Dermatology specialist Leo Pharma A/S is moving into gene therapy with the $50 million acquisition of Replay Holdings LLC, a seed-stage company that is developing high capacity herpes simplex virus vectors to treat rare inherited diseases.
The EMA’s Committee for Medicinal products for Human Use (CHMP) is standing by its opinion on Leqembi (lecanemab) after the European .mission pushed back against a recommendation in November 2024 that the Alzheimer’s disease drug be approved
About a year after the U.S. FDA cleared Krystal Biotech Inc.’s topical gene therapy, Vyjuvek (beremagene geperpavec), as the first drug for recessive or dominant dystrophic epidermolysis bullosa, competitor Abeona Therapeutics Inc. disclosed a complete response letter (CRL) from the FDA related to its cell-based graft therapy, prademagene zamikeracel.
After the initial approvals in monogenic inherited diseases, the scope of gene therapy is widening, with new delivery routes, novel vectors, cell-specific targeting and products aiming to treat chronic disorders, all making headway in 2023.
Right on schedule the U.S. FDA gave its blessing for Krystal Biotech Inc.’s topical gene therapy, Vyjuvek (beremagene geperpavec, or B-VEC), an orphan drug, to become the first approved treatment for the rare skin condition dystrophic epidermolysis bullosa (DEB).
