Abeona Therapeutics Announces Two Presentations Related to Its RDEB Clinical Program at the Society for Pediatric Dermatology 45th Annual Meeting - Seite 2

The Full Burden of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

M. Peter Marinkovich, M.D., Bullous Disease Clinic Director, Stanford University Medical Center, and Investigator in the VIITAL^TM study, presented findings from a literature review of 65 studies that provide new insights on the disease burden from the perspective of patients with RDEB and their families. Key observations of the clinical, humanistic and economic burden of RDEB include:

• Large, chronic wounds comprise a major clinical burden of RDEB and are correlated with pain.
• Many patients experience anxiety and depression.
• Parents of children with RDEB reported negative effects on their relationship, choosing to not have more children.
• 50% of U.S. families characterized the economic impact of managing RDEB as “high” or “severe.”

Abeona’s posters from the SPD 45^th Annual Meeting are available on the “News/Events” page under the “Investors & Media” section of Abeona’s website at www.abeonatherapeutics.com.

About Recessive Dystrophic Epidermolysis Bullosa
Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

About EB-101
EB-101 is an autologous, gene-corrected cell therapy currently being investigated in the pivotal Phase 3 VIITAL^TM study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITAL^TM study is a multi-center, randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 30 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient’s own skin cells (keratinocytes and their progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITAL^TM study at its fully-functional gene and cell therapy manufacturing facility in Cleveland, OH. In a Phase 1/2a clinical trial, EB-101 provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. More information on the clinical trials of EB-101 can be found at https://www.abeonatherapeutics.com/clinical-trials/rdeb and ClinicalTrials.gov (Identifier: NCT04227106).