Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company. The Company is engaged in developing cell and gene therapies for life-threatening diseases. The Companyâs lead clinical program is for prademagene zamikeracel (pz-cel), its investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa (RDEB). The Companyâs fully integrated cell and gene therapy Good Manufacturing Practice (cGMP) manufacturing facility served as the manufacturing site for pz-cel used in its Phase III VIITAL trial and is capable of supporting commercial production of pz-cel upon FDA approval. The Companyâs development portfolio also features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. The Companyâs novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases.
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