What changed in this filing

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Item 1A. Risk Factors

2024-03-18 compared with 2023-03-29 · 451 added, 499 removed, 1110 unchanged (46% of the section changed)

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financial results, and future growth prospects.

Additional risks and uncertainties that are not currently known to us or that we do not

currently believe to be material may also negatively affect our business, financial condition, financial results, and future growth prospects.

FACTOR SUMMARY

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include, but are not limited to the following:

cell and gene therapy product candidates are based on proprietary methodologies, which makes it difficult to predict the time and

cost of product candidate development and regulatory approval.

Additionally, regulatory requirements governing cell and gene therapy

products have evolved and may continue to change in the future.

we do not obtain the necessary U.S.

or worldwide regulatory approvals to commercialize EB-101, we will not be able to sell EB-101.

if we receive regulatory approval for EB-101, our lead drug candidate, we may not be able to successfully commercialize the product

and the revenue that we generate from its sales, if any, may be limited.

may encounter substantial delays in our clinical studies or we may fail to demonstrate safety and efficacy to the satisfaction of

applicable regulatory authorities.

Additionally, we may find it difficult to enroll patients in our clinical studies, which could

delay or prevent clinical studies of our product candidates.

have received and may apply for additional designations such as breakthrough therapy designation, RMAT designation, fast track designation,

and rare pediatric disease designation from the FDA intended to facilitate or encourage product candidate development.

receive any such designations or be able to maintain them.

Moreover, any such designations may not lead to faster development or

regulatory review or approval and it does not increase the likelihood that our product candidates will receive marketing approval.

certain of our product candidates have received orphan drug designation from the FDA, there is no guarantee that we will be able

to maintain this designation, receive this designation for any of our other product candidates, or receive or maintain any corresponding

benefits, including periods of exclusivity.

if we obtain regulatory approval for a product candidate, our products will remain subject to regulatory scrutiny.

COVID-19 pandemic and efforts to reduce its spread have affected our operations and significantly impacted worldwide economic conditions,

and could continue to have a material effect on our operations, business, and financial condition.

could experience production problems in our manufacturing facilities that result in delays in our development or commercialization

We might also experience delays in manufacturing if any of our vendors, contract laboratories or suppliers are found to

be out of compliance with current Good Manufacturing Practice.

we fail to comply with applicable regulations, the relevant regulatory authority may require remedial measures that may be costly

or time-consuming to implement and that may include the suspension of a clinical trial or commercial sales or the closure of a manufacturing

widespread outbreak of an illness, communicable disease, or any other public health crisis could adversely affect our business, results

of operations and financial condition.

expect to rely on third parties, and these third parties may not perform satisfactorily.

Additionally, our reliance on third parties

requires us to share our trade secrets, which increases the possibility that a competitor will discover them or that our trade secrets

will be misappropriated.

drug candidates are subject to the risks of failure inherent in the development of pharmaceutical products based on new technologies,

and our failure to develop safe and commercially viable drugs would severely limit our ability to become profitable or to achieve

significant revenues.

may be unable to successfully develop, market, or commercialize our products or our product candidates without establishing new relationships

and maintaining current relationships and our ability to successfully commercialize, and market our product candidates could be limited

if a number of these existing relationships are terminated.

may incur substantial product liability expenses due to the use or misuse of our products for which we may be unable to obtain insurance

ability to successfully develop and commercialize our drug candidates will substantially depend upon the availability of reimbursement

funds for the costs of the resulting drugs and related treatments.

market may not accept any pharmaceutical products that we develop, and adverse public perception of gene therapy products may negatively

affect demand for, or regulatory approval of, our product candidates.

may be subject to federal, state, and foreign healthcare laws and regulations, including fraud and abuse laws, false claims laws,

health information privacy and security laws and data privacy laws.

If we are unable to comply, or have not fully complied, with

such laws, we could face substantial penalties.

business could suffer if we lose the services of, or fail to attract, key personnel.

toward managed health care and downward price pressures on medical products and services may limit our ability to profitably sell

any drugs that we may develop.

rights to develop and commercialize our product candidates are subject to, in part, the terms and conditions of licenses granted

to us by others.

we are unable to obtain and maintain patent protection for our product candidates and technology, or if the scope of the patent protection

obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical

intellectual property licenses with third parties may be subject to disagreements over contract interpretation.

may not be successful in obtaining necessary rights to our product candidates through acquisitions and in-licenses.

may not be able to protect our intellectual property rights around the world.

patents covering our product candidates could be found invalid or unenforceable if challenged in court.

We may not be able to protect

our trade secrets in court, and intellectual property litigation could cause us to spend substantial resources.

Third-parties

may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be

uncertain and could harm our business.

may be subject to claims asserting that our employees, consultants or advisors have wrongfully used or disclosed alleged trade secrets

of their current or former employers or claims asserting ownership of what we regard as our own intellectual property.

we do not obtain patent term extension and data exclusivity for our product candidates, our business may be harmed.

have experienced a history of losses;

we expect to incur future losses and we may be unable to obtain necessary additional capital

to fund operations in the future.

Our cell and gene therapy

product candidates are based on proprietary methodologies, which makes it difficult to predict the time and cost of product candidate

development and regulatory approval.

Additionally, regulatory requirements governing cell and gene therapy products have evolved

and may continue to change in the future.

If we do not obtain the

necessary U.S.

or worldwide regulatory approvals to commercialize pz-cel, we will not be able to sell pz-cel.

if we receive regulatory approval for pz-cel, our lead drug candidate, we may not be able to successfully manufacture or

commercialize the product and the revenue that we generate from its sales, if any, may be limited.

We may encounter substantial

delays in our clinical studies, or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities.

Additionally, we may find it difficult to enroll patients in our clinical studies, which could delay or prevent clinical studies

of our product candidates.

We have received and may

apply for additional designations such as breakthrough therapy designation, RMAT designation, fast track designation, and rare pediatric

disease designation from the FDA intended to facilitate or encourage product candidate development.

We may not receive any such designations

or be able to maintain them.

Moreover, any such designations may not lead to faster development or regulatory review or approval

and it does not increase the likelihood that our product candidates will receive marketing approval.

While certain of our product

candidates have received orphan drug designation from the FDA, there is no guarantee that we will be able to maintain this designation,

receive this designation for any of our other product candidates, or receive or maintain any corresponding benefits, including periods

of exclusivity.

Even if we obtain regulatory

approval for a product candidate, our products will remain subject to regulatory scrutiny.

We could experience production

problems in our manufacturing facility that result in delays in our development or commercialization programs.

We might also experience

delays in manufacturing if any of our vendors, contract laboratories or suppliers are found to be out of compliance with current

Good Manufacturing Practice.

If we fail to comply with

applicable regulations, the relevant regulatory authority may require remedial measures that may be costly or time-consuming to implement

and that may include the suspension of a clinical trial or commercial sales or the closure of a manufacturing facility.

The widespread outbreak

of an illness, communicable disease, or any other public health crisis could adversely affect our business, results of operations

and financial condition.

We expect to rely on third

parties, and these third parties may not perform satisfactorily.

Additionally, our reliance on third parties requires us to share

our trade secrets, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated.

Our drug candidates are

subject to the risks of failure inherent in the development of pharmaceutical products based on new technologies, and our failure

to develop safe and commercially viable drugs would severely limit our ability to become profitable or to achieve significant revenues.

We may be unable to successfully

develop, market, or commercialize our products or our product candidates without establishing new relationships and maintaining current

relationships and our ability to successfully commercialize, and market our product candidates could be limited if a number of these

existing relationships are terminated.

We may incur substantial

product liability expenses due to the use or misuse of our products for which we may be unable to obtain insurance coverage.

Our ability to successfully

develop and commercialize our drug candidates will substantially depend upon the availability of reimbursement funds for the costs

of the resulting drugs and related treatments.

The market may not accept

any pharmaceutical products that we develop, and adverse public perception of gene therapy products may negatively affect demand

for, or regulatory approval of, our product candidates.

We may be subject to federal,

state, and foreign healthcare laws and regulations, including fraud and abuse laws, false claims laws, health information privacy

and security laws and data privacy laws.

If we are unable to comply, or have not fully complied, with such laws, we could face substantial

Trends toward managed health

care and downward price pressures on medical products and services may limit our ability to profitably sell any drugs that we may

Our rights to develop and

commercialize our product candidates are subject to, in part, the terms and conditions of licenses granted to us by others.

If we are unable to obtain

and maintain patent protection for our product candidates and technology, or if the scope of the patent protection obtained is not

sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours.

Our intellectual property

licenses with third parties may be subject to disagreements over contract interpretation.

We may not be successful

in obtaining necessary rights to our product candidates through acquisitions and in-licenses.

Issued patents covering

our product candidates could be found invalid or unenforceable if challenged in court.

We may not be able to protect our trade secrets

in court, and intellectual property litigation could cause us to spend substantial resources.

Third-parties may initiate

legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and

could harm our business.

We may be subject to claims

asserting that our employees, consultants or advisors have wrongfully used or disclosed alleged trade secrets of their current or

former employers or claims asserting ownership of what we regard as our own intellectual property.

If we do not obtain patent

term extension and data exclusivity for our product candidates, our business may be harmed.

We have experienced a history

we expect to incur future losses and we may be unable to obtain necessary additional capital to fund operations in the

We do not have significant operating revenue and may never achieve profitability.

to achieve and maintain effective internal controls could have a material adverse effect on our business.

expect to continue to need to raise additional capital to operate our business, and our failure to obtain funding when needed or

on terms that are favorable to us may force us to delay, reduce or eliminate our development programs or aspects thereof.

market price of our common stock may be volatile and adversely affected by several factors.

additional funds by issuing securities or through licensing or lending arrangements or through our at-the-market sale agreement may

cause dilution to our existing stockholders, restrict our operations or require us to relinquish proprietary rights.

quarterly operating results may fluctuate significantly.

of our charter documents could discourage an acquisition of our company.

We expect to continue to

need to raise additional capital to operate our business, and our failure to obtain funding when needed or on terms that are favorable

to us may force us to delay, reduce or eliminate our development programs or aspects thereof.

Failure to achieve and

maintain effective internal controls could have a material adverse effect on our business.

The market price of our

common stock may be volatile and adversely affected by several factors.

Raising additional funds

by issuing securities or through licensing or lending arrangements or through our at-the-market sale agreement may cause dilution

to our existing stockholders, restrict our operations or require us to relinquish proprietary rights.

Breaches of data security

or unauthorized disclosures of personal information could effect our business or make us subject to liability.

related to the discovery and development of our product candidates

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Preclinical and early clinical studies may also reveal unfavorable product candidate characteristics, including safety concerns.

also experience numerous unforeseen events during, or as a result of, clinical trials that could delay or prevent our ability to receive

marketing approval or commercialize our product candidates, including:

or IRBs may not authorize us or our investigators to commence or continue a clinical trial, conduct a clinical trial at a prospective

trial site, or amend trial protocols, or regulators or IRBs may require that we modify or amend our clinical trial protocols;

may experience delays in reaching, or fail to reach, agreement on acceptable clinical trial contracts or clinical trial protocols

with prospective trial sites and our CROs;

may require us to perform additional or unanticipated clinical trials to obtain approval or we may be subject to additional post-marketing

testing, surveillance, or REMS requirements to maintain regulatory approval;

in a clinical trial may not become apparent until the trial is well advanced;

trials of our product candidates may produce negative or inconclusive results, or our studies may fail to reach the necessary level

of statistical significance, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon product

development programs;

trials of our product candidates may require us to provide follow-up patient visits for safety for a minimum of five years even if

we were to terminate and/or abandon a product development program;

third-party contractors may fail to comply with regulatory requirements or the clinical trial protocol, or fail to meet their contractual

obligations to us in a timely manner, or at all, or we may be required to engage in additional clinical trial site monitoring;

the regulators, or IRBs may require the suspension or termination of clinical research for various reasons, including noncompliance

with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks, undesirable side

effects, or other unexpected characteristics (alone or in combination with other products) of the product candidate, or due to findings

of undesirable effects caused by a chemically or mechanistically similar therapeutic or therapeutic candidate;

in marketing approval and regulatory review policies or changes in or the enactment of additional statutes or regulations;

cost of clinical trials of and marketing applications for our product candidates may be greater than we anticipate;

supply or quality of our product candidates or other materials necessary to conduct clinical trials may be insufficient or inadequate;

may decide, or regulators may require us, to conduct or gather, as applicable, additional clinical trials, analyses, reports, data,

or preclinical trials, or we may abandon product development programs;

may fail to reach an agreement with regulators or IRBs regarding the scope, design, or implementation of our clinical trials.

instance, the FDA or comparable foreign regulatory authorities may require changes to our study design that make further study impractical

or not financially prudent;

may have delays in adding new investigators or clinical trial sites, or we may experience a withdrawal of clinical trial sites;

may be regulatory questions or disagreements regarding interpretations of data and results, or new information may emerge regarding

our product candidates;

may make changes to our product candidates or their manufacturing process that necessitate additional studies or that result in our

product candidates not performing as expected;

FDA or comparable foreign regulatory authorities may disagree with our study design, including endpoints, or our interpretation of

data from preclinical studies and clinical trials or find that a product candidate’s benefits do not outweigh its safety risks;

FDA or comparable foreign regulatory authorities may not accept data from studies with clinical trial sites in foreign countries;

FDA or comparable regulatory authorities may disagree with our intended indications;

FDA or comparable foreign regulatory authorities may fail to approve or subsequently find fault with the manufacturing processes

or our contract manufacturer’s manufacturing facility for clinical and future commercial supplies;

data collected from clinical trials of our product candidates may not be sufficient to the satisfaction of the FDA or comparable

foreign regulatory authorities to support the submission of a marketing application, or other comparable submission in foreign jurisdictions

or to obtain regulatory approval in the United States or elsewhere;

one of our product candidates does not receive marketing approval in one country, it may impact our ability to receive marketing

approval in other countries;

FDA or comparable regulatory authorities may take longer than we anticipate to make a decision on our product candidates;

may not be able to demonstrate that a product candidate provides an advantage over current standards of care or current or future

competitive therapies in development.

may also experience numerous unforeseen events during, or as a result of, clinical trials that could delay or prevent our ability to

receive marketing approval or commercialize our product candidates, including:

regulators or IRBs may

not authorize us or our investigators to commence or continue a clinical trial, conduct a clinical trial at a prospective trial site,

or amend trial protocols, or regulators or IRBs may require that we modify or amend our clinical trial protocols;

we may experience delays

in reaching, or fail to reach, agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial

sites and our contract research organizations (“CROs’);

regulators may require

us to perform additional or unanticipated clinical trials to obtain approval or we may be subject to additional post-marketing testing,

surveillance, or REMS requirements to maintain regulatory approval;

flaws in a clinical trial

may not become apparent until the trial is well advanced;

clinical trials of our

product candidates may produce negative or inconclusive results, or our studies may fail to reach the necessary level of statistical

significance, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon product development

clinical trials of our

product candidates may require us to provide follow-up patient visits for safety for a minimum of five years even if we were to terminate

and/or abandon a product development program;

our third-party contractors

may fail to comply with regulatory requirements or the clinical trial protocol, or fail to meet their contractual obligations to

us in a timely manner, or at all, or we may be required to engage in additional clinical trial site monitoring;

we, the regulators, or

IRBs may require the suspension or termination of clinical research for various reasons, including noncompliance with regulatory

requirements or a finding that the participants are being exposed to unacceptable health risks, undesirable side effects, or other

unexpected characteristics (alone or in combination with other products) of the product candidate, or due to findings of undesirable

effects caused by a chemically or mechanistically similar therapeutic or therapeutic candidate;

changes in marketing approval

and regulatory review policies or changes in or the enactment of additional statutes or regulations;

the cost of clinical trials

of and marketing applications for our product candidates may be greater than we anticipate;

the supply or quality of

our product candidates or other materials necessary to conduct clinical trials may be insufficient or inadequate;

we may decide, or regulators

may require us, to conduct or gather, as applicable, additional clinical trials, analyses, reports, data, or preclinical trials,

or we may abandon product development programs;

we may fail to reach an

agreement with regulators or IRBs regarding the scope, design, or implementation of our clinical trials.

For instance, the FDA or

comparable foreign regulatory authorities may require changes to our study design that make further study impractical or not financially

we may have delays in adding

new investigators or clinical trial sites, or we may experience a withdrawal of clinical trial sites;

there may be regulatory

questions or disagreements regarding interpretations of data and results, or new information may emerge regarding our product candidates;

we may make changes to

our product candidates or their manufacturing process that necessitate additional studies or that result in our product candidates

not performing as expected;

the FDA or comparable foreign

regulatory authorities may disagree with our study design, including endpoints, or our interpretation of data from preclinical studies

and clinical trials or find that a product candidate’s benefits do not outweigh its safety risks;

the FDA or comparable foreign

regulatory authorities may not accept data from studies with clinical trial sites in foreign countries;

the FDA or comparable regulatory

authorities may disagree with our intended indications;

the FDA or comparable foreign

regulatory authorities may fail to approve or subsequently find fault with the manufacturing processes or our contract manufacturer’s

manufacturing facility for clinical and future commercial supplies;

the data collected from

clinical trials of our product candidates may not be sufficient to the satisfaction of the FDA or comparable foreign regulatory authorities

to support the submission of a marketing application, or other comparable submission in foreign jurisdictions or to obtain regulatory

approval in the United States or elsewhere;

if one of our product candidates

does not receive marketing approval in one country, it may impact our ability to receive marketing approval in other countries;

the FDA or comparable regulatory

authorities may take longer than we anticipate to make a decision on our product candidates;

we may not be able to demonstrate

that a product candidate provides an advantage over current standards of care or current or future competitive therapies in development.

in launching clinical trials resulting from FDA or other regulatory actions, such as a clinical hold letter, would delay the commercialization

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For example, in September

2019, we received a clinical hold letter in connection with our Phase 3 clinical trial for EB-101 stating that the FDA would not provide

approval for us to begin our planned Phase 3 clinical trial for EB-101 until we submitted additional data points on transport stability

of EB-101 to clinical sites.

2019, we received a clinical hold letter in connection with our phase 3 clinical trial for pz-cel stating that the FDA would not provide

approval for us to begin our planned phase 3 clinical trial for pz-cel until we submitted additional data points on transport stability

of pz-cel to clinical sites.

Although the FDA removed the clinical hold in December 2019 and provided clearance for us to proceed with

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Patient enrollment is affected by factors including:

of the disease under investigation;

of the study protocol;

and nature of the patient population;

criteria for and design of the study in question;

risks and benefits of the product candidate under study, including as a result of adverse effects observed in similar or competing

and availability of clinical study sites for prospective patients;

of competing therapies and clinical studies;

to facilitate timely enrollment in clinical studies;

to compensate patients for their time and effort;

that enrolled patients will drop out before completion or not return for post-treatment follow-up;

to obtain or maintain patient informed consents;

effectiveness

of publicity created by clinical trial sites regarding the trial;

referral practices of physicians;

to monitor patients adequately during and after treatment.

severity of the disease

under investigation;

design of the study protocol;

size and nature of the

patient population;

eligibility criteria for

and design of the study in question;

perceived risks and benefits

of the product candidate under study, including as a result of adverse effects observed in similar or competing therapies;

proximity and availability

of clinical study sites for prospective patients;

availability of competing

therapies and clinical studies;

efforts to facilitate timely

enrollment in clinical studies;

ability to compensate patients

for their time and effort;

risk that enrolled patients

will drop out before completion or not return for post-treatment follow-up;

inability to obtain or

maintain patient informed consents;

effectiveness of publicity

created by clinical trial sites regarding the trial;

patient referral practices

of physicians;

ability to monitor patients

adequately during and after treatment.

also plan to seek initial marketing approval in the European Union in addition to the U.S.

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clinical superiority would need to be shown, is evolving.

While the agency has issued a guidance on the topic, certain decisions may

need to be made on a case by case basis, given the novelty of the technology.

Moreover, third-party payors may reimburse for products

off-label even if not indicated for the orphan condition.

While the agency has issued guidance on the topic, certain decisions may need

to be made on a case by case basis, given the novelty of the technology.

Moreover, third-party payors may reimburse for products off-label

even if not indicated for the orphan condition.

if we obtain regulatory approval for a product candidate, our products will remain subject to regulatory scrutiny.

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or if we later discovery previously unknown safety, efficacy, or manufacturing issues, the following may result:

on manufacturing, distribution, marketing, or labeling of such products, including restrictions on the indication or approved patient

population, and required additional warnings, such as black box warnings, contraindications, and precautions;

to conduct post-marketing studies or clinical trials, or to institute risk mitigation strategies, such as REMS;

of corrective information;

product may become less competitive, we may face reputational harm, or we may face liability for any harm caused to patients or subjects;

modifications

on the way the product is administered;

modifications

on promotional pieces;

of warning, untitled, or cyber letters asserting that we are in violation of the law, or of safety alerts, Dear Healthcare Provider

letters, press releases, or other communications containing warnings or other safety information about the product;

or imposition civil or criminal penalties or monetary fines, restitution, or disgorgement of profits or revenues;

or withdrawal of regulatory approval;

or termination of any ongoing clinical studies;

to approve a pending marketing application, such as a BLA or supplements to a BLA submitted by us;

detention, or recall of product;

to permit the import or export of our products;

to allow us to enter into supply contracts, including government contracts, exclusion from federal healthcare programs, FDA debarment,

consent decrees, or corporate integrity agreements.

restrictions on manufacturing,

distribution, marketing, or labeling of such products, including restrictions on the indication or approved patient population, and

required additional warnings, such as black box warnings, contraindications, and precautions;

requirements to conduct

post-marketing studies or clinical trials, or to institute risk mitigation strategies, such as REMS;

issuance of corrective

the product may become

less competitive, we may face reputational harm, or we may face liability for any harm caused to patients or subjects;

modifications on the way

the product is administered;

modifications on promotional

issuance of warning, untitled,

or cyber letters asserting that we are in violation of the law, or of safety alerts, Dear Healthcare Provider letters, press releases,

or other communications containing warnings or other safety information about the product;

injunction or imposition

civil or criminal penalties or monetary fines, restitution, or disgorgement of profits or revenues;

suspension or withdrawal

of regulatory approval;

suspension or termination

of any ongoing clinical studies;

refusal to approve a pending

marketing application, such as a BLA or supplements to a BLA submitted by us;

seizure, detention, or

recall of product;

refusal to permit the import

or export of our products;

refusal to allow us to

enter into supply contracts, including government contracts, exclusion from federal healthcare programs, FDA debarment, consent decrees,

or corporate integrity agreements.

government investigation of alleged violations of law could require us to expend significant time and resources in response and could

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that a given lot will perform in the intended manner.

are several risks specific to the manufacturing process for EB-101 which require close attention.

are several risks specific to the manufacturing process for pz-cel which require close attention.

As an autologous product there are

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epidermal sheets must be discarded and the manufacturing process must be repeated.

currently do not have a backup manufacturer to supply clinical trial material for EB-101.

currently do not have a backup manufacturer to supply clinical trial material for pz-cel.

An alternative manufacturer would need to be

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If we or our contract manufacturers cannot successfully manufacture material that conforms to our specifications

and the strict regulatory requirements of the FDA or other regulatory authorities, they will not be able to secure or maintain regulatory

approval for their manufacturing facilities.

Any such deviations may also require remedial measures that may be costly and/or time-consuming

for us or a third party to implement and may include the temporary or permanent suspension of a clinical trial or commercial sales or

the temporary or permanent closure of a facility.

Any such remedial measures imposed upon or by us or third parties with whom we contract

could materially harm our business.

Any delays in obtaining products or product candidates that comply with the applicable regulatory

requirements may result in delays to clinical trials, product approvals, and commercialization.

It may also require that we conduct additional

and the strict regulatory requirements of the FDA or other regulatory authorities, we or our contract manufacturers will not be able

to secure or maintain regulatory approval for their manufacturing facilities.

Any such deviations may also require remedial measures

that may be costly and/or time-consuming for us or a third party to implement and may include the temporary or permanent suspension of

a clinical trial or commercial sales or the temporary or permanent closure of a facility.

Any such remedial measures imposed upon or

by us or third parties with whom we contract could materially harm our business.

Any delays in obtaining products or product candidates

that comply with the applicable regulatory requirements may result in delays to clinical trials, product approvals, and commercialization.

It may also require that we conduct additional studies.

have manufacturing agreements with third parties that provide for, among other things, production of product candidates for our current

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reliance on these third parties entails risks to which we would not be subject if we manufactured the product candidates ourselves, including:

control for certain aspects of manufacturing activities;

control over the protection of our trade secrets and know-how from misappropriation or inadvertent disclosure;

to negotiate manufacturing agreements with third parties under commercially reasonable terms;

on the third party for regulatory compliance and quality assurance;

or nonrenewal of manufacturing and service agreements with third parties in a manner or at a time that is costly or damaging to us;

to the operations of our third-party manufacturers and service providers caused by conditions unrelated to our business or operations,

including the bankruptcy of the manufacturer or service provider.

reduced control for certain

aspects of manufacturing activities;

reduced control over the

protection of our trade secrets and know-how from misappropriation or inadvertent disclosure;

inability to negotiate

manufacturing agreements with third parties under commercially reasonable terms;

reliance on the third party

for regulatory compliance and quality assurance;

termination or nonrenewal

of manufacturing and service agreements with third parties in a manner or at a time that is costly or damaging to us;

disruptions to the operations

of our third-party manufacturers and service providers caused by conditions unrelated to our business or operations, including the

bankruptcy of the manufacturer or service provider.

of these events could lead to clinical trial delays or failure to obtain regulatory approval or impact our ability to successfully commercialize

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we do not obtain the necessary U.S.

or worldwide regulatory approvals to commercialize EB-101, we will not be able to sell EB-101.

we cannot obtain regulatory approval for EB-101, we will not be able to generate revenue from this product candidate.

or worldwide regulatory approvals to commercialize pz-cel, we will not be able to sell pz-cel.

we cannot obtain regulatory approval for pz-cel, we will not be able to generate revenue from this product candidate.

As a result, our

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We cannot assure you that we will receive the approvals

necessary to commercialize EB-101 or any other product candidate we may develop in the future.

In order to obtain FDA approval of EB-101

necessary to commercialize pz-cel or any other product candidate we may develop in the future.

In order to obtain FDA approval of pz-cel

or any other product candidate requiring FDA approval, we must successfully complete an FDA BLA review.

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future legislation or administrative action or changes in FDA policy that occur prior to or during our regulatory review.

Delays in obtaining

regulatory approvals may:

commercialization of, and our ability to derive product revenues from, EB-101 or any other product candidate;

costly procedures on us;

any competitive advantages that we may otherwise enjoy.

in obtaining regulatory approvals may:

delay commercialization

of, and our ability to derive product revenues from, pz-cel or any other product candidate;

impose costly procedures

diminish any competitive

advantages that we may otherwise enjoy.

if we comply with all FDA requests, the FDA may ultimately reject our BLA.

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subjects or require that we conduct further studies with more subjects.

We may never obtain regulatory approval for EB-101, or any other

We may never obtain regulatory approval for pz-cel, or any other

future potential product candidate.

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the approvals necessary to commercialize any product candidate for sale outside the U.S.

if we receive regulatory approval for EB-101, our lead drug candidate, we may not be able to successfully commercialize the product and

if we receive regulatory approval for pz-cel, our lead drug candidate, we may not be able to successfully commercialize the product and

the revenue that we generate from its sales, if any, may be limited.

approved for marketing, the commercial success of EB-101 will depend upon the product’s acceptance by the medical community, including

approved for marketing, the commercial success of pz-cel will depend upon the product’s acceptance by the medical community, including

physicians, patients and healthcare payors.

The degree of market acceptance for our drug candidate will depend on a number of factors,

and perceived efficacy and safety of EB-101;

convenience, dosing burden and ease of administration;

or perceived advantages or disadvantages over alternative treatments;

post-marketing commitments imposed by regulatory authorities, such as patient registries;

of sales, marketing and distribution support;

of our future products, both in absolute terms and relative to alternative treatments;

effect of current and future healthcare laws on EB-101;

of coverage and reimbursement from government and other third party payers.

actual and perceived efficacy

and safety of pz-cel;

relative convenience, dosing

burden and ease of administration;

potential or perceived

advantages or disadvantages over alternative treatments;

potential post-marketing

commitments imposed by regulatory authorities, such as patient registries;

strength of sales, marketing

and distribution support;

price of our future products,

both in absolute terms and relative to alternative treatments;

the effect of current and

future healthcare laws on pz-cel;

availability of coverage

and reimbursement from government and other third party payers.

our drug candidate is approved but does not achieve an adequate level of acceptance by physicians, healthcare payors and patients, we

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reduce the demand for, or the price of, our products.

We have not commenced efforts to have our product candidates reimbursed by government

or third-party payors.

If coverage and reimbursement are not available or are available only at limited levels, we may not be able to

commercialize our products.

In recent years, officials have made numerous proposals to change the health care system in the U.S.

proposals include measures that would limit or prohibit payments for certain medical treatments or subject the pricing of drugs to government

In addition, in many foreign countries, particularly the countries of the European Union, the pricing of prescription drugs

is subject to government control.

If our products are or become subject to government regulation that limits or prohibits payment for

our products, or that subjects the price of our products to governmental control, we may not be able to generate revenue, attain profitability

or commercialize our products.

We have not commenced efforts to have our product candidates reimbursed by the

government or third-party payors.

If coverage and reimbursement are not available or are available only at limited levels, we may not

be able to commercialize our products.

In recent years, officials have made numerous proposals to change the health care system in the

These proposals include measures that would limit or prohibit payments for certain medical treatments or subject the pricing of

drugs to government control.

In addition, in many foreign countries, particularly the countries of the European Union, the pricing of

prescription drugs is subject to government control.

If our products are or become subject to government regulation that limits or prohibits

payment for our products, or that subjects the price of our products to governmental control, we may not be able to generate revenue,

attain profitability or commercialize our products.

a result of legislative proposals and the trend towards managed health care in the U.S., third-party payors are increasingly attempting

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or require payment of increased rebates and our ability to generate revenues and achieve and maintain profitability.

laws, regulations and judicial decisions, or new interpretations of existing laws, regulations, and decisions, that relate to healthcare

laws, regulations and judicial decisions, or new interpretations of existing laws, regulations, and decisions, which relate to healthcare

availability, methods of delivery or payment for products and services, or sales, marketing, or pricing, may limit our potential revenue,

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and directors, private litigation and/or adverse publicity that negatively affects our business.

breaches and other disruptions could compromise our information and expose us to liability, which would cause our business and reputation

the ordinary course of our business, we collect and store sensitive data, including intellectual property, our proprietary business information

and that of our suppliers and business partners, as well as personally identifiable information of clinical trial participants and employees.

Similarly, our business partners and third-party providers possess certain of our sensitive data.

The secure maintenance of this information

is critical to our operations and business strategy.

Despite our security measures, our information technology and infrastructure may

be vulnerable to attacks by hackers or breached due to employee error, malfeasance, or other disruptions.

Any such breach could compromise

our networks and the information stored there could be accessed, publicly disclosed, lost, or stolen.

If such an event were to occur

and cause interruptions in our operations, it could result in a disruption of our development programs and our business operations, whether

due to a loss of our trade secrets or other proprietary information or other similar disruptions.

For example, the loss of clinical trial

data from completed or future clinical trials could result in delays in our regulatory approval efforts and significantly increase our

costs to recover or reproduce the data.

To the extent that any disruption or security breach were to result in a loss of, or damage to,

our data or applications, or inappropriate disclosure of confidential or proprietary information, we could incur liability, our competitive

position could be harmed, and the further development and commercialization of our product candidates could be delayed.

business could suffer if we lose the services of, or fail to attract, key personnel.

depend highly upon the efforts of our senior management.

The loss of the services of these individuals could delay or prevent the achievement

of our research, development, marketing, or product commercialization objectives.

We do not have employment contracts with our other

key personnel.

We do not maintain any “key-man” insurance policies on any of our key employees and we do not intend to obtain

such insurance.

In addition, due to the specialized scientific nature of our business, we are highly dependent upon our ability to attract

and retain qualified scientific and technical personnel and consultants.

There is intense competition among major pharmaceutical and

chemical companies, specialized biotechnology firms and universities and other research institutions for qualified personnel in the areas

of our activities and we may be unsuccessful in attracting and retaining these personnel.

have experienced turnover in our senior management team, and the loss of one or more of our executive officers or key employees or an

inability to attract and retain highly skilled employees could adversely affect our business.

success depends largely upon the continued services of our key executive officers.

We have in the past and may in the future experience

changes in our executive management team resulting from the departure of executives or subsequent hiring of new executives, which may

be disruptive to our business.

To continue to develop our pipeline and execute our strategy, we also must attract and retain highly skilled

personnel in our industry.

toward managed health care, health technology assessment, and downward price pressures on medical products and services may limit our

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requiring payment of increased manufacturer rebates;

trend toward managed health care in the U.S.

and the concurrent growth of Health Maintenance Organizations (“HMOs”) and

similar organizations that can control or significantly influence the purchase of healthcare services and products;

federal, and foreign legislative proposals to control drug prices, reform healthcare or reduce government insurance programs.

the trend toward managed

health care in the U.S.

and the concurrent growth of Health Maintenance Organizations (“HMOs”) and similar organizations

that can control or significantly influence the purchase of healthcare services and products;

state, federal, and foreign

legislative proposals to control drug prices, reform healthcare or reduce government insurance programs.

cost containment measures that healthcare providers are instituting, including practice protocols and guidelines and clinical pathways,

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Disputes may arise regarding intellectual property subject to a licensing agreement, including:

scope of rights granted under the license agreement and other interpretation-related issues;

extent to which our technology and processes infringe intellectual property rights of the licensor that are not subject to the licensing

sublicensing of patent and other rights under our collaborative development relationships;

diligence obligations under the license agreement and what activities satisfy those diligence obligations;

inventorship or ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors

and us and our partners;

priority of invention of patented technology.

the scope of rights granted

under the license agreement and other interpretation-related issues;

the extent to which our

technology and processes infringe intellectual property rights of the licensor that are not subject to the licensing agreement;

the sublicensing of patent

and other rights under our collaborative development relationships;

our diligence obligations

under the license agreement and what activities satisfy those diligence obligations;

the inventorship or ownership

of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners;

the priority of invention

of patented technology.

any dispute over in-licensed intellectual property prevents or impairs our ability to maintain our current licensing arrangements on

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rights in technology resulting from the collaboration.

Regardless of such option, we may be unable to negotiate a license within the

Regardless of such an option, we may be unable to negotiate a license within the

specified timeframe or under terms that are acceptable to us.

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business significantly.

and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and other requirements

imposed by government patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.

maintenance fees, renewal fees, annuity fees and various other government fees on patents and/or applications will be due to be paid

to the USPTO and various government patent agencies outside of the United States over the lifetime of our licensed patents and/or applications

and any patent rights we may own in the future.

We generally rely on our outside counsel or our licensing partners to pay these fees

due to non-U.S.

patent agencies.

The USPTO and various non-U.S.

government patent agencies require compliance with several procedural,

documentary, fee payment and other similar provisions during the patent application process.

We employ reputable law firms and other

professionals to help us comply and we are also dependent on our licensors to take the necessary action to comply with these requirements

with respect to our licensed intellectual property.

In many cases, an inadvertent lapse can be cured by payment of a late fee or by other

means in accordance with the applicable rules.

There are situations, however, in which non-compliance can result in abandonment or lapse

of the patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction.

event, potential competitors might be able to enter the market and this circumstance could harm our business.

may not be able to protect our intellectual property rights throughout the world.

prosecuting, and defending patents on product candidates in all countries throughout the world would be prohibitively expensive, and

our intellectual property rights in some countries outside the United States could be less extensive than in the United States.

the laws of some foreign countries do not protect intellectual property rights to the same extent as federal and state laws in the United

Consequently, we may not be able to prevent third parties from practicing our inventions in all countries outside the United

States, or from selling or importing products made using our inventions in and into the United States or other jurisdictions.

may use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and, further, may

export otherwise infringing products to territories where we have patent protection, but enforcement is not as strong as that in the

United States.

These products may compete with our products and our patents or other intellectual property rights may not be effective

or sufficient to prevent them from competing.

companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions.

legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets

and other intellectual property protection, particularly those relating to biotechnology products, which could make it difficult for

us to stop the infringement of our patents or marketing of competing products in violation of our proprietary rights generally.

an April 2014 report from the Office of the United States Trade Representative identified a number of countries, including India and

China, where challenges to the procurement and enforcement of patent rights have been reported.

Several countries, including India and

China, have been listed in the report every year since 1989.

Proceedings to enforce our patent rights in foreign jurisdictions could

result in substantial costs and divert our efforts and attention from other aspects of our business, could put our patents at risk of

being invalidated or interpreted narrowly and our patent applications at risk of not issuing and could provoke third parties to assert

claims against us.

We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be

commercially meaningful.

Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain

a significant commercial advantage from the intellectual property that we develop or license.

patents covering our product candidates could be found invalid or unenforceable if challenged in court.

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could have a similar negative impact on our business, reputation, financial condition, results of operations and prospects.

property litigation could cause us to spend substantial resources and distract our personnel from their normal responsibilities.

may infringe our intellectual property rights or the intellectual property rights of our licensing partners, or we may be required to

defend against claims of infringement.

To counter infringement or unauthorized use claims or to defend against claims of infringement

can be expensive and time consuming.

Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property

claims may cause us to incur significant expenses and could distract our technical and management personnel from their normal responsibilities.

In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments and

if securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of

our common stock.

Such litigation or proceedings could substantially increase our operating losses and reduce the resources available

for development activities or any future sales, marketing, or distribution activities.

We may not have sufficient financial or other

resources to conduct such litigation or proceedings adequately.

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 and more mature and developed intellectual property

Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could adversely affect

our ability to compete in the marketplace.

may be subject to claims asserting that our employees, consultants, or advisors have wrongfully used or disclosed alleged trade secrets

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costs and be a distraction to management.

patent law could diminish the value of patents in general, thereby impairing our ability to protect our product candidates.

success depends heavily on intellectual property, especially on patents.

Obtaining and enforcing patents in the gene therapy industry

involves both technological and legal complexity.

Therefore, obtaining and enforcing patents is costly, time-consuming, and inherently

of 2013, the United States transitioned to a “first-to-file” system for deciding which party should be granted a patent when

two or more patent applications claiming the same invention are filed by different parties.

A third party that files a patent application

in the USPTO before us could therefore be awarded a patent covering an invention of ours even if we made the invention before it was

made by the third party.

The change to “first-to-file” from “first-to-invent” is one of the changes to the patent

laws of the U.S.

resulting from the Leahy-Smith America Invents Act (the “AIA”).

Among some of the other significant changes

to the patent laws are changes that limit where a patentee may file a patent infringement suit and provide opportunities for third parties

to challenge any issued patent in the USPTO via procedures including post-grant and inter partes review.

These adversarial actions

at the USPTO review patent claims without the presumption of validity afforded to U.S.

patents in lawsuits in U.S.

federal courts and

use a lower burden of proof than that used in litigation in U.S.

federal courts.

Therefore, it is generally considered easier for a competitor

or third party to have a patent invalidated in a USPTO post-grant review or inter partes review proceeding than in a litigation

federal court.

If any of our patents are challenged by a third party in such a USPTO proceeding, there is no guarantee that

we or our licensors or collaborators will be successful in defending the patent, which would result in a loss of the challenged patent

The AIA and its implementation could increase the uncertainties and costs surrounding the prosecution of our patent applications

and the enforcement or defense of any issued patents, all of which could harm our business and financial condition.

also may be subject to a third-party pre-issuance submission of prior art to the USPTO or become involved in other contested proceedings

such as opposition, derivation, reexamination, inter partes review, post-grant review or interference proceedings challenging

our patent rights or the patent rights of others.

An adverse determination in any such submission, proceeding or litigation could reduce

the scope of, or invalidate, our patent rights, allow third parties to commercialize our technology or products and compete directly

with us, without payment to us, or result in our inability to manufacture or commercialize products without infringing third-party patent

In addition, if the breadth or strength of protection provided by our patents and patent applications is threatened, it could

dissuade companies from collaborating with us to license, develop or commercialize current or future products.

we do not obtain patent term extension and data exclusivity for our product candidates, our business may be harmed.

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products following our patent expiration, and our revenue could be materially reduced.

property rights do not necessarily address all potential threats.

degree of future protection afforded by our intellectual property rights is uncertain because intellectual property rights have limitations,

and such rights may not adequately protect our business or permit us to maintain our competitive advantage.

may be able to make gene therapy products that are similar to our product candidates but that are not covered by the claims of the

patents that we license or may own in the future;

or our license partners or current or future collaborators, might not have been the first to make the inventions covered by the issued

patent or pending patent application that we license or may own in the future;

or our license partners or current or future collaborators, might not have been the first to file patent applications covering certain

of our or their inventions;

may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our owned or

licensed intellectual property rights;

is possible that our pending patent applications or those that we may own in the future will not lead to issued patents;

patents that we hold rights to may be held invalid or unenforceable, including as a result of legal challenges by our competitors;

competitors might conduct research and development activities in countries where we do not have patent rights and then use the information

learned from such activities to develop competitive products for sale in our major commercial markets;

may not develop additional proprietary technologies that are patentable;

patents of others may have an adverse effect on our business;

may choose not to file a patent application for certain trade secrets or know-how, and a third party may subsequently file a patent

application covering such intellectual property.

any of these events occur, they could significantly harm our business, financial condition, results of operations and prospects.

related to our financial condition and capital requirements

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fund operations in the future.

have recorded minimal revenue to date and have incurred an accumulated deficit of approximately $695.3 million through December 31, 2022.

The net loss for the year ended December 31, 2022, was $39.7 million, including impairment charges of $5.6 million.

Our losses have resulted

principally from costs incurred in research and development activities related to our efforts to develop clinical drug candidates and

from the associated administrative costs.

have recorded minimal revenue to date and have incurred an accumulated deficit of $749.5 million through December 31, 2023.

for the year ended December 31, 2023, was $54.2 million.

Our losses have resulted principally from costs incurred in research and development

activities related to our efforts to develop clinical drug candidates and from the associated administrative costs.

require substantial capital for our development programs and operating expenses, to pursue regulatory clearances and to prosecute and

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We anticipate that our expenses will increase substantially if and as we:

regulatory and marketing approvals for our product candidates that successfully complete clinical studies;

our research and preclinical and clinical development of our product candidates;

develop the manufacturing process for our vectors or our product candidates;

the scope of our current clinical studies for our product candidates;

or add additional manufacturers or suppliers;

to identify and validate additional product candidates;

or in-license other product candidates and technologies;

milestone or other payments under any license agreements;

protect and expand our intellectual property portfolio;

a sales, marketing and distribution infrastructure in the United States and Europe to commercialize any products for which we may

obtain marketing approval;

and retain skilled personnel;

additional infrastructure to support our operations as a larger public company and our product development and planned future commercialization

efforts, including manufacturing capacity;

any delays or encounter issues with any of the above.

seek regulatory and marketing

approvals for our product candidates that successfully complete clinical studies;

continue our research and

preclinical and clinical development of our product candidates;

further develop the manufacturing

process for our vectors or our product candidates;

expand the scope of our

current clinical studies for our product candidates;

change or add additional

manufacturers or suppliers;

seek to identify and validate

additional product candidates;

acquire or in-license other

product candidates and technologies;

make milestone or other

payments under any license agreements;

maintain, protect and expand

our intellectual property portfolio;

establish a sales, marketing

and distribution infrastructure in the United States and Europe to commercialize any products for which we may obtain marketing approval;

attract and retain skilled

build additional infrastructure

to support our operations as a larger public company and our product development and planned future commercialization efforts, including

manufacturing capacity;

experience any delays or

encounter issues with any of the above.

net losses we incur may fluctuate significantly from quarter to quarter and year to year, such that a period-to-period comparison of

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of December 31, 2023, our cash, cash equivalents, restricted cash and short-term investments were $52.6 million.

Following a comprehensive

portfolio review in early 2022, we have decided to focus our research and development resources on the EB-101 program with the objective

of reducing operating expenses and extending our cash runway.

As part of this portfolio prioritization, we have intensified our pursuit

of a strategic partnership to take over development activities for our AAV-based gene therapy ABO-102 for MPS IIIA and we have discontinued

development of our AAV-based gene therapy ABO-101 for MPS IIIB.

Based upon these current operating plans, our ability to access additional

financial resources and/or our financial flexibility to further reduce operating expenses if required, we believe that we have sufficient

resources to fund operations through at least the next 12 months from the date of the issuance of our consolidated financial statements.

However, our operating plan may change as a result of many factors currently unknown to us, and we may need to seek additional funds

sooner than planned, through public or private equity, government or other third-party funding, marketing and distribution arrangements

and other collaborations, strategic alliances and licensing arrangements or a combination of these approaches.

In any event, we will

require additional capital to obtain potential regulatory approval for, and to potentially commercialize, our product candidates.

if we believe we have sufficient funds for our current or future operating plans, we may seek additional capital if market conditions

are favorable or if we have specific strategic objectives.

Based upon our existing

cash resources and our recently announced credit facility of up to $50 million, we believe that we have sufficient resources to fund

operations through at least the next 12 months from the date of the issuance of our consolidated financial statements.

However, our operating

plan may change as a result of many factors currently unknown to us, and we may need to seek additional funds sooner than planned, through

public or private equity, government or other third-party funding, marketing and distribution arrangements and other collaborations,

strategic alliances and licensing arrangements or a combination of these approaches.

In any event, we will require additional capital

to obtain potential regulatory approval for, and to potentially commercialize, our product candidates.

Even if we believe we have sufficient

funds for our current or future operating plans, we may seek additional capital if market conditions are favorable or if we have specific

strategic objectives.

additional fundraising efforts may divert our management from their day-to-day activities, which may adversely affect our ability to

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that are favorable to us may force us to delay, reduce or eliminate our development programs or commercialization efforts.

will need to raise additional capital to fund our future operations and we cannot be certain that funding will be available to us on

acceptable terms on a timely basis, or at all.

We expect to continue to spend substantial amounts on regulatory approval efforts, product

development (including commercialization activities), and conducting potential future pre-clinical or clinical trials for our product

Our ability to raise capital through the sale of securities may be limited by our number of authorized shares of common stock

and various rules of the SEC and the Nasdaq that place limits on the number and dollar amount of securities that we may sell.

to raise additional funds on acceptable terms or at all, we may be unable to complete planned preclinical and clinical trials, obtain

approval of our product candidates from the FDA and other regulatory authorities, or successfully commercialize any of our product candidates.

may need to raise additional capital to fund our future operations and we cannot be certain that funding will be available to us on acceptable

terms on a timely basis, or at all.

We expect to continue to spend substantial amounts on regulatory approval efforts, product development

(including commercialization activities), and conducting potential future pre-clinical or clinical trials for our product candidates.

Our ability to raise capital through the sale of securities may be limited by our number of authorized shares of common stock and various

rules of the SEC and the Nasdaq that place limits on the number and dollar amount of securities that we may sell.

If we fail to raise

additional funds on acceptable terms or at all, we may be unable to complete planned preclinical and clinical trials, obtain approval

of our product candidates from the FDA and other regulatory authorities, or successfully commercialize any of our product candidates.

In addition, we could be forced to delay, discontinue, or curtail product development, or forego licensing in attractive business opportunities.

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market price of our common stock could fluctuate significantly in response to various factors and events, including:

ability to integrate operations, technology, products, and services;

ability to execute our business plan;

results below expectations;

announcements

concerning product development results, including clinical trial results;

or legal developments in the U.S.

our ability to integrate

operations, technology, products, and services;

our ability to execute

our business plan;

operating results below

expectations;

announcements concerning

product development results, including clinical trial results;

regulatory or legal developments

or EU, including decisions from regulatory agencies relating to our product candidates;

or public concern about the safety of our potential products;

issuance of additional securities, including debt or equity or a combination thereof, which will be necessary to fund our operating

announcements

of technological innovations or new products by us or our competitors;

of any strategic relationship;

developments, including, without limitation, changes in healthcare policies or practices or third-party reimbursement policies;

and other external factors;

period-to-period

fluctuations in our financial results;

an active trading market in our common stock develops and is maintained.

litigation or public concern

about the safety of our potential products;

our issuance of additional

securities, including debt or equity or a combination thereof, which will be necessary to fund our operating expenses;

announcements of technological

innovations or new products by us or our competitors;

loss of any strategic relationship;

industry developments,

including, without limitation, changes in healthcare policies or practices or third-party reimbursement policies;

economic and other external

period-to-period fluctuations

in our financial results;

whether an active trading

market in our common stock develops and is maintained.

addition, the securities markets have from time-to-time experienced significant price and volume fluctuations that are unrelated to the

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stockholders and the terms of these securities may include liquidation or other preferences that adversely affect our existing stockholders.

have not paid cash dividends in the past and do not expect to pay cash dividends in the foreseeable future.

Any return on investment

may be limited to the value of our common stock.

have never paid cash dividends on our common stock and do not anticipate paying cash dividends on our common stock in the foreseeable

The payment of dividends on our capital stock will depend on our earnings, financial condition and other business and economic

factors affecting us at such time as the board of directors may consider relevant.

If we do not pay dividends, our common stock may be

less valuable because a return on stockholder investment will only occur if the common stock price appreciates.

quarterly operating results may fluctuate significantly.

expect our operating results to be subject to quarterly fluctuations.

Our net loss and other operating results will be affected by numerous

factors, including:

in the level of expenses related to our development programs;

or termination of clinical trials;

intellectual property infringement lawsuit or arbitration in which we are, or may become, involved;

developments affecting our product candidates;

execution of any collaborative, licensing or similar arrangements, and the timing of payments we may make or receive under these

arrangements.

our quarterly operating results fall below the expectations of investors or securities analysts, the price of our common stock could

decline substantially.

Furthermore, any quarterly fluctuations in our operating results may, in turn, cause the price of our common stock

to fluctuate substantially.

of our charter documents could discourage an acquisition of our company that would benefit our stockholders and may have the effect of

entrenching, and making it difficult to remove, management.

of our Certificate of Incorporation and Bylaws may make it more difficult for a third party to acquire control of us, even if a change

in control would benefit our stockholders.

In particular, shares of our preferred stock may be issued in the future without further stockholder

approval and upon such terms and conditions, and having such rights, privileges and preferences, as our Board of Directors may determine,

including, for example, rights to convert into our common stock.

The rights of the holders of our common stock will be subject to, and

may be adversely affected by, the rights of the holders of any of our preferred stock that may be issued in the future.

of our preferred stock, while providing desirable flexibility in connection with possible acquisitions and other corporate purposes,

could have the effect of making it more difficult for a third party to acquire control of us.

This could limit the price that certain

investors might be willing to pay in the future for shares of our common stock and discourage these investors from acquiring a majority

of our common stock.

Further, the existence of these corporate governance provisions could have the effect of entrenching management

and making it more difficult to change our management.

ability to use our net operating loss carry forwards may be subject to limitation.

a change of more than 50% in the ownership of a company’s stock, by value, over a three-year period constitutes an ownership change

federal income tax purposes.

An ownership change may limit our ability to use our net operating loss carryforwards attributable

to the period prior to the change.

As a result, if we earn net taxable income, our ability to use our pre-change net operating loss carryforwards

to offset U.S.

federal taxable income may become subject to limitations, which could potentially result in increased future tax liability

As of December 31, 2022, we had net operating loss carryforwards aggregating approximately $359.0 million.

UNRESOLVED STAFF COMMENTS

federal income tax purposes or applicable state tax law.

An ownership change may limit our ability to use our net operating

loss carryforwards attributable to the period prior to the change.

As a result, if we earn net taxable income, our ability to use our

pre-change net operating loss carryforwards to offset U.S.

federal and state taxable income may become subject to limitations, which

could potentially result in increased future tax liability for us.

As of December 31, 2023, we had net operating loss carryforwards aggregating

$377.5 million.

related to cybersecurity

disruptions of information technology (“IT”) systems, breaches of data security, or unauthorized disclosures of personal

information (including sensitive personal information) could adversely affect our business and could subject us to liability or reputational

operate information systems that contain limited amounts of client data.

As a routine element of our business, we collect, analyze, and

retain data pertaining to the clinical trials we conduct for our products.

Unauthorized third parties could attempt to gain entry to

such information systems to steal data or disrupt the systems or for financial gain.

Like other companies we may experience threats and

incursions to our data and systems, including malicious software and viruses, phishing, business email compromise and social engineering

attacks or other cyber-attacks.

The number and complexity of these threats continue to increase over time.

have implemented and maintain security systems measures and safeguards, which we believe to be reasonable, to protect our information

systems and confidential information, including personal information, and that of our customers, clients and suppliers that is held or

processed by us, against unauthorized access or disclosure and to prevent, detect, contain, respond to, and mitigate security-related

threats and potential incidents.

We undertake ongoing improvements to the security of our systems, connected devices, and information-sharing

products in order to minimize potential vulnerabilities, in accordance with industry and regulatory standards.

Despite such efforts,

our safeguards may fail, or we may be subject to breaches of our security resulting in unauthorized access to our facilities or information

systems and the information we are trying to protect.

Moreover, our business or operations may be affected in the event our customers,

clients and suppliers experience data security incidents, cyber-attacks or extended interruptions of their services or systems.

are continuously evaluating and, where appropriate, enhancing our IT systems to address our planned growth, including to support our

planned manufacturing operations.

There are inherent costs and risks associated with implementing the enhancements to our IT systems,

including potential delays in access to, or errors in, critical business and financial information, substantial capital expenditures,

additional administrative time and operating expenses, retention of sufficiently skilled personnel to implement and operate the enhanced

systems, demands on management time, and costs of delays or difficulties in transitioning to the enhanced systems, any of which could

harm our business and results of operations.

In addition, the implementation of enhancements to our IT systems may not result in productivity

improvements at a level that outweighs the costs of implementation, or at all.

we do not believe cybersecurity incidents have resulted in any material impact on our business, operations or financial results or our

ability to service our customers or run our business, past and future incidents resulting in unauthorized access to our facilities or

information systems, or those of our suppliers, or accidental loss or disclosure of proprietary or confidential information about us,

our clients or our customers could result in, among other things, a total shutdown of our systems that would disrupt our ability to conduct

business or pay vendors and employees, violations of applicable privacy and other laws, significant legal and financial exposure, damage

to our reputation, and a loss of investor confidence in our security measures.

Additional impacts from cybersecurity incidents could

include remediation costs to our customers or business partners, such as liability for stolen assets or information, repairs of system

damage, and incentives for continued business;

increased cybersecurity protection costs, which may include the costs of making organizational

changes, deploying additional personnel, resources and security technologies, training employees, and engaging third-party experts and

lost revenue resulting from the unauthorized use of proprietary information or the failure to retain or attract business

partners following an incident;

increased insurance premiums;

and damage to the Company’s competitiveness, stock price, and long-term

shareholder value.

In addition, cybersecurity risks and data security incidents could lead to unfavorable publicity, governmental inquiry

and oversight, regulatory actions by federal, state and non-U.S.

governmental authorities, litigation by affected parties and possible

financial obligations for damages related to the theft or misuse of such information, any of which could have a material adverse effect

on our profitability and cash flow.

information regarding our processes and practices related to information and cybersecurity, please see Item 1C of this report, “Cybersecurity”.

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.

What changed 10-K

Item 1A. Risk Factors