LONDON, UK – Spur Therapeutics today released new data from the GALILEO-1 and GALILEO-2 trials of avigbagene parvec (FLT201), its investigational gene therapy for Gaucher disease type 1 (GD1), which highlight encouraging improvements in skeletal disease, a major and persistent source of morbidity in patients despite long-term standard-of-care treatment. These data are...

FORT LEE, N.J. — Kedrion Biopharma is raising awareness for Plasminogen Deficiency Type 1 (PLGD‑1), a rare genetic disorder, through a series of global virtual and in‑person events held in recognition of Plasminogen Deficiency Awareness Day on May 4. PLGD‑1 is an ultra‑rare condition affecting approximately 1.6 per 1 million people...

NEWTON, Mass. — Satellite Biosciences, Inc. (Satellite Bio), a biotechnology company developing off-the-shelf liver therapies designed to restore liver function in patients with severe liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SB-101 for the treatment of urea...