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rare diseases

  • Advanced Therapies,Biotech & Pharma Business,Drug Discovery & Development,Manufacturing & Supply Chain,Therapeutic Areas

    UMass Chan, Caring Cross and Trenchant BioSystems form sickle cell gene therapy manufacturing alliance

    UMass Chan Medical School, Caring Cross and Trenchant BioSystems have [...]

    May 5, 2026
  • Advanced Therapies,Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Therapeutic Areas

    BridgeBio submits FDA NDA for BBP-418 in limb-girdle muscular dystrophy type 2I/R9

    BridgeBio Pharma has submitted a New Drug Application (NDA) to [...]

    May 5, 2026
  • Clinical & Regulatory,Therapeutic Areas

    Chiesi gains CHMP backing for lomitapide in children with rare cholesterol disorder

    Chiesi Global Rare Diseases has received a positive CHMP opinion [...]

    May 5, 2026
  • Advanced Therapies,Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Manufacturing & Supply Chain,Therapeutic Areas

    Hansa Biopharma’s BLA for imlifidase accepted by FDA

    Hansa Biopharma’s Biologics License Application (BLA) for imlifidase has been [...]

    May 5, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Therapeutic Areas

    Polaryx therapeutics selects contract research organization for SOTERIA phase 2 basket trial

    Polaryx Therapeutics, Inc has selected a leading contract research organization [...]

    May 5, 2026
  • Advanced Therapies,Biotech & Pharma Business,Clinical & Regulatory,Pharmaceuticals and therapeutics,Therapeutic Areas

    Hans Schambye appointed as CEO of BOOST Pharma as BT‑101 advances toward Phase 3

    BOOST Pharma has appointed Hans Schambye as its new chief [...]

    May 5, 2026
  • Advanced Therapies,Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

    iXCells Biotechnologies partners with Rosebud Biosciences to advance organoid models for rare diseases

    iXCells Biotechnologies and Rosebud Biosciences have announced a partnership to [...]

    May 5, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Therapeutic Areas

    Veristat names Kim McLean Boericke as CEO to drive next phase of clinical research growtheristat names Kim McLean Boericke as CEO to drive next phase of clinical research growth

    Veristat, a global clinical research organization (CRO) and consultancy specializing [...]

    May 5, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

    Minovia secures two new US patents for mitochondrial augmentation therapy platform

    Minovia Therapeutics has been granted two new US patents covering [...]

    May 5, 2026
  • Advanced Therapies,Clinical & Regulatory,Therapeutic Areas

    EMA grants orphan drug designation to Kedrion treatment for congenital aceruloplasminemia

    Kedrion has received orphan drug designation from the European Medicines [...]

    May 5, 2026
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