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rare diseases

  • Clinical Development,Drug Development,Healthcare leadership,Movers & Shakers,Neurosciences,Oncology,Rare Diseases

    Allucent adds strategy leaders to expand therapeutic expertise

    Allucent has appointed new therapeutic strategy leaders across key disease [...]

    April 9, 2026
  • Cardiovascular diseases,Clinical Trials,Rare Diseases,Regulatory Affairs,Research & Development

    Cereno Scientific reports long-term safety data from CS1 pulmonary arterial hypertension program

    Cereno Scientific has reported initial long-term safety findings from a [...]

    April 7, 2026
  • Clinical Development,Clinical Trials,Rare Diseases,Research & Development,Technology and platforms

    PRISM ALS initiative develops patient-derived stem cell models to improve ALS/MND drug discovery

    A new global initiative aims to address long-standing limitations in [...]

    April 7, 2026
  • Cell & Gene Therapy,Hematology,Manufacturing,Partnerships & Funding,Rare Diseases,Research & Development,Technology and platforms

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

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

    March 31, 2026
  • Clinical Development,Drug Development,FDA,Genetic Diseases,Orphan drugs,Pharmaceuticals and therapeutics,Rare Diseases

    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 [...]

    March 31, 2026
  • Cardiovascular diseases,Clinical Trials,Drug approval,Rare Diseases

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

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

    March 31, 2026
  • Biologics & Biosimilars,Biomanufacturing,Biotech,Clinical Development,Clinical Trials,Drug approval,Drug Development,FDA,Rare Diseases,Research & Development

    Hansa Biopharma’s BLA for imlifidase accepted by FDA

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

    February 19, 2026
  • Biotech,Clinical Development,Clinical Trials,Pharmaceuticals and therapeutics,Rare Diseases,Research & Development

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

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

    February 18, 2026
  • Biotech,Cell & Gene Therapy,Clinical Development,Movers & Shakers,Pharmaceuticals and therapeutics,Rare Diseases

    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 [...]

    February 11, 2026
  • Biologics & Biosimilars,Cell & Gene Therapy,Clinical Development,Drug Development,Rare Diseases,Research & Development

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

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

    January 29, 2026
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