What changed in this filing

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Item 1A. Risk Factors

2023-03-29 compared with 2022-03-31 · 296 added, 367 removed, 1313 unchanged (34% of the section changed)

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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

Additionally, regulatory requirements governing cell and gene therapy products

have evolved and may continue to change in the future.

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.

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.

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 has 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 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.

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

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

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

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 coverage.

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.

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 to ours.

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.

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

We may not be able to protect our trade secrets in court, and intellectual property

litigation could cause us to spend substantial resources.

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.

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.

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.

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

to fund operations in the future.

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.

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.

can be no assurance that we will be able to regain compliance with continued listing standards

of the Nasdaq.

related to the discovery and development of our product candidates

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 subsequently obtaining regulatory approval.

Only a few gene therapy products have been

approved in the U.S.

have concentrated our therapeutic product research and development efforts on our cell and gene therapy platform, and our future

success depends on the successful development of this therapeutic approach.

There can be no assurance that any development problems we

experience in the future related to our gene and cell therapy platform will not cause significant delays or unanticipated costs, or that

such development problems can be solved.

We may also experience delays in developing a sustainable, reproducible and commercial-scale

manufacturing process or transferring that process to commercial partners, which may prevent us from completing our clinical studies

or commercializing our products on a timely or profitable basis, if at all.

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 subsequently obtaining regulatory approval.

Only a few gene therapy products have been approved

have concentrated our therapeutic product research and development efforts on our cell and gene therapy platform, and our future success

depends on the successful development of this therapeutic approach.

There can be no assurance that any development problems we experience

in the future related to our cell and gene therapy platform will not cause significant delays or unanticipated costs, or that such development

problems can be solved.

We may also experience delays in developing a sustainable, reproducible and commercial-scale manufacturing process

or transferring that process to commercial partners, which may prevent us from completing our clinical studies or commercializing our

products on a timely or profitable basis, if at all.

addition, the clinical study requirements of the FDA, the EMA, and other regulatory agencies and the criteria these regulators use to

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requirements governing cell and gene therapy products have evolved and may continue to change in the future.

For example, the

FDA has established the OTAT within CBER to consolidate the review of gene therapy and related products, and the Cellular, Tissue and

Gene Therapies Advisory Committee to advise CBER on its review.

For example, the FDA has

established the Office Tissues and Advanced Therapies within CBER to consolidate the review of gene therapy and related products, and

the Cellular, Tissue and Gene Therapies Advisory Committee to advise CBER on its review.

requirements in the United States and in other jurisdictions governing gene therapy products have changed frequently and will continue

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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;

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

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

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

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;

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

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;

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.

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.

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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in the future.

If patients are unwilling to participate in our cell and gene therapy studies because of negative publicity from

adverse events in the biotechnology or gene therapy industries or for other reasons, including competitive clinical studies for similar

patient populations, the timeline for recruiting patients, conducting studies, and obtaining regulatory approval of potential products

may be delayed.

If patients are unwilling to participate in our cell and gene therapy studies because of negative publicity from adverse

events in the biotechnology or gene therapy industries or for other reasons, including competitive clinical studies for similar patient

populations, the timeline for recruiting patients, conducting studies, and obtaining regulatory approval of potential products may be

These delays could result in increased costs, delays in advancing our product development, delays in testing the effectiveness

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and nature of the patient population;

● eligibility

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 therapies;

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;

● availability

of competing therapies and clinical studies;

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

● 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;

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;

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

modifications

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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;

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

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.

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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compliance with post-approval regulations may have a negative effect on our operating results and financial condition.

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

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

the past two years, the COVID-19 pandemic has resulted in intermittent shutdowns of non-essential businesses throughout the world.

impact of the COVID-19 pandemic has also resulted in social, economic, and labor instability in the countries in which we, or the third

parties with whom we engage, operate.

At various times, the COVID-19 pandemic has substantially burdened healthcare systems worldwide,

sometimes delaying enrollment in and progression of clinical trials.

Required inspections and reviews by regulatory agencies have also

been delayed at times due to the focus of resources on COVID-19, as well as travel and other restrictions.

For example, our Phase 3 VIITAL TM

clinical trial was temporarily paused in March 2020 due to the COVID-19 pandemic and the restrictions established by our clinical

trial site at Stanford University in Palo Alto, California, but resumed in June 2020.

Significant delays in the timing of our clinical

trials and in regulatory reviews could adversely affect our ability to commercialize our product candidates.

may experience disruptions from COVID-19 that impact our business, supply chain, manufacturing operations, clinical trials, and pre-clinical

studies, including:

of key clinical trial activities, including limitations on travel imposed or recommended by federal or state governments, employers,

need to postpone, modify, suspend, or terminate clinical trials;

may withdraw from clinical trials;

may experience study or manufacturing deviations or noncompliance, requiring that we consult with regulatory authorities, and IRBs,

and which may compromise the ultimate study results or quality of the manufactured products;

delays or inability to obtain raw materials, ingredients, or other necessary supplies, including if third party suppliers need to

prioritize other products or customers over us, including under the Defense Production Act;

or difficulties in enrolling patients in our clinical trials;

or difficulties in clinical site initiation, including difficulties in recruiting clinical site investigators and clinical site staff;

or difficulties in manufacturing clinical drug material;

of healthcare resources away from the conduct of clinical trials, including the diversion of hospitals serving as our clinical trial

sites and hospital staff supporting the conduct of our clinical trials;

in employee resources that would otherwise be focused on the conduct of our manufacturing operations, clinical trials, and preclinical

studies, including because of sickness of employees or their families or the desire of employees to avoid contact with large groups

ultimate impact of the COVID-19 pandemic remains uncertain and subject to change.

Due to the potential impact of the COVID-19 outbreak

on clinical trials, drug development, and manufacturing, the FDA issued guidance concerning how sponsors and investigators may address

these challenges, as well as guidance specific to gene therapies and comparable foreign regulatory authorities have done likewise.

guidance recommended that gene therapy manufacturers perform a risk assessment to identify, evaluate, and mitigate factors that may allow

for the transmission of the SARS-CoV-2 virus.

The FDA specifically recommended that manufacturers consider areas, such as donor assessments,

cellular and tissue source materials, manufacturing processes, manufacturing facility controls, product and material testing, and the

number of individuals who may receive the product.

Per the guidance, risk assessment and mitigation strategies should be submitted to

COVID-19 pandemic may also continue to result in changes in laws and regulations.

For example, in March 2020, the U.S.

Congress passed

the Coronavirus Aid, Relief, and Economic Security Act (“CARES Act”), which includes various provisions regarding FDA drug

shortage reporting requirements, as well as provisions regarding supply chain security, such as risk management plan requirements, and

the promotion of supply chain redundancy and domestic manufacturing.

This and any future changes in law may require that we change our

internal processes and procedures to ensure continued compliance.

These changes could have a material impact on our ability to access

the capital markets as needed and on our operations and business, and those of the third parties on which we rely.

related to manufacturing

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or otherwise adversely affect our business.

are susceptible to production interruptions that may impede our ability to manufacture cell and gene therapy products and produce

an adequate product supply to support clinical trials and potentially future commercialization.

Several factors could cause production

interruptions, including equipment malfunctions, facility contamination, raw material shortages or contamination, natural disasters,

public health emergencies such as the COVID-19 pandemic, disruption in utility services, human error, or disruptions in the operations

of our suppliers.

Our products and product candidates are biologic drugs requiring processing steps that are more complex than those

required for most chemical pharmaceuticals.

We characterize our processes and products, and perform testing to ensure the safety, quality

and efficacy of each product produced.

While we take significant measures to fully understand and characterize each product, the steps

we take may not be sufficient to ensure that a given lot will perform in the intended manner.

are susceptible to production interruptions that may impede our ability to manufacture cell and gene therapy products and produce an

adequate product supply to support clinical trials and potentially future commercialization.

Several factors could cause production interruptions,

including equipment malfunctions, facility contamination, raw material shortages or contamination, natural disasters, public health emergencies

such as the COVID-19 pandemic, disruption in utility services, human error, or disruptions in the operations of our suppliers.

and product candidates are biologic drugs requiring processing steps that are more complex than those required for most chemical pharmaceuticals.

We characterize our processes and products, and perform testing to ensure the safety, quality and efficacy of each product produced.

While we take significant measures to fully understand and characterize each product, the steps we take may not be sufficient to ensure

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.

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control for certain aspects of manufacturing activities;

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

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;

● 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.

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.

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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or liabilities, which could harm our business, financial condition, results of operations and prospects.

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

of operations and financial condition.

could be negatively impacted by the widespread outbreak of an illness, communicable disease, or any other public health crisis that results

in economic or trade disruptions, including the disruption of global supply chains.

The COVID-19 pandemic negatively impacted the economy

on a global, national, and local level, disrupted global supply chains, and created volatility and disruption of financial markets.

from governmental authorities and companies to reduce the spread of COVID-19 affected economic activity through various containment measures

including, among others, business closures, work stoppages, quarantine and work-from-home guidelines, limiting capacity at public spaces

and events, vaccination requirements, or restrictions of global and regional travel.

Another outbreak of an illness, a communicable disease,

or any other public health crisis, and any resulting impacts, such as an extended period of global supply chain and/or economic disruption,

labor shortages, or government-mandated actions in response to such public health crisis could materially affect our business, results

of operations, access to sources of liquidity, and financial condition.

related to our reliance on third-parties

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we rely on third parties to manufacture our vectors and our product candidates, and because we collaborate with various organizations

and academic institutions on the advancement of our cell and gene therapy platform, we must, at times, share trade secrets with

We seek to protect our proprietary technology in part by entering into confidentiality agreements and, if applicable, material

transfer agreements, collaborative research agreements, consulting agreements or other similar agreements with our collaborators, advisors,

employees, and consultants prior to beginning research or disclosing proprietary information.

These agreements typically limit the rights

of the third parties to use or disclose our confidential information, such as trade secrets.

Despite the contractual provisions employed

when working with third parties, the need to share trade secrets and other confidential information increases the risk that such trade

secrets become known by our competitors, are inadvertently incorporated into the technology of others, or are disclosed or used in violation

and academic institutions on the advancement of our cell and gene therapy platform, we must, at times, share trade secrets with them.

We seek to protect our proprietary technology in part by entering into confidentiality agreements and, if applicable, material transfer

agreements, collaborative research agreements, consulting agreements or other similar agreements with our collaborators, advisors, employees,

and consultants prior to beginning research or disclosing proprietary information.

These agreements typically limit the rights of the

third parties to use or disclose our confidential information, such as trade secrets.

Despite the contractual provisions employed when

working with third parties, the need to share trade secrets and other confidential information increases the risk that such trade secrets

become known by our competitors, are inadvertently incorporated into the technology of others, or are disclosed or used in violation

of these agreements.

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our trade secrets would impair our competitive position and have an adverse impact on our business.

associated with commercializing our product candidates

related to with commercializing our product candidates

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.

As a result, our

ability to generate revenue from product commercialization may be further delayed.

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

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

Obtaining FDA approval of any

other product candidate generally requires significant research and testing, referred to as preclinical studies, as well as human tests,

referred to as clinical trials.

Satisfaction of the FDA’s regulatory requirements typically takes many years, depends upon the

type, complexity and novelty of the product candidate and requires substantial resources for research, development and testing.

predict whether our research and clinical approaches will result in products that the FDA considers safe for humans and effective for

indicated uses.

The FDA has substantial discretion in the product approval process and may require us to conduct additional preclinical

and clinical testing or to perform post-marketing studies.

The approval process may also be delayed by changes in government regulation,

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.

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

In addition, the FDA could determine that we must test additional

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

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

future potential product candidate.

Failure to obtain FDA approval of any of our product candidates will severely undermine our business

by leaving us without the ability to generate additional accretive revenues.

There is no guarantee that we will ever be able to develop

or acquire other product candidates.

In foreign jurisdictions, we must receive approval from the appropriate regulatory authorities before

we can commercialize any products or product candidates outside the U.S.

Foreign regulatory approval processes generally include all

of the risks and uncertainties associated with the FDA approval procedures described above.

We cannot assure you that we will receive

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

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

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.

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

may not generate sufficient revenue and we may not be able to achieve or sustain profitability.

Our efforts to educate the medical community

and third-party payors on the benefits of our drug candidates may require significant resources and may never be successful.

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

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third-party-payors’

increasing challenges to the prices charged for medical products and services, including

by limiting coverage and reimbursement and requiring payment of increased manufacturer rebates;

increasing challenges to the prices charged for medical products and services, including by limiting coverage and reimbursement and

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.

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.

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

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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 agreement;

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

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;

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.

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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 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.

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.

relating to our financial condition and capital requirements

related to our financial condition and capital requirements

have experienced a history of losses;

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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, 2021 was $84.9 million, including a goodwill impairment charge of $32.5

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.

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.

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;

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

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

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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;

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;

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.

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of December 31, 2022, our cash, cash equivalents, restricted cash and short-term investments were $52.5 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.

E ven 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

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.

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

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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.

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.

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.

Currently, we do not have sufficient shares of common stock authorized under our Certificate of Incorporation

to conduct an offering of common stock.

If we fail to raise additional funds on acceptable terms or at all, we may be unable to complete

planned preclinical and clinical trials or obtain approval of our product candidates from the FDA and other regulatory authorities.

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

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.

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

Any additional sources of financing will likely involve the issuance of our equity or debt securities, which will have a dilutive effect

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or legal developments in the U.S.

or EU, including decisions from regulatory agencies relating

to our product candidates;

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 expenses;

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

announcements

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of any strategic relationship;

developments, including, without limitation, changes in healthcare policies or practices

or third-party reimbursement policies;

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

and other external factors;

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or termination of clinical trials;

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

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.

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

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and making it more difficult to change our management.

can be no assurance that we will be able to regain and maintain compliance with continued listing standards of the Nasdaq Capital Market.

Nasdaq Capital Market’s continued listing standards for our common stock require, among other things, that (i) we maintain a closing

bid price for our common stock of at least $1.00, and (ii) we maintain:

(A) stockholders’ equity of $2.5 million;

(B) market value

of listed securities of $35 million;

or (C) net income from continuing operations of $500,000 in the most recently completed fiscal year

or in two of the last three most recently completed fiscal years.

Any failures to satisfy any continued listing requirements could lead

to the receipt of a deficiency notice from the Nasdaq and ultimately to a delisting from trading of our common stock.

November 16, 2021, we received a deficiency letter notifying us that we had not maintained a closing bid price for our common stock of

at least $1.00 for a 30-day period.

In accordance with Nasdaq rules, we have been provided an initial period of 180 calendar days, or

until May 16, 2022 (the “Compliance Date”), to regain compliance with the bid price requirement.

If we do not regain compliance

with the bid price requirement by the Compliance Date, we may be eligible for an additional 180 calendar day compliance period.

do not regain compliance with the bid price requirement by May 16, 2022 and are not eligible for an additional compliance period at that

time, our common stock will be subject to delisting from the Nasdaq Capital Market.

We cannot be certain that we will be able to regain

compliance and then maintain compliance with the minimum bid price and the other standards in order to maintain a listing of our common

stock on the Nasdaq Capital Market.

our common stock were delisted from the Nasdaq Capital Market, among other things, this could result in a number of negative implications,

including reduced liquidity in our common stock as a result of the loss of market efficiencies associated with the Nasdaq and the loss

of federal preemption of state securities laws as well as the potential loss of confidence by suppliers, customers and employees, institutional

investor interest, fewer business development opportunities, greater difficulty in obtaining financing and breaches of certain contractual

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

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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