U.S. FDA Approves ZEVASKYN (prademagene zamikeracel), the First and Only Cell-Based Gene Therapy for Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) - ZEVASKYN fills a critical need for people living with

FDA Approves ZEVASKYN (prademagene zamikeracel), the First and Only Cell-Based Gene Therapy for Patients with Recessive Dystrophic

Epidermolysis Bullosa (RDEB)

ZEVASKYN fills a critical need for people living with RDEB, a debilitating dermatological condition with no cure -

Approval based on the pivotal Phase 3 VIITAL study, showing ZEVASKYN resulted in significant wound healing and pain reduction

after a single treatment with a favorable safety profile -

ZEVASKYN to be available through Qualified Treatment Centers beginning in 3Q 2025 -

Abeona Assist patient services program offers personalized support for eligible patients and families throughout their treatment

journey with ZEVASKYN -

Rare Pediatric Disease Priority Review Voucher (PRV) granted by FDA -

Abeona Therapeutics to host conference call today, Tuesday, April 29, 2025, at 8:00 a.m. ET -

April 29, 2025 - Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the U.S. Food and Drug Administration (FDA) has approved

ZEVASKYN (pronounced as ZEE-vah-skin') (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel,

as the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic

epidermolysis bullosa (RDEB), a serious and debilitating genetic skin disease. There is no cure for RDEB and ZEVASKYN is the only FDA-approved

product to treat RDEB wounds with a single application.

approval of ZEVASKYN represents a pivotal moment in the treatment of RDEB, answering the call of people living with the clinical, economic,

and human impact of this devastating disease," said Vish Seshadri, Ph.D., M.B.A., Chief Executive Officer of Abeona. "We

have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic

and prone to infection. Through a single surgical application, ZEVASKYN can now offer people with RDEB the opportunity for wound healing

and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal Phase 3 study."

gene therapy with robust body of clinical evidence

FDA approval of ZEVASKYN is based on the pivotal Phase 3 VIITAL study (NCT04227106), a multi-center, randomized, intrapatient-controlled

trial that met its two co-primary efficacy endpoints demonstrating statistically significant healing of 50 percent or more from baseline

in large chronic RDEB wounds, and pain reduction from baseline as assessed by the Wong-Baker FACES scale, as evaluated at six months

43 large and chronic wounds treated with a single application of ZEVASKYN, 81 percent of wounds showed 50 percent or more healing (P<0.0001)

as evaluated at six months, compared to 16 percent in 43 matched control wounds treated with standard of care. The most common adverse

events were observed in fewer than five percent of patients and included procedural pain and itch.

was well-tolerated and efficacious in clinical studies, providing clinically meaningful improvements in wound healing, pain reduction,

and other associated symptoms in large chronic RDEB wounds after a single application," said Jean Tang, M.D., Ph.D., professor

of dermatology and lead principal investigator of the VIITAL study. "In the completed Phase 1/2a study of ZEVASKYN, we have

observed wound healing and pain reduction that have lasted for years after a single application. Today we can celebrate the availability

of an exciting new therapeutic option made possible by the incredible courage of patients and families who participated in these clinical

the Phase 1/2a study of ZEVASKYN (NCT01263379), a single center, open label study in 38 chronic wounds across 7 patients showed that

a single surgical application of ZEVASKYN was associated with long-term improvement at treated sites over a median follow-up of 6.9 years;

many years of work, it is great to see this FDA approval of ZEVASKYN. The EB patients deserve all that we can do for them," said

M. Peter Marinkovich, M.D., associate professor of dermatology and co-principal investigator of the VIITAL study.

on the strength of our data across clinical trials, we are confident in ZEVASKYN's ability to deliver long-term results after a

single treatment application," said Madhav Vasanthavada, Ph.D., M.B.A., Chief Commercial Officer of Abeona. "We are committed

to working closely with both commercial and government payers on outcome-based agreements that stand behind the promise of ZEVASKYN for

patients, and expedite access."

both clinical studies, ZEVASKYN was well-tolerated with no treatment-related serious adverse events observed to date.

L. Bruckner, MD, Co-Director of the EB Clinic at Children's Hospital of Colorado and Professor of Dermatology, University of Colorado

School of Medicine, said, "The FDA approval of ZEVASKYN marks a monumental step forward for individuals living with RDEB and their

families, offering a much-needed, long-lasting treatment option for this devastating condition and providing hope for improved quality

of life for these patients."

Paller, M.D., pediatric dermatologist and clinical researcher, said, "Grafting gene-corrected skin onto chronically open wounds

of patients with recessive dystrophic epidermolysis bullosa promises the potential to provide long-term healing of wounds, reduction

in pain and reduced risk of infection. This therapeutic option will nicely complement recently approved topical products."

Seshadri added, "We are grateful to the patients, their families, and caregivers for their support of ZEVASKYN. We express our

gratitude to debra of America for their unwavering support throughout the development journey, in particular, for their interactions

with the FDA in support of ZEVASKYN and on behalf of the EB community that have helped make today's regulatory approval possible.

We are also thankful for the clinical study investigators, study site personnel, and the entire Abeona team for their collective commitment

and determination through the development process, and contribution to this milestone achievement. We look forward to providing the RDEB

community access to now-approved ZEVASKYN."

the underlying cause of RDEB

mutations in both copies of the COL7A1 gene that expresses Type VII collagen, people with RDEB have extremely fragile skin characterized

by extensive blistering and severe wounds that often cover more than 30 percent of a patient's body surface, and in some cases

up to 80 percent. RDEB wounds cause debilitating pain and systemic complications impacting the length and quality of life. These wounds

are difficult to heal, can remain open for years, and many that do close tend to reopen.

consists of a patient's own skin cells (keratinocytes) that have been genetically modified, to produce functional Type VII collagen.

ZEVASKYN sheets are surgically applied to the patient's wounded areas. In a single application of ZEVASKYN, up to 12 credit card-sized

sheets can be joined together to cover large areas or applied to multiple distinct wounds, allowing for signficant coverage of affected

Kopelan, M.A., Executive Director of debra of America, the only national advocacy organization that provides all-inclusive care to the

epidermolysis bullosa community, and father to Rafi, a teenager with RDEB, said, "I, and the team at debra of America, are very

excited about the FDA's approval of ZEVASKYN. Given that this therapeutic product addresses even the largest, most difficult, and

problematic chronic wounds, we believe that the application of ZEVASKYN can significantly increase the quality of life of patients. Furthermore,

I believe ZEVASKYN has the potential to transform the day-to-day standard of care for patients who suffer with these large chronic nonhealing

wounds that cause significant pain and stress not only for the patient, but also for their caregivers. We are honored to have formed

such a close relationship with Abeona over the years and look forward to deepening our partnership by helping ensure there is broad access

for the patient population to ZEVASKYN, which I know is their ultimate goal."

Hund, M.B.A., Chief Executive Officer of EB Research Partnership (EBRP), the largest global organization dedicated to funding research

to treat and cure epidermolysis bullosa (EB), said, "The mission of EBRP is to advance commercially sustainable research aimed

at treating and ultimately curing EB. We are honored to partner with the entire Abeona team and commend their leadership, determination,

and passion to deliver much needed innovative solutions for individuals and their families living with EB. They continue to share our

values and commitment to accelerate treatments to the EB community as quickly as possible. Abeona's development and advancement

of ZEVASKYN delivers a landmark moment for the global EB community, and their leadership in gene therapy holds so much promise to innovate

the therapeutic landscape for not only EB, but many other rare diseases and conditions. EBRP is looking forward to collaborating with

Abeona to continue to support the EB community."

availability in the third quarter of 2025

is expected to be available beginning in the third quarter of 2025 through ZEVASKYN Qualified Treatment Centers (QTCs). The QTCs are

well-recognized epidermolysis bullosa treatment centers with cell and gene therapy experience, situated across the U.S. to ensure patients

nationwide have access to this important treatment.

comprehensive patient support program, Abeona Assist , offers personalized support, including helping patients understand their

insurance benefits and financial assistance options, and providing travel and logistical assistance for eligible patients. For more information

on how to access ZEVASKYN, patients, caregivers, and providers may call Abeona Assist at 1-855-ABEONA-1 (1-855-223-6621) or email MyNavigator@AbeonaAssist.com.

are grateful to the dedicated scientists whose work over the past decade made the development of ZEVASKYN possible," said Marissa

Perman, MD, Section Chief of Dermatology and Director of the Epidermolysis Bullosa Multidisciplinary Clinic at Children's Hospital

of Philadelphia and paid consultant to Abeona. "Having a new, uniquely differentiated, gene therapy for our patients with

RDEB is a significant milestone in helping these special patients live fuller, pain-free and itch-free lives with less wounds. As a physician

caring for patients with RDEB, I look forward to the opportunity to see this treatment in our practice."

Teng, MD, PhD, professor in dermatology with multiple hospital affiliations, said, "I'm thrilled to celebrate a groundbreaking

advancement in therapeutic development for recessive dystrophic EB, a condition that has long needed an innovative solution. ZEVASKYN

offers additional hope for patients and families affected by this painful and devastating skin disorder. This milestone is a testament

to the dedication of scientists, researchers and medical professionals who have worked tirelessly to bring cutting edge treatments to

those in need. It represents a scientific triumph, a profound step toward improving quality of life for individuals affected. We look

forward to seeing the impact that this therapy will have on so many lives."

connection with the FDA approval, Abeona received a Rare Pediatric Disease Priority Review Voucher (PRV). The Company plans to monetize

Therapeutics will host a conference call and webcast with accompanying slides and patient videos today, Tuesday, April 29, 2025, at 8:00

a.m. ET. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 320280 five minutes prior

to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed at https://investors.abeonatherapeutics.com/events.