Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, develops gene and cell therapies for life-threatening rare genetic diseases. Its lead programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa; ABO-102, an adeno-associated virus-based gene therapy for Sanfilippo syndrome type A; and ABO-101, an AAV-based gene therapy for Sanfilippo syndrome type B. The company also develops ABO-201 to treat CLN3 disease; ABO-401 for the treatment of cystic fibrosis; and ABO-5OX for the treatment of genetic eye disorders. In addition, it is developing AAV-based gene therapy through its AIM vector platform programs.
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