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Abeona Therapeutics® Congratulates Ultragenyx on U.S. FDA Approval of FAYUVI™ Gene Therapy for Sanfilippo Syndrome Type A (MPS IIIA)

  • The FDA approved FAYUVI™ (rebisufligene etisparvovec-hopf) for treating Sanfilippo syndrome type A (MPS IIIA) on September 18, 2026.
  • Ultragenyx Pharmaceutical Inc. holds global development & commercialization rights after Abeona Therapeutics Inc. out-licensed the program in May 2022.
  • Abeona developed the therapy originally designated as ABO-102, which originated from research by Drs. Haiyan Fu & Douglas McCarty.
  • FAYUVI delivers a functional copy of the SGSH gene via an AAV9 viral vector to address the root genetic cause of MPS IIIA.
  • Abeona is eligible to receive commercial milestone payments & royalties tied to future product sales under its licensing agreement.
  • MPS IIIA is a fatal pediatric neurodegenerative disease caused by SGSH enzyme deficiency leading to heparan sulfate accumulation.
  • Abeona Therapeutics also markets ZEVASKYN® for recessive dystrophic epidermolysis bullosa & is developing ABO-701 for solid tumors.