Core Insights - ZEVASKYN™ (prademagene zamikeracel) is the first FDA-approved autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][2][9] - The pivotal Phase 3 VIITAL study demonstrated significant wound healing and pain reduction after a single treatment, with 81% of treated wounds showing at least 50% healing compared to 16% in control wounds [5][13] - The publication of the VIITAL study results in The Lancet marks a significant milestone for Abeona Therapeutics as it prepares for the U.S. launch of ZEVASKYN [3][4] Company Overview - Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with ZEVASKYN being a key product in its portfolio [15] - The company operates a fully integrated cGMP cell and gene therapy manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of ZEVASKYN [15] Study Details - The VIITAL study was an intra-patient randomized, open-label, controlled Phase 3 trial involving 11 patients with RDEB, assessing 43 pairs of large chronic wounds [3][4] - The study's co-primary endpoints of wound healing and pain reduction were successfully met, with significant statistical differences observed [5][13] Clinical Results - At week 24, 65% of treated wounds achieved 75% or more healing compared to 7% of control wounds, indicating a substantial improvement in treatment efficacy [13] - Mean change in wound pain was -3.1 in treated wounds versus -0.9 in control wounds, demonstrating a significant reduction in pain levels [13] Safety Profile - No serious ZEVASKYN-related adverse events were reported, consistent with previous clinical experiences, and no cases of squamous cell carcinoma were observed in treated wounds [13][14]

Core Points - ZEVASKYN is the first and only cell-based gene therapy approved for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [2][8] - Ann & Robert H. Lurie Children's Hospital of Chicago is the first Qualified Treatment Center (QTC) for ZEVASKYN, with treatments expected to begin in Q3 2025 [1][3] - Abeona Therapeutics has launched the Abeona Assist™ program to provide personalized support for patients and families throughout the treatment journey [1][5] Company Overview - Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with ZEVASKYN being a key product in its portfolio [15] - The company operates a cGMP manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of ZEVASKYN [15] Treatment Details - ZEVASKYN incorporates the COL7A1 gene into a patient's skin cells to produce functional type VII collagen, which is essential for skin integrity [8] - The therapy has shown clinically meaningful results in wound healing and pain reduction with a single application [8] Hospital and Research Center - Lurie Children's Hospital has been a center of excellence for genetic skin diseases for over 30 years and is recognized for its expertise in treating epidermolysis bullosa [3][12] - The hospital has been providing FDA-approved gene therapies since 2019 and is expanding its offerings, with ZEVASKYN being the tenth gene therapy available [4]

Abeona Therapeutics Inc.'s ABEO stock traded higher on Wednesday.The Food and Drug Administration (FDA) on Tuesday approved Abeona’s Zevaskyn (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB).Zevaskyn is the only FDA-approved product to treat RDEB wounds with a single application.Zevaskyn consists of a patient's skin cells (ke ...