Abeona Therapeutics Inc. is a clinical‐stage biopharmaceutical company focused on the development of innovative gene and cell therapies for rare genetic diseases and cancers. The company’s research pipeline includes adeno‐associated viral (AAV) gene therapies targeting Sanfilippo syndrome types A and B, as well as programs in epidermolysis bullosa and solid tumors. Abeona’s proprietary EB‐101 therapy for the treatment of recessive dystrophic epidermolysis bullosa uses patient‐derived skin cells genetically modified to express functional collagen VII, while its lead AAV programs, ABO‐101 and ABO‐102, are designed to deliver corrective genes to the central nervous system via a single intravenous or intracerebral administration.
Since its formation in 2014 as a spin‐out from UCLA’s Gene Therapy Program, Abeona has pursued a strategy of leveraging cutting‐edge vector engineering and manufacturing capabilities to advance first‐in‐class therapies. The company collaborates with academic institutions and contract development and manufacturing organizations to optimize vector design and scale up production. Abeona’s efforts in process development and quality control aim to meet regulatory standards for global commercialization, with its clinical manufacturing facilities located in Cleveland, Ohio, and additional development operations in Europe.
Abeona serves a worldwide patient population affected by rare and debilitating disorders. Its clinical trials have been conducted in the United States, Europe and Australia, reflecting the company’s commitment to broad geographic reach and regulatory engagement across multiple regions. Strategic partnerships and licensing agreements help Abeona extend its global footprint, ensuring access to specialized expertise and facilitating potential commercialization in diverse markets.
Leadership at Abeona is spearheaded by President and Chief Executive Officer John Shaw, who brings extensive experience in biotechnology and gene therapy development. Under his guidance, the company has expanded its pipeline and strengthened its corporate infrastructure. Abeona’s board and scientific advisory committees comprise experts in neurology, dermatology and hematology, supporting the company’s mission to deliver transformative therapies for patients with unmet medical needs.
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