GTX-102
Angelman Syndrome
Phase 1/2Active
Key Facts
About Ultragenyx Pharmaceutical
Ultragenyx is a mission-driven, commercial-stage biotech dedicated to pioneering novel treatments for serious rare and ultra-rare genetic diseases. Founded in 2010, the company has successfully transitioned to a revenue-generating entity with three approved products and has built one of the largest and most diverse clinical pipelines in the rare disease sector. Its strategy employs a flexible, multi-platform approach—including gene therapy, mRNA, and enzyme replacement—to target the root cause of monogenic disorders. Led by CEO Emil D. Kakkis, M.D., Ph.D., Ultragenyx operates globally with a deep commitment to underserved patient communities.
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| Drug | Company | Phase |
|---|---|---|
| HLX-0553 | Healx | Preclinical |
| Undisclosed Pipeline Programs | AS2 Bio | Pre-clinical |
| Rugonersen (OHB-724) | Oak Hill Bio | Phase 3 |
| NNZ-2591 | Neuren Pharmaceuticals | Phase 2 |