Abeona Announces FDA Grants RMAT Designation to ABO-102 Gene Therapy in MPS IIIA

Abeona Therapeutics (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, announced today that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ABO-102, the Company’s AAV-mediated gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA), a rare autosomal-recessive lysosomal storage disease.

As quoted in the press release:

“We are encouraged to have received the first gene therapy RMAT designation in MPS IIIA and look forward to further collaborating with the FDA to determine next steps in the development pathway for ABO-102,” said Carsten Thiel, Ph.D., CEO of Abeona Therapeutics. “This action further reinforces the clinical significance in the data observed in the ongoing Phase 1/2 trial and the high unmet need for effective treatment options for patients suffering from MPS IIIA.”

Established under the 21st Century Cures Act, RMAT designation is an expedited program for the advancement and approval of regenerative medicine products. A regenerative medicine is eligible for the designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such a disease or condition. RMAT allows companies developing regenerative medicine and gene therapies to work more closely and frequently with the FDA and grants all of the benefits of Breakthrough Therapy Designation.

Click here to read the full press release.