All articles tagged with #krystal biotech
Krystal Biotech announced positive interim results from its Phase 1 trial of KB407, a gene therapy for cystic fibrosis, confirming successful lung delivery and expression of wild-type CFTR protein across diverse patients, paving the way for a larger, repeat-dosing study expected to start in 2026.
The FDA has approved Vyjuvek, a redosable gene therapy made by Krystal Biotech, for patients with dystrophic epidermolysis bullosa, a genetic disease that causes painful blisters and persistent wounds. The topical treatment delivers a healthy copy of the gene that encodes the protein type VII collagen to the targeted skin cells, enabling healing to occur. Patients with the condition live covered in bandages to try to stop new blisters from forming and to dress their wounds.
The FDA has approved Krystal Biotech's gene therapy, Vyjuvek, for treating patients with dystrophic epidermolysis bullosa (DEB), a rare genetic disease that causes the skin to be as fragile as a butterfly's wings. The therapy is the first of its kind and offers a long-awaited solution for families afflicted by the condition, for which there are no approved treatments in the US.
The US FDA has approved Krystal Biotech's Vyjuvek, a topical gene therapy for patients with dystrophic epidermolysis bullosa, a rare genetic skin disorder. The therapy is expected to be available in the US in Q3 2023 and is Krystal's first to be approved in the country. The FDA approval is supported by data from early-to-mid-stage and late-stage studies, which showed Vyjuvek completely healed wounds in about 65% of the participants.