KB407
Cystic Fibrosis (CF)
Phase 1Active
Key Facts
About Krystal Biotech
Krystal Biotech's mission is to develop and deliver redosable gene therapies for severe, life-threatening rare diseases. Its landmark achievement is the 2023 FDA approval of VYJUVEK®, the first and only redosable gene therapy, which validates its proprietary HSV-1 vector platform. The company's strategy is to commercialize VYJUVEK globally while rapidly advancing a deep internal pipeline across multiple therapeutic areas, supported by in-house GMP manufacturing capabilities. This integrated approach positions Krystal as a leader in next-generation, repeat-administration genetic medicines.
View full company profileTherapeutic Areas
Other Cystic Fibrosis (CF) Drugs
| Drug | Company | Phase |
|---|---|---|
| CFTR Program (via Boehringer Ingelheim) | AlveoGene | Phase 1 |
| QCT Standardization in Cystic Fibrosis | Thirona | Development/Validation |