Abeona Therapeutics receives FDA orphan drug designation for ABO-202 gene therapy program in infantile Batten disease. Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, announced Monday that the FDA has granted orphan drug designation to its ABO-202 program (AAV-CLN1), an adeno-associated viral (AAV)-based gene…
Abeona’s Sanfilippo Gene Therapy Data Produces Encouraging Results
Abeona Therapeutics announces new phase 1/2 safety and biopotency data of investigational gene therapy for Sanfilippo syndrome. Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, reported preliminary 30-day safety and biopotency signals from the first patient dosed in the company’s ongoing Phase 1/2 trial for…
Abeona Gene Therapy Gains FDA Regenerative Medicine Designation
Abeona Therapeutics Inc., a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, announced that the U.S. Food and Drug Administration (FDA) has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to EB-101, the company`s gene-corrected autologous cell therapy product for patients with recessive dystrophic epidermolysis bullosa…