KB104
Netherton Syndrome
PreclinicalActive
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 Netherton Syndrome Drugs
| Drug | Company | Phase |
|---|---|---|
| ATR-12 | Azitra | Phase 1 |
| QRX003 (via Quoin Pharmaceuticals) | Skinvisible Pharmaceuticals | Clinical Trials |
| Pipeline Program | MatriSys Bioscience | Preclinical |
| SXR1096 | Sixera Pharma | Phase 1 |
| BCX17725 | BioCryst Pharmaceuticals | Pre-Clinical/Phase 1 |
| QRX003 | Quoin Pharmaceuticals | Phase 2/3 |