PTC518
Huntington's disease
Phase 2Active
Key Facts
About PTC Therapeutics
PTC Therapeutics is a patient-centric, commercial-stage biopharma focused on discovering, developing, and commercializing clinically differentiated medicines for rare diseases. The company has achieved multiple regulatory firsts, including the first global treatment for Duchenne muscular dystrophy (Translarna), the first brain-infused gene therapy (Upstaza), and the first small molecule splicing modifier for spinal muscular atrophy (Evrysdi). Its strategy leverages two proprietary scientific platforms—Splicing and Inflammation & Ferroptosis—to build a diversified pipeline, supported by a global commercial infrastructure aimed at sustainable growth and expanding patient access.
View full company profileTherapeutic Areas
Other Huntington's disease Drugs
| Drug | Company | Phase |
|---|---|---|
| NUZ-001 | Neurizon Therapeutics | Preclinical |
| Huntington's Disease Program | Rumi Scientific | Pre-clinical |
| SOM3355 | SOM Biotech | Phase 3 |
| LETI-101 | ElevateBio | Preclinical |
| Somatic Expansion Inhibitor Program | LoQus23 Therapeutics | Preclinical |
| Preclinical Program | Excision BioTherapeutics | Preclinical |
| NA-841 | Biomed | Phase 2A |
| T3D-959 (Future) | T3D Therapeutics | Pre-clinical / Research |
| HD mAb Therapy | HD Immune | Pre-clinical |
| TT-P34 | Teitur Trophics | Pre-clinical |
| INT41 | Vybion | Pre-clinical |
| Huntington's Program | reMYND | Pre-clinical |