UX111
Sanfilippo Syndrome Type A (MPS IIIA)
Phase 1/2Active
Key Facts
About Abeona Therapeutics
Abeona Therapeutics has successfully transitioned to a commercial-stage company with the launch of ZEVASKYN®, while advancing a pipeline of novel gene therapies for rare diseases with high unmet need. Its strategy is built on a proprietary AIM™ vector platform designed for enhanced delivery and efficacy, and is vertically integrated with internal manufacturing capabilities. The company is focused on inherited retinal diseases and neurological disorders, with key partnered programs in Sanfilippo Syndrome and Rett Syndrome. Abeona's mission is to deliver transformative, one-time treatments, aiming to make cures the new standard of care.
View full company profileTherapeutic Areas
Other Sanfilippo Syndrome Type A (MPS IIIA) Drugs
| Drug | Company | Phase |
|---|---|---|
| JR-441 | JCR Pharmaceuticals | Phase 1/2 |