What changed in this filing

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Item 1A. Risk Factors

2025-03-20 compared with 2024-03-18 · 242 added, 334 removed, 1227 unchanged (32% of the section changed)

6 unchanged sentences

include, but are not limited to the following:

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.

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 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

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.

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.

could experience production problems in our manufacturing facility 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.

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.

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.

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

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.

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.

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

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.

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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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.

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 contract research organizations (“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.

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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Patient enrollment is affected by factors including:

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.

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.

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

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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;

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.

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.

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

15 unchanged sentences

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

Complete Response Letter related to our Biologics License Application for pz-cel for the treatment of patients with recessive dystrophic

epidermolysis bullosa may impair our ability to successfully commercialize pz-cel.

April 2024, we received a CRL related to our BLA for pz-cel for the treatment of patients with RDEB.

In the CRL, the FDA noted that

certain additional information needed to satisfy CMC requirements must be satisfactorily resolved before the application can be approved.

In August 2024, we completed a Type A Meeting with the FDA to discuss our forthcoming resubmission of our BLA and in October 2024,

we resubmitted our BLA.

The FDA notified the Company in November 2024 that the BLA was accepted for review, with an assigned PDUFA

target action date of April 29, 2025.

A delay in receiving approval of the BLA could shorten any periods during which we may have the

exclusive right to commercialize our pz-cel or allow our competitors to bring products to market before we do.

This may impair our ability

to successfully commercialize pz-cel.

If any of the foregoing were to occur, our business, financial condition, results of operations,

and prospects will be materially harmed.

related to manufacturing

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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:

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.

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.

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

related to with commercializing our product candidates

related to commercializing our product candidates

we do not obtain the necessary U.S.

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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.

commercialization of, and our ability to derive product revenues from, pz-cel 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.

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

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.

and perceived efficacy and safety of pz-cel;

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 pz-cel;

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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requiring payment of increased manufacturer rebates;

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.

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.

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:

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.

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.

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

258 unchanged sentences

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

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.

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.

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, 2024, our cash, cash equivalents, restricted cash and short-term investments were $98.1 million.

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.

Based upon our

existing cash resources and the $4.8 million in net proceeds from our subsequent sales of common stock under stock under the ATM

Agreement, 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.

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 may 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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the SEC or other regulatory authorities.

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 commercialization efforts.

may 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 commercialization efforts.

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

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

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

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.

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

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.

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.

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

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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