Vanessa Pataia,  —

PTR-01 Shows Signs of Efficacy in RDEB Patients, Early Trial Data Reveal

Treatment with PTR-01 is well-tolerated and safe, and increases the levels of collagen type VII (C7) in the skin of adults with recessive dystrophic epidermolysis bullosa (RDEB), early data from a Phase 1/2 trial show. The PTR-01-001 study (NCT03752905) seeks to evaluate PTR-01 treatment in…

Phase 3 Trial of Gene Therapy B-VEC for DEB Expected to Start by June

Krystal Biotech is planning to start a Phase 3 trial of B-VEC, a gene therapy candidate for the treatment of dystrophic epidermolysis bullosa (DEB), by June this year.  “We are pleased to report that our manufacturing batch…