ABEO Profile
Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, specializes in developing innovative gene and cell therapies for life-threatening rare genetic diseases. The company's lead program, EB-101, is an autologous, gene-corrected cell therapy currently in Phase III clinical trials for recessive dystrophic epidermolysis bullosa (RDEB), a severe skin disorder. This therapy aims to provide long-term benefits by correcting the underlying genetic defect in patients' skin cells.
In addition to EB-101, Abeona is advancing several other promising therapies. ABO-102 is an adeno-associated virus (AAV)-based gene therapy designed to treat Sanfilippo syndrome type A, a rare and fatal neurodegenerative disease in children. The company is also developing ABO-201 for CLN3 disease, a disorder leading to progressive vision loss, motor decline, and early death, and ABO-401 for cystic fibrosis, aiming to address the genetic mutations causing this chronic respiratory disease. Furthermore, the ABO-50X program focuses on genetic eye disorders, expanding the company's therapeutic reach.
Abeona leverages its proprietary AIM vector platform to develop AAV-based gene therapies, enhancing the precision and effectiveness of its treatments. This platform enables targeted delivery of therapeutic genes to specific tissues, improving treatment outcomes for patients with rare genetic conditions. The company's research and development efforts are grounded in a deep understanding of genetic diseases and a commitment to innovative scientific approaches.
Formerly known as PlasmaTech Biopharmaceuticals, Inc., Abeona Therapeutics Inc. adopted its current name in June 2015 to better reflect its focus on advanced therapies. Incorporated in 1974, the company is headquartered in New York, New York, and continues to build on its legacy of pioneering treatments for rare genetic disorders. Abeona's robust pipeline and strategic partnerships underscore its dedication to transforming the lives of patients with unmet medical needs.
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