication than we expect.
We cannot commercialize a product until the appropriate regulatory authorities have reviewed and approved the product candidate.
1 unchanged sentence
Additional delays may result if an FDA Advisory Committee or comparable foreign 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 agency 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
+Added: changes in regulatory agency policy during the period of product development, clinical trials and the review process.
Regulatory agencies may also approve a treatment candidate for fewer or more limited indications than requested, may not approve the price we intend to charge for our product candidate, may limit our ability to promote the product, may impose significant limitations upon the approval of the product, including, but not limited to, narrow indications, significant warnings, precautions or contraindications with respect to conditions of use, or may grant approval subject to the performance of post-marketing studies.
2 unchanged sentences
Inadequate funding for the FDA and other government agencies could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
−Removed: The ability of the FDA to review and approve new products can be affected by a variety of factors, including government budget and funding levels, ability to hire and retain key personnel and accept the payment of user
−Removed: fees, and statutory, regulatory, and policy changes.
+Added: The ability of the FDA to review and approve new products can be affected by a variety of factors, including government budget and funding levels, ability to hire and retain key personnel and accept the payment of user fees, and statutory, regulatory, and policy changes.
Average review times at the agency have fluctuated in recent years as a result.
In addition, government funding of other government agencies on which our operations may rely, including those that fund research and development activities is subject to the political process, which is inherently fluid and unpredictable.
−Removed: Disruptions at the FDA and other agencies may also slow the time necessary for new drugs to be reviewed and/or approved by necessary government agencies, which would adversely affect our business.
−Removed: For example, in recent years, including in 2018 and 2019, the U.S.
−Removed: government shut down several times and certain regulatory agencies, such as the FDA, had to furlough critical employees and stop critical activities.
−Removed: Separately, in response to the COVID-19 pandemic, the FDA postponed most inspections of foreign manufacturing facilities and products, postponed routine surveillance inspections of domestic manufacturing facilities and is conducting only teleconference meetings.
−Removed: Regulatory authorities in the United States and outside the United States may adopt similar restrictions or other policy measures in response to any future similar events.
−Removed: If a prolonged government shutdown occurs, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our business.
−Removed: Further, in our operations as a public company, future government shutdowns could impact our ability to access the public markets and obtain necessary capital in order to properly capitalize and continue our operations.
Even if we obtain regulatory approval for a product candidate, our products will remain subject to regulatory scrutiny.
21 unchanged sentences
Even if the FDA grants marketing approval for a product candidate, comparable regulatory authorities of foreign countries must also approve the manufacturing and marketing of the product candidates in those countries.
−Removed: Approval procedures vary among jurisdictions and can involve requirements and administrative review periods different from, and greater than, those in the United States, including additional preclinical studies or clinical trials.
−Removed: In many countries outside the United States, a product candidate must be approved for reimbursement before it can be approved for sale in that country.
−Removed: In some cases, the price that we intend to charge for our products, if approved, is also subject to approval.
−Removed: Even if a product candidate is approved, the FDA or comparable regulatory authorities in other countries, as the case may be, may limit the indications for which the product may be marketed, require extensive warnings on the product labeling or require expensive and time-consuming clinical trials or reporting as conditions of approval.
−Removed: Regulatory authorities in countries outside of the United States also have requirements for approval of product candidates with which we must comply prior to marketing in those countries.
+Added: Approval procedures vary among jurisdictions and can involve requirements and administrative review periods different from, and greater than, those in the United States.
Obtaining foreign regulatory approvals and compliance with foreign regulatory requirements could result in significant delays, difficulties and costs for us and could delay or prevent the introduction of our product candidates in certain countries.
3 unchanged sentences
If we fail to comply with the regulatory requirements in international markets and/or fail to receive applicable marketing approvals, our target market will be reduced, our ability to realize the full market potential of our product candidates will be compromised and our business may be adversely affected.
−Removed: Our future prospects may also depend on our collaborators’ ability to successfully develop a pipeline of additional product candidates, and we and our collaborators may not be successful in efforts to use our platform technologies to identify or discover additional product candidates.
+Added: Our future prospects may also depend on our ability to successfully develop a pipeline of additional product candidates beyond our initial product candidate, and we and our collaborators may not be successful in efforts to use our platform technologies to identify or discover additional product candidates.
The success of our business depends primarily upon our ability to identify, develop and commercialize products based on our platform technology.
−Removed: We do not have any products on the market and are early in our development efforts.
−Removed: Our product candidates derived from our platform technology may not successfully complete investigational new drug, or IND, enabling studies, and our research programs may fail to identify other potential product candidates for clinical development for a number of reasons.
−Removed: Our and our collaborators’ research methodology may be unsuccessful in identifying potential product candidates, our potential product candidates
−Removed: may not demonstrate the necessary preclinical outcomes to progress to clinical studies, or our product candidates may be shown to have harmful side effects or may have other characteristics that may make the products unmarketable or unlikely to receive marketing approval.
+Added: Our and our collaborators’ research methodology may be unsuccessful in identifying potential product candidates beyond our initial product candidate, BB-301, additional potential product candidates may not demonstrate the necessary preclinical outcomes to progress to clinical studies, or additional product candidates may be shown to have harmful side effects or may have other characteristics that may make the products unmarketable or unlikely to receive marketing approval.
If any of these events occur, we may be forced to discontinue our development efforts for a program or programs.
15 unchanged sentences
Risks Related to Our Reliance on Third Parties
−Removed: Our prospects for successful development and commercialization of our products are dependent to varying degrees upon the research, development, commercialization, and marketing efforts of any potential collaborators.
+Added: Our prospects for successful development and commercialization of our products are dependent to varying degrees upon the research, development, commercialization, and marketing efforts of our collaborators.
We rely on third parties for certain aspects of the research, development, commercialization and marketing of our current and any future product candidates.
−Removed: Other than as provided for in our collaboration agreements, we have no control over the resources, time and effort that our collaborators may devote to the development of product
+Added: Other than as provided for in our collaboration agreements, we have no control over the resources, time and effort that our collaborators may devote to the development of product candidates.
We are dependent on our collaborators to conduct some aspects of the research and development of each of our product candidates, and expect to need access to them to facilitate and/or to complete the regulatory process.
18 unchanged sentences
We rely on third parties to conduct our preclinical studies and clinical trials.
−Removed: We do not have the ability to conduct all aspects of our preclinical testing or any future clinical trials ourselves.
−Removed: We are dependent on third parties to conduct the preclinical studies for our product candidates and will depend on third parties to conduct any future clinical trials for our product candidates, and therefore the timing of the initiation and completion of these trials and studies is reliant on third parties and may occur at times substantially
−Removed: different from our estimates or expectations.
−Removed: In the case of clinical trials, we expect to rely on CROs and third-party collaborators to conduct any future clinical trials in accordance with our clinical protocols and regulatory requirements.
−Removed: We expect our CROs, investigators and third-party collaborators will play a significant role in the conduct of these trials and subsequent collection and analysis of data.
−Removed: There is no guarantee that any CROs, investigators or the other third-party collaborators on which we rely for administration and conduct of our future clinical trials will devote adequate time and resources to such trials or perform as contractually required.
−Removed: If any of these third parties fails to meet expected deadlines, fails to adhere to our clinical protocols, fails to meet regulatory requirements or otherwise performs in a substandard manner, any future clinical trials may be extended, delayed or terminated.
−Removed: If our current or any of our future clinical trial sites terminates for any reason, we may lose all of the information on subjects enrolled in any such clinical trials.
+Added: We do not have the ability to conduct all aspects of our preclinical testing or clinical trials ourselves.
+Added: We are dependent on third parties to conduct the preclinical studies for our product candidates and to conduct clinical trials for our product candidates, and therefore the timing of the initiation and completion of these trials and studies is reliant on third parties and may occur at times substantially different from our estimates or expectations.
+Added: In the case of clinical trials, we rely on CROs and other third-party collaborators to conduct clinical trials in accordance with our clinical protocols and regulatory requirements.
+Added: Our CROs, investigators and third-party collaborators play a significant role in the conduct of these trials and subsequent collection and analysis of data.
+Added: There is no guarantee that any CROs, investigators or the other third-party collaborators on which we rely for
+Added: administration and conduct of our clinical trials will devote adequate time and resources to such trials or perform as contractually required.
+Added: If any of these third parties fails to meet expected deadlines, fails to adhere to our clinical protocols, fails to meet regulatory requirements or otherwise performs in a substandard manner, our clinical trials may be extended, delayed or terminated.
+Added: If our clinical trial sites terminate for any reason, we may lose all of the information on subjects enrolled in any such clinical trials.
If we cannot contract with acceptable third parties on commercially reasonable terms, or if these third parties do not carry out their contractual duties, satisfy legal and regulatory requirements for the conduct of preclinical studies or clinical trials or meet expected deadlines, our clinical development programs could be delayed or discontinued.
9 unchanged sentences
Suppliers and manufacturers must meet applicable manufacturing requirements and undergo rigorous facility and process validation tests required by regulatory authorities in order to comply with regulatory standards, such as cGMP.
−Removed: In the event that any of our suppliers or manufacturers fails to comply with such requirements or to perform its obligations to us in relation to quality, timing or otherwise, or if our supply of components or other
−Removed: materials becomes limited or interrupted for other reasons, we may be forced to manufacture the materials ourselves or enter into an agreement with another third party, which would be costly and delay any future clinical trials.
+Added: In the event that any of our suppliers or manufacturers fails to comply with such requirements or to perform its obligations to us in relation to quality, timing or otherwise, or if our supply of components or other materials becomes limited or interrupted for other reasons, we may be forced to manufacture the materials ourselves or enter into an agreement with another third party, which would be costly and delay any future clinical trials.
In some cases, the technical skills or technology required to manufacture our product candidates may be unique or proprietary to the original manufacturer and we may have difficulty, or there may be contractual restrictions prohibiting us from, transferring such skills or technology to another third party.
These factors increase our reliance on our manufacturers and may require us to obtain a license from a manufacturer in order to have another third-party manufacture our product candidates.
−Removed: If we are required to change manufacturers for any reason, we will be required to verify that the new manufacturer maintains facilities and procedures that comply with quality standards and with all applicable regulations and guidelines of the FDA and comparable foreign regulatory authorities.
−Removed: The delays and costs associated with the verification of a new manufacturer could increase our costs and delay the development of our product candidates.
We expect to continue to rely on third-party manufacturers for preclinical and clinical grade product candidates and if we receive regulatory approval for any product candidate.
−Removed: To the extent that we have existing, or enter into future, manufacturing arrangements with third parties, we will depend on these third parties to perform their obligations in a timely manner consistent with contractual and regulatory requirements, including those related to quality control and assurance.
−Removed: If we are unable to obtain or maintain third-party manufacturing for product candidates, or to do so on commercially reasonable terms, we may not be able to develop and commercialize our product candidates successfully.
+Added: If we are unable to obtain or maintain third-party manufacturing for product candidates, or to do so on commercially reasonable terms, we may not be able to
+Added: develop and commercialize our product candidates successfully.
Our or a third party’s failure to execute on our manufacturing requirements could adversely affect our business in a number of ways, including:
an inability to conduct necessary preclinical studies to progress our product candidates to clinical trials;
−Removed: an inability to initiate or continue any future clinical trials of product candidates under development;
+Added: an inability to initiate or continue clinical trials of product candidates under development;
delay in submitting regulatory applications, or receiving regulatory approvals, for product candidates;
3 unchanged sentences
in the event of approval to market and commercialize a product candidate, an inability to meet commercial demands for our products.
−Removed: Our third-party manufacturers may be subject to damage or interruption from, among other things, events such as the COVID-19 pandemic, fire, natural or man-made disaster, power loss, telecommunications failure, unauthorized entry, computer viruses, denial-of-service attacks, acts of terrorism, human error, vandalism or sabotage, financial insolvency, bankruptcy and similar events.
+Added: Our third-party manufacturers may be subject to damage or interruption from, among other things, events such as pandemics, fire, natural or man-made disaster, power loss, telecommunications failure, unauthorized entry, computer viruses, denial-of-service attacks, acts of terrorism, human error, vandalism or sabotage, financial insolvency, bankruptcy and similar events.
We and our licensees or collaborators may disagree over our right to receive payments under any potential collaboration agreements with them, potentially resulting in costly litigation and loss of reputation.
3 unchanged sentences
If a dispute arises, it may result in costly patent office procedures and litigation, and our collaborator may refuse to pay us while the dispute is ongoing.
−Removed: Furthermore, regardless of any resort to legal action, a dispute with a collaborator over intellectual property rights may damage our relationship with that collaborator and may also harm our reputation
−Removed: in the industry.
+Added: Furthermore, regardless of any resort to legal action, a dispute with a collaborator over intellectual property rights may damage our relationship with that collaborator and may also harm our reputation in the industry.
Even if we are entitled to payments from our collaborators, we may not actually receive these payments, or we may experience difficulties in collecting the payments to which we believe we are entitled.
19 unchanged sentences
Risks Related to Commercialization of Our Product Candidates
−Removed: We have not entered into agreements with any third-party manufacturers to support commercialization of our product candidates.
−Removed: We have not yet secured manufacturing capabilities for commercial quantities of our product candidates or established facilities in the desired locations to support commercialization of our product candidates.
−Removed: We intend to rely on third-party manufacturers for commercialization, but have not entered into any agreements with such manufacturers to support our product candidates currently in development.
+Added: If we are unable to enter into agreements with third-party manufacturers on commercially reasonable terms, the commercialization of our product candidates may be adversely affected.
+Added: We intend to rely on third-party manufacturers for commercialization and production of commercial quantities of our product candidates.
We may be unable to negotiate agreements with third-party manufacturers to support our commercialization activities on commercially reasonable terms.
We may encounter technical or scientific issues related to manufacturing or development that we may be unable to resolve in a timely manner or with available funds.
−Removed: Currently, we do not have the capacity to manufacture our product candidates on a commercial scale.
−Removed: In addition, our product candidates are novel, and only one manufacturer currently has experience producing our product candidates on a large scale.
If we are unable to engage manufacturing partners to produce our product candidates on a larger scale on reasonable terms, our commercialization efforts will be harmed.
−Removed: Even if we timely develop a manufacturing process and successfully transfer it to the third-party manufacturers of our product candidates, if such third-party manufacturers are unable to produce the necessary quantities of our product candidates, or do so in compliance with cGMP or with pertinent foreign regulatory requirements, and within our planned time frame and cost parameters, the development and sales of our product candidates, if approved, may be impaired.
+Added: If such third-party manufacturers are unable to produce the necessary quantities of our product candidates, or do so in compliance with cGMP or with pertinent foreign regulatory requirements, and within our planned time frame and cost parameters, the development and sales of our product candidates, if approved, may be impaired.
In some jurisdictions, approval of the manufacturer may be required.
1 unchanged sentence
If we are unable to enter into agreements with third parties to commercialize our product candidates or establish sales and marketing capabilities to market and sell our product candidates, we may be unable to generate any revenues.
−Removed: We currently have no sales and marketing organization and have no experience selling and marketing pharmaceutical products.
−Removed: To successfully commercialize any product candidates that may be approved, we will need to develop these capabilities, either through our relationships with collaborators or our own.
+Added: To successfully commercialize any product candidates that may be approved, we will need to develop our sales and marketing capabilities, either through our relationships with collaborators or our own.
We may seek to enter into collaborations with other entities to utilize their marketing and distribution capabilities, but we may be unable to enter into marketing agreements on favorable terms, if at all.
−Removed: If our future collaborators do not commit sufficient resources to commercialize our future products, if any, and we are unable to develop the necessary marketing capabilities on our own, we will be unable to generate sufficient product revenue to sustain our business.
+Added: If our future collaborators do not commit
+Added: sufficient resources to commercialize our future products, if any, and we are unable to develop the necessary marketing capabilities on our own, we will be unable to generate sufficient product revenue to sustain our business.
The establishment and development of our own sales force or the establishment of a contract sales force to market any products we may develop 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 develop this capability.
We will be competing with many companies that currently have extensive and well- funded marketing and sales operations to recruit, hire, train and retain marketing and sales personnel.
3 unchanged sentences
Even if we obtain approval for a product candidate, we may not generate or sustain revenue from sales of the product if the product cannot be sold at a competitive cost or if it fails to achieve market acceptance by physicians, patients, third-party payers or others in the medical community.
−Removed: These market participants may be hesitant to adopt a novel treatment based on ddRNAi or silence and replace technology, and we may not be able
−Removed: to convince the medical community and third-party payers to accept and use, or to provide favorable reimbursement for, any product candidates developed by us or our existing or future collaborators.
+Added: These market participants may be hesitant to adopt a novel treatment based on ddRNAi or silence and replace technology, and we may not be able to convince the medical community and third-party payers to accept and use, or to provide favorable reimbursement for, any product candidates developed by us or our existing or future collaborators.
Market acceptance of our product candidates will depend on, among other factors:
13 unchanged sentences
Additional risks apply in relation to any disease indications we pursue which are classified as rare diseases and allow for orphan drug designation by regulatory agencies in major commercial markets, such as the United States or European Union.
−Removed: If pricing is not approved or accepted in the market at an appropriate level for any approved product for which we pursue and receive an orphan drug designation, such product may not generate enough revenue to offset costs of development, manufacturing, marketing and commercialization despite any benefits received from the orphan drug designation, such as market exclusivity, for a period of time.
+Added: If pricing is not approved or accepted in the market at an appropriate level for any approved product for which we pursue and receive an orphan drug designation, such product may not generate enough revenue to offset costs of development, manufacturing, marketing and commercialization despite any benefits
+Added: received from the orphan drug designation, such as market exclusivity, for a period of time.
Orphan exclusivity could temporarily delay or block approval of one of our products if a competitor obtains orphan drug designation for its product first.
5 unchanged sentences
We compete with a variety of multinational pharmaceutical companies and specialized biotechnology companies, as well as technology being developed at universities and other research institutions.
−Removed: Our competitors have developed, are
−Removed: developing or could develop product candidates and processes competitive with our product candidates.
+Added: Our competitors have developed, are developing or could develop product candidates and processes competitive with our product candidates.
Competitive therapeutic treatments include those that have already been approved and accepted by the medical community, patients and third-party payers, and any new treatments that enter the market.
1 unchanged sentence
This increasingly competitive landscape may compromise the development of our product candidates.
−Removed: We are aware of multiple companies that are working in the field of RNAi therapeutics, including Alnylam, Arbutus, and Arrowhead.
+Added: We are aware of multiple companies that are working in the field of RNAi therapeutics, including Alnylam.
Some of our current product candidates, if approved, would compete with approved and currently marketed treatments.
40 unchanged sentences
Accordingly, in markets outside the United States, the reimbursement for our products may be reduced compared with the United States and may be insufficient to generate commercially reasonable revenue and profits.
−Removed: In the United States, the Patient Protection and Affordable Care Act, as amended by the Health Care Education Reconciliation Act, each enacted in March 2010, as amended, or the ACA, greatly expanded health insurance coverage in the United States, but has also been the target of litigation and Congressional reform efforts since its adoption, including unsuccessful efforts to seek repeal of the entire ACA.
−Removed: While efforts to strike down the ACA
−Removed: in the courts currently seem stalled, uncertainty regarding the future of the ACA remains, including with respect to legislative efforts.
−Removed: Any significant efforts at the federal or state levels to reform the healthcare system by changing the way healthcare is provided or funded—including through the expansion of Medicare to larger sections of the population—could have a material impact on our business.
−Removed: In addition, the ACA, and similar laws, may cause such organizations to limit both coverage and level of reimbursement for new products approved and, as a result, they may not cover or provide adequate payment for our product candidates.
−Removed: For example, other legislative changes adopted since the ACA was enacted ACA may cause such organizations to limit both coverage and level of reimbursement for new products approved and, as a result, they may not cover or provide adequate payment for our product candidates.
−Removed: In addition, other legislative changes have been adopted since the ACA was enacted.
−Removed: These changes included aggregate reductions to Medicare payments to providers of up to 2% per fiscal year effective April 2013 that, due to subsequent legislative amendments to the statute, will stay in effect through 2029 unless additional Congressional action is taken.
−Removed: Note, however, the Coronavirus Aid, Relief, and Economic Security Act (CARES Act), which was signed into law on March 27, 2020, designed to provide financial support and resources to individuals and businesses affected by the COVID-19 pandemic, suspended the 2% Medicare sequester from May 1, 2020 through December 31, 2020, and extended the sequester by one year, through 2030.
−Removed: On December 14, 2018, a Texas U.S.
−Removed: District Court Judge ruled that the ACA is unconstitutional in its entirety because the “individual mandate” was repealed by Congress as part of the 2017 Cuts and Jobs Act (the “Tax Act”).
−Removed: Additionally, on December 18, 2019, the U.S.
−Removed: Court of Appeals for the 5th Circuit upheld the District Court ruling that the individual mandate was unconstitutional and remanded the case back to the District Court to determine whether the remaining provisions of the ACA are invalid as well.
−Removed: On March 2, 2020, the United States Supreme Court granted the petitions for writs of certiorari to review this case, although it is unclear when the Supreme Court will make a decision.
−Removed: It is also unclear how such litigation and other efforts to repeal and replace the ACA will impact the ACA and our business.
−Removed: In addition, other legislative changes have been proposed and adopted in the United States since the ACA was enacted.
−Removed: In January 2013, former President Obama signed into law the American Taxpayer Relief Act of 2012, which, among other things, reduced Medicare payments to several providers, and increased the statute of limitations period for the government to recover overpayments to providers from three to five years.
−Removed: This law may result in additional reductions in Medicare and other healthcare funding, which could have a material adverse effect on customers for our product candidates, if approved, and accordingly, our financial operations.
We expect to experience pricing pressures in connection with the sale of any of our product candidates due to the trend toward managed healthcare, value and risk-based payment arrangements that put healthcare providers at direct financial risk for the healthcare resources used to care for patient populations and additional legislative changes.
9 unchanged sentences
Treasury Department.
−Removed: Changes to tax laws (which changes may have retroactive application) could adversely affect us or holders of our
−Removed: common stock, warrants and pre-funded warrants.
+Added: Changes to tax laws (which changes may have retroactive application) could adversely affect us or holders of our common stock, warrants and pre-funded warrants.
In recent years, many such changes have been made and changes are likely to continue to occur in the future.
8 unchanged sentences
The applicable federal, state and foreign healthcare laws and regulations that may affect our ability to operate include, but are not limited to, the following:
−Removed: the federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under federal and state healthcare programs such as Medicare and Medicaid;
+Added: the federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly,
+Added: in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under federal and state healthcare programs such as Medicare and Medicaid;
federal civil and criminal false claims laws and civil monetary penalty laws, including the federal False Claims Act, which impose criminal and civil penalties, including civil whistleblower or qui tam actions, against individuals or entities for knowingly presenting, or causing to be presented, to the federal government, including the Medicare and Medicaid programs, claims for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government;
3 unchanged sentences
analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws, which may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payers, including private insurers;
−Removed: state and foreign laws
−Removed: that require biopharmaceutical or biotechnology companies to comply with the industry voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government or otherwise restrict payments that may be made to healthcare providers;
+Added: state and foreign laws that require biopharmaceutical or biotechnology companies to comply with the industry voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government or otherwise restrict payments that may be made to healthcare providers;
state and foreign laws that require biopharmaceutical or biotechnology manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures;
16 unchanged sentences
The competition for collaborators is significant, and the negotiation process is time- consuming and complex.
−Removed: Anew collaboration may be on terms that are not optimal for us and we may not be to
−Removed: maintain any new or existing collaboration if, for example, development or approval of a product candidate is delayed, sales of an approved product candidate do not meet expectations or the collaborator discontinues the collaboration.
+Added: Anew collaboration may be on terms that are not optimal for us and we may not be to maintain any new or existing collaboration if, for example, development or approval of a product candidate is delayed, sales of an approved product candidate do not meet expectations or the collaborator discontinues the collaboration.
Any such collaboration, or other strategic transaction, may require us to incur non- recurring or other charges, increase our expenditures, pose significant integration or implementation challenges or disrupt our management or business.
3 unchanged sentences
Any inability to attract and retain qualified key management and technical personnel would impair our ability to implement our business plan.
−Removed: Our success largely depends on the continued service of key management and other specialized personnel, including Dr.
−Removed: Jerel Banks (Chief Executive Officer) and Ms.
−Removed: Megan Boston (Executive Director).
+Added: Our success largely depends on the continued service of key management and other specialized personnel, including our chief executive officer, chief financial officer and chief operating officer.
The loss of one or more members of our management team or other key employees or advisors, if not adequately replaced, could delay or increase the cost of our research and development programs and materially harm our business, financial condition, results of operations and prospects.
11 unchanged sentences
In addition, we are limited in our ability to prevent them from establishing competing businesses or developing competing products.
−Removed: For example, if a key scientist acting as a principal investigator in any of our future clinical
−Removed: trials identifies a potential product or compound that is more scientifically interesting to his or her professional interests, his or her availability to remain involved in any future clinical trials could be restricted or eliminated.
+Added: For example, if a key scientist acting as a principal investigator in any of our future clinical trials identifies a potential product or compound that is more scientifically interesting to his or her professional interests, his or her availability to remain involved in clinical trials could be restricted or eliminated.
We may experience difficulties in managing our growth and expanding our operations.
We have limited experience in development and commercialization of pharmaceutical products.
−Removed: As our product candidates continue to advance through preclinical studies and any future clinical trials and potentially toward regulatory approval and commercial sale, we will need to expand our development, regulatory, manufacturing and sales capabilities or contract with other organizations to provide these capabilities for us.
+Added: As our product candidates continue to advance through preclinical studies and any clinical trials and potentially toward regulatory approval and commercial sale, we will need to expand our development, regulatory, manufacturing and sales capabilities or contract with other organizations to provide these capabilities for us.
In the future, we expect to have to manage additional relationships with collaborators or partners, suppliers and other organizations.
3 unchanged sentences
We are exposed to the risk of fraud or other misconduct by our employees, independent contractors, principal investigators, CROs, consultants, commercial partners and vendors.
−Removed: Misconduct by these parties could include intentional failures to comply with the regulations of the FDA and comparable foreign regulators, provide accurate information to the FDA and comparable foreign regulators, comply with healthcare fraud and abuse laws and regulations in the United States and abroad, report financial information or data accurately or disclose unauthorized activities to us.
+Added: Misconduct by these parties could include
+Added: intentional failures to comply with the regulations of the FDA and comparable foreign regulators, provide accurate information to the FDA and comparable foreign regulators, comply with healthcare fraud and abuse laws and regulations in the United States and abroad, report financial information or data accurately or disclose unauthorized activities to us.
In particular, sales, marketing and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices.
1 unchanged sentence
Such misconduct could also involve the improper use of information obtained in the course of any future clinical trials, which could result in regulatory sanctions and cause serious harm to our reputation.
−Removed: We have adopted a code of conduct applicable to all of our employees, but 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 governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations.
+Added: We have adopted a code of conduct, insider trading policy and other policies applicable to all of our employees, but 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 governmental 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, including the imposition of civil, criminal and administrative penalties, damages, monetary fines, possible exclusion from participation in Medicare, Medicaid and other federal healthcare programs, contractual damages, reputational harm, diminished profits and future earnings, and curtailment of our operations, any of which could adversely affect our ability to operate our business and our results of operations.
13 unchanged sentences
increased cost, or impairment of our ability, to obtain or maintain product liability insurance coverage.
−Removed: We carry combined general public and products liability (including human clinical trials extension) insurance of $20 million (per any one occurrence) and $10 million (per any one occurrence and in the aggregate), respectively.
−Removed: We believe our product liability insurance coverage is sufficient in light of our current clinical programs.
+Added: We carry customary levels of general public and products liability (including human clinical trials extension) insurance We believe our product liability insurance coverage is sufficient in light of our current clinical programs.
However, we may not be able to maintain insurance coverage at a reasonable cost or in sufficient amounts to protect us against losses due to liability.
1 unchanged sentence
On occasion, large judgments have been awarded against other pharmaceutical companies in class action lawsuits based on pharmaceutical products, or medical treatments that had unanticipated adverse effects.
−Removed: A successful product liability claim or series of claims brought against us could cause the price of our common stock to decline and, if judgments exceed our insurance coverage, could materially and adversely affect our financial position.
+Added: A successful product liability claim or series of claims brought against us could cause the price of our common stock to
+Added: decline and, if judgments exceed our insurance coverage, could materially and adversely affect our financial position.
During the course of treatment, patients may suffer adverse events, including death, for reasons that may be related to our product candidates.
17 unchanged sentences
Our internal computer and information technology systems, or those of our collaborators and other development partners, third-party CROs or other contractors or consultants, may fail or suffer security breaches, which could result in a disruption of our product development programs.
−Removed: Despite the implementation of security measures, our internal computer and information technology systems and those of our current and any future CROs and other contractors, consultants and collaborators are vulnerable to damage from computer viruses, cyber-attacks, unauthorized access, natural disasters, terrorism, war and telecommunication and electrical failures.
+Added: Despite the implementation of security measures, our internal computer and information technology systems and those of our current and any future CROs and other contractors, consultants and collaborators are vulnerable to damage from computer viruses, cyber-attacks, unauthorized access, natural disasters, terrorism, war and
+Added: telecommunication and electrical failures.
Such events could cause interruptions of our operations.
14 unchanged sentences
In addition, we do not maintain standalone cyber-security insurance and have limited insurance coverage in the event of any breach or disruption of our or our collaborators’, CROs’, or vendors’ systems, including any unauthorized access or loss of any personal data that we may collect, store or otherwise process.
−Removed: The costs related to significant security breaches or disruptions could be material and exceed
−Removed: the limits of any insurance coverage we may have.
+Added: The costs related to significant security breaches or disruptions could be material and exceed the limits of any insurance coverage we may have.
To the extent that any disruption or security breach were to result in a loss of, or damage to, our data or systems, or inappropriate disclosure of confidential or proprietary information, including data related to our personnel, we could incur liability and the further development and commercialization of our product candidates could be delayed and our business and operations could be adversely affected and/or could result in the loss or disclosure of critical or sensitive data, which could result in financial, legal, business or reputational harm to us.
6 unchanged sentences
If our facility is unable to operate because of an accident or incident or for any other reason, even for a short period of time, any or all of our research and development programs may be harmed.
−Removed: Any business interruption may have a material adverse effect on our business, financial position, results of operations and prospects.
+Added: business interruption may have a material adverse effect on our business, financial position, results of operations and prospects.
The investment of our cash and cash equivalents is subject to risks which may cause losses and affect the liquidity of these investments.
11 unchanged sentences
Therefore, our patents and patent applications may not be prosecuted and enforced in a manner consistent with the best interests of our business.
−Removed: It is possible that defects of form in the preparation or filing of our patents or patent applications may exist, or may arise in the future, for example
−Removed: with respect to proper priority claims, inventorship, claim scope, patent term adjustments, etc., although we are unaware of any such defects.
+Added: It is possible that defects of form in the preparation or filing of our patents or patent applications may exist, or may arise in the future, for example with respect to proper priority claims, inventorship, claim scope, patent term adjustments, etc., although we are unaware of any such defects.
If we or our current licensors or licensees, or any future licensors or licensees, fail to establish, maintain or protect such patents and other intellectual property rights, such rights may be reduced or eliminated.
7 unchanged sentences
If such prior art exists, it can invalidate a patent or prevent a patent from issuing from a pending patent application.
−Removed: Even if patents do successfully issue and even if such patents cover our product candidates, third parties may initiate opposition, interference, re-examination, post-grant review, inter parties review, nullification or derivation action in court or before patent offices or similar proceedings challenging the validity, enforceability or scope of such patents, which may result in the patent claims being narrowed or invalidated.
+Added: Even if patents do successfully issue and even if such patents cover our product candidates, third parties may initiate opposition, interference, re-examination, post-grant review, inter parties review, nullification or derivation action in court or before patent offices or similar proceedings challenging the validity, enforceability or scope of such patents, which may result in the patent claims being
+Added: narrowed or invalidated.
Furthermore, even if our patents and patent applications are unchallenged, they may not adequately protect our intellectual property, provide exclusivity for our product candidates or prevent others from designing around our claims.
12 unchanged sentences
This risk is material in light of the length of the development process of our products and lifespan of our current patent portfolio.
−Removed: In addition to the protection afforded by patents, we rely on trade secret protection and confidentiality agreements to protect proprietary know-how that is not patentable or that we elect not to patent, processes for which patents are difficult to enforce and any other elements of our product candidate discovery and
−Removed: development processes that involve proprietary know-how, information or technology that is not covered by patents.
+Added: In addition to the protection afforded by patents, we rely on trade secret protection and confidentiality agreements to protect proprietary know-how that is not patentable or that we elect not to patent, processes for which patents are difficult to enforce and any other elements of our product candidate discovery and development processes that involve proprietary know-how, information or technology that is not covered by patents.
However, trade secrets can be difficult to protect.
8 unchanged sentences
In addition, others may independently discover our trade secrets and proprietary information.
−Removed: For example, the FDA, as part of its Transparency Initiative, is currently considering whether to make additional information publicly available on a routine basis, including information that we may consider to be trade secrets or other proprietary information, and it is not clear at the present time how the FDA’s disclosure policies may change in the future, if at all.
+Added: For example, the FDA, as part of its Transparency Initiative, is currently considering whether to make additional information publicly available on a routine basis, including information that we may
+Added: consider to be trade secrets or other proprietary information, and it is not clear at the present time how the FDA’s disclosure policies may change in the future, if at all.
Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive and time-consuming, and the outcome is unpredictable.
13 unchanged sentences
We have not had and do not have primary control over these activities for certain of our patents or patent applications and other intellectual property rights we license, and therefore cannot guarantee that these patents and applications will be prosecuted or enforced in a manner consistent with the best interests of our business.
−Removed: We cannot be certain that such activities by third parties have
−Removed: been or will be conducted in compliance with applicable laws and regulations or will result in valid and enforceable patents and other intellectual property rights.
+Added: We cannot be certain that such activities by third parties have been or will be conducted in compliance with applicable laws and regulations or will result in valid and enforceable patents and other intellectual property rights.
Additionally, we may not be able to control the publication or other disclosures of research carried out by our licensors relating to technology that could otherwise prove patentable.
6 unchanged sentences
Under our existing license and sublicense agreements, we are subject to various obligations, including diligence obligations with respect to development and commercialization activities and payment obligations.
−Removed: In spite of our efforts, our licensors may conclude that we have materially breached our obligations under such license agreements and terminate the license agreements, thereby removing or limiting our ability to develop and commercialize product candidates and technology covered by these license agreements.
+Added: In spite of our
+Added: efforts, our licensors may conclude that we have materially breached our obligations under such license agreements and terminate the license agreements, thereby removing or limiting our ability to develop and commercialize product candidates and technology covered by these license agreements.
We may need to obtain additional licenses from third parties to advance our research or allow commercialization of our product candidates, such as if we identify new technology that would advance our programs or if an existing license agreement is terminated.
16 unchanged sentences
Our results of operations will be affected by the level of royalty payments that we are required to pay to third parties.
−Removed: We are a party to license agreements that require us to remit royalty payments and other payments related to in-licensed intellectual property.
−Removed: Under our in-license agreements, we may pay up-front fees and milestone payments and be subject to future royalties.
+Added: We have been and may in the future be party to license agreements that require us to remit royalty payments and other payments related to in-licensed intellectual property.
+Added: Under in-license agreements, we may be required to pay up-front fees and milestone payments and be subject to future royalties.
We cannot precisely predict the amount, if any, of royalties we will owe in the future, and if our calculations of royalty payments are incorrect, we may owe additional royalties, which could negatively affect our results of operations.
25 unchanged sentences
However, our product candidates may require specific formulations to work effectively and efficiently and rights to such formulations may be held by others.
−Removed: In addition, we may need additional intellectual property rights as we develop future product candidates.
+Added: In addition, we may need additional intellectual property rights as
+Added: we develop future product candidates.
In particular, we are aware of a third party patent directed to AAV vectors that expires in 2026.
15 unchanged sentences
To counter infringement or unauthorized use, we may be required to file infringement claims, which can be expensive and time-consuming.
−Removed: In addition, in an infringement proceeding, a court may decide that a patent of ours or our licensors is not valid, is unenforceable or is not infringed, or may refuse to stop the other party from using the technology at issue on the
−Removed: grounds that our patents do not cover the technology in question.
+Added: In addition, in an infringement proceeding, a court may decide that a patent of ours or our licensors is not valid, is unenforceable or is not infringed, or may refuse to stop the other party from using the technology at issue on the grounds that our patents do not cover the technology in question.
An adverse result in any litigation or defense proceedings could put one or more of our patents at risk of being invalidated or interpreted narrowly and could put our patent applications at risk of not issuing.
9 unchanged sentences
The outcome following legal assertions of invalidity and unenforceability is unpredictable.
−Removed: With respect to the validity question, for example, we cannot be certain that no invalidating prior art exists or that the patent examiner was aware of all material prior art during prosecution.
+Added: With respect to the validity question, for example, we cannot be certain that no
+Added: invalidating prior art exists or that the patent examiner was aware of all material prior art during prosecution.
Moreover, in some circumstances, we do not have the right to control the preparation, filing and prosecution of patent applications, or to maintain, enforce or defend the patents, covering technology that we license from third parties.
76 unchanged sentences
While our stockholder base and relative holdings may change over time, a number of institutional investors and similar stockholders currently hold significant ownership positions in our outstanding common stock and outstanding Warrants to acquire Common Stock.
+Added: On September 26, 2024, Suvretta Capital, on behalf of itself and each of the Suvretta Funds, entered into a waiver with the Company, pursuant to which, among other things (i) Suvretta Capital waived the 19.99% beneficial ownership limitation set forth in each of the warrants held by the Suvretta Funds, and (ii) Suvretta Capital and the Company agreed that Suvretta Capital will not be permitted to complete an exercise of the warrants held by the Suvretta Funds to the extent the beneficial ownership (calculated as provided in the applicable warrants) of Suvretta Capital in the Company following such exercise would exceed 49.9%.
In addition, in connection with the April 2024 private placement, we entered into the Board Designation Agreement with Suvretta Capital pursuant to which the Company appointed Kishen Mehta to the Board.
34 unchanged sentences
The presence of any material weaknesses in our internal control over financial reporting could result in financial statement errors which, in turn, could lead to errors in our financial reports and/or delays in our financial reporting, which could require us to restate our operating results.
−Removed: We might not identify one or more material
−Removed: weaknesses in our internal controls in connection with evaluating our compliance with Section 404(a) of the Sarbanes- Oxley Act.
+Added: We might not identify one or more material weaknesses in our internal controls in connection with evaluating our compliance with Section 404(a) of the Sarbanes- Oxley Act.
In order to maintain and improve the effectiveness of our disclosure controls and procedures and internal controls over financial reporting, we will need to expend significant resources and provide significant management oversight.
2 unchanged sentences
The report of our management for the fiscal year ended June 30, 2025 is included in Item 9A – Controls and Procedures of this Annual Report on Form 10-K.
−Removed: As further outlined in that report, our management has concluded that our internal control over financial reporting was not effective as of June 30, 2024, due to a material weakness in our internal controls resulting from our accounting personnel not being able to process and account for complex, non-routine transactions in accordance with GAAP.
−Removed: Such finding related to how the exercise price adjustment feature in the Company’s Series 2 warrant agreement was triggered by the Company’s August 11, 2023 public offering.
−Removed: Specifically, the exercise price of the Series 2 Warrants was reset as of that date from $11.22 to $1.9299.
−Removed: The adjustment to the exercise price of such warrants results, solely for accounting purposes, in a deemed dividend totaling $618,987.
−Removed: This amount was not recognized in the Company’s reported results for the quarter ended September 30, 2023, the three- and six-month periods ending December 31, 2023, and the three- and nine-month periods ending March 31, 2024.
−Removed: The impact of this adjustment to the exercise price will be accounted for as an increase in the Company’s accumulated deficit with an offsetting increase in Additional Paid-In Capital.
−Removed: In addition, the deemed dividend will be added to the Company’s net loss, increasing loss attributable to common stockholders for purposes of computing earnings per share (See Note 15 to the Notes to Consolidated Financial Statements titled “Updated quarterly results”).
−Removed: Management evaluated the adjustments and concluded that it was immaterial, both qualitatively and quantitatively.
−Removed: Management concluded that we lack sufficient personnel and outside consultants with technical accounting expertise to process and account for complex and non-routine transactions.
−Removed: A material weakness is a deficiency, or combination of deficiencies, in internal control over financial reporting, such that there is a reasonable possibility that a material misstatement of our annual or interim financial statements will not be prevented or detected on a timely basis.
−Removed: Notwithstanding the material weakness identified above, management has concluded that the particular transaction at issue was not material to the Company In order to remediate this matter, we plan to retain the assistance of additional accounting experts to assist in the accounting and reporting of complex, non-routine transactions.
−Removed: Such remedial measures will take time to implement and test and there can be no assurance that such measures will be sufficient to remedy the material weakness identified or that additional material weaknesses or other control or significant deficiencies will not be identified in the future.
+Added: As further outlined in that report, our management has concluded that our internal control over financial reporting was not effective as of June 30, 2025, due to a material weakness in our internal controls in connection with inadequate design and implementation of controls over our share-based compensation calculation review process.
+Added: This material weakness resulted in the restatement of our unaudited condensed consolidated financial statements as of and for the quarterly periods
+Added: ended March 31, 2025, and December 31, 2024, included in this Annual Report on Form 10-K.
+Added: Refer to Note 3, Restatement of Prior Period Financial Statements, in the consolidated financial statements in Part II, Item 8 for additional information.
+Added: Any failure to remediate the identified material weakness, or to develop or maintain effective controls, or any difficulties encountered in the implementation or improvement of such controls, could harm our operating results or cause us to fail to meet our reporting obligations and may result in a restatement of our financial statements for prior periods, such as the restatement of our previously issued unaudited condensed consolidated financial statements described in more detail in this Annual Report on Form 10-K.
+Added: Any failure to remediate the identified material weakness, or to implement and maintain effective internal control over financial reporting also could adversely affect the results of management evaluations and independent registered public accounting firm audits of our internal control over financial reporting that we are required to include in our periodic reports that will be filed with the SEC.
+Added: We can provide no assurance that the measures we are taking and plan to take in the future will remediate the material weakness identified in connection with the restatement, or that any additional material weaknesses or restatements of financial results will not arise in the future due to a failure to implement and maintain adequate internal control over financial reporting or circumvention of these controls.
+Added: In addition, even if we are successful in strengthening our controls and procedures, in the future those controls and procedures may not be adequate to prevent or identify irregularities or errors or to facilitate the fair presentation of our financial statements.
+Added: We continue to evaluate steps to remediate the material weakness.
+Added: Such remedial measures will take time to implement and test and there can be no assurance that such measures will be sufficient to remedy the material weakness around stock-based compensation identified or that additional material weaknesses or other control or significant deficiencies will not be identified in the future.
If we continue to experience material weaknesses in our internal controls or fail to maintain or implement required new or improved controls, investors may lose confidence in our operating results, the price of our common stock could decline and we may be subject to litigation or regulatory enforcement actions.
In addition, if we are unable to meet the requirements of Section 404 of the Sarbanes-Oxley Act, our common stock may not be able to remain listed on Nasdaq.
+Added: We have restated certain of our prior issued interim financial statements, which has resulted in unanticipated costs and may lead to additional risks and uncertainties, including loss of investor confidence and, as a result, the value of our common stock.
+Added: As discussed in Note 3 to our consolidated financial statements included elsewhere in this Annual Report on Form 10-K, we determined to restate our previously issued unaudited condensed consolidated financial statements as of and for the quarterly periods ended March 31, 2025, and December 31, 2024, after we identified the inaccurate calculation of share-based compensation expense due to a system configuration issue that was not detected by our share-based compensation review controls.
+Added: As a result of this error and the resulting restatement of our unaudited condensed consolidated financial statements for the impacted periods, we have incurred, and may continue to incur, unanticipated costs for accounting and legal fees in connection with or related to the restatement and have become subject to a number of additional risks and uncertainties, including the increased possibility of litigation and regulatory inquiries.
+Added: Any of the foregoing may affect investor confidence in the accuracy of our financial disclosures and may raise reputational risks for our business, both of which could harm our business and financial results.
We have never declared or paid dividends on our common stock and we do not anticipate paying dividends in the foreseeable future.
1 unchanged sentence
For the foreseeable future, we currently intend to retain all available funds and any future earnings to support our operations and to finance the growth and development of our business.
−Removed: Any future determination to declare cash dividends will be made at the discretion of our Board, subject to compliance with applicable laws and covenants under current or future credit facilities, which may restrict or limit our ability to pay dividends, and will depend on our financial condition, operating results, capital requirements, general business conditions and other factors that our Board may deem
+Added: Any future determination to declare cash dividends will be made at the discretion of our Board, subject to compliance with applicable laws and covenants under current or future credit facilities, which may restrict or limit our ability to pay dividends, and will depend on our financial condition,
+Added: operating results, capital requirements, general business conditions and other factors that our Board may deem relevant.
We do not anticipate paying any cash dividends on our common stock in the foreseeable future.
19 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.