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rare diseases

  • Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    Polaryx advances Phase 2 trial of PLX-200 in four rare diseases

    Polaryx Therapeutics is preparing to start its Phase 2 SOTERIA [...]

    August 26, 2026
  • Advanced Therapies,Drug Discovery & Development,Manufacturing & Supply Chain,Therapeutic Areas

    Andelyn and Queen’s University partner on AAV9 gene therapy for GM2 gangliosidoses

    Andelyn Biosciences and Queen’s University have partnered to develop and [...]

    August 19, 2026
  • Advanced Therapies,Clinical & Regulatory,Genomics and sequencing,Pharmaceuticals and therapeutics,Research & Development

    Atsena receives EMA orphan designation for two inherited retinal disease gene therapies

    Atsena Therapeutics has received EMA orphan designation for two gene [...]

    July 24, 2026
  • Advanced Therapies,Biotech & Pharma Business,Manufacturing & Supply Chain

    VIVEbiotech expands in vivo lentiviral vector portfolio to 15 programmes

    VIVEbiotech has expanded its in vivo lentiviral vector portfolio to [...]

    June 15, 2026
  • Biotech & Pharma Business,Opinion & Events,Therapeutic Areas

    Chiesi launches second Find For Rare research grants programme at ERA 2026

    Chiesi Global Rare Diseases has launched the second edition of [...]

    June 9, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

    Allucent adds strategy leaders to expand therapeutic expertise

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

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

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

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

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

    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 [...]

    May 5, 2026
  • 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
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