KB105
TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
PreclinicalActive
Key Facts
Indication
TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
Phase
Preclinical
Status
Active
Company
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.
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