StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
By exploiting the genetic variation in cancer cells, an already approved cancer drug demonstrated enhanced effects against cancer cells in specific patient groups.
FDA accepts BLA resubmission of pz-cel in recessive dystrophic epidermolysis bullosa and sets PDUFA target action date of April 29, 2025 Company makes significant progress toward potential commerciali ...