PTC518
Huntington's disease
Phase 2Clinical
Key Facts
About PTC Therapeutics
PTC Therapeutics is a patient-centric, publicly traded biopharma leader dedicated to translating groundbreaking science into life-changing treatments for rare diseases. Its key achievements include commercializing a series of 'firsts' in rare disease treatment and building a diversified pipeline targeting neurological and metabolic disorders. The company's strategic direction is fueled by its deep expertise in two core scientific platforms—splicing modifiers and inflammation/ferroptosis inhibitors—and a robust global commercial infrastructure to support its mission of delivering transformative medicines worldwide.
View full company profileTherapeutic Areas
Other Huntington's disease Drugs
| Drug | Company | Phase |
|---|---|---|
| SRP-1005 (ARO-HTT) | Arrowhead Pharmaceuticals | Phase 1 |
| Undisclosed HD Program(s) | WaVe Life Sciences | Preclinical |
| AMT-130 | uniQure | Phase 1/2 |
| Undisclosed Program | Sangamo Therapeutics | Preclinical |
| Pepinemab (VX15/2503) | Vaccinex | Phase 2 |