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Rare Diseases

  • Clinical Development,Clinical Trials,Pharmaceuticals and therapeutics,Precision medicine,Rare Diseases

    A potential first-in-class treatment for children with achondroplasia is now in clinical testing

    Tyra Biosciences has dosed the first child in BEACH301, its [...]

    August 22, 2025
  • Clinical Trials,Rare Diseases,Regulatory Affairs

    FDA approves BRINSUPRI as first treatment for non-cystic fibrosis bronchiectasis

    Insmed Incorporated, a global biopharmaceutical company focused on serious diseases, [...]

    August 14, 2025
  • Artificial Intelligence,Drug Development,Partnerships & Funding,Rare Diseases

    Evogene and Tel Aviv University join forces to tackle metabolite aggregation in metabolic diseases

    Evogene Ltd. has announced a scientific collaboration with Professor Ehud [...]

    August 12, 2025
  • Drug Development,Partnerships & Funding,Rare Diseases,Research & Development

    DEBRA Research and Queen Mary University of London partner to advance drug development for rare skin disorder

    DEBRA Research, a non-profit focused on Epidermolysis bullosa (EB), has [...]

    August 7, 2025
  • Artificial Intelligence,Digital Health,Rare Diseases,Regulatory Affairs

    EU court upholds annulment of titanium dioxide carcinogen classification in major regulatory ruling

    The Court of Justice of the European Union (CJEU) has [...]

    August 8, 2025
  • CDMOs & Manufacturing,Cell & Gene Therapy,Clinical Development,Global health,Partnerships & Funding,Rare Diseases,Technology and platforms

    Sidra Medicine awarded $100K G-Rex grant for CAR-T manufacturing platform

    ScaleReady has awarded a $100,000 G-Rex grant to Sidra Medicine, [...]

    August 6, 2025
  • CDMOs & Manufacturing,Cell & Gene Therapy,Rare Diseases,Research & Development

    University of Minnesota investigators awarded $665,000 in ScaleReady G-Rex Grant funding

    Three research teams to advance manufacturing and development of cell [...]

    August 1, 2025
  • Clinical Development,Precision medicine,Rare Diseases,Real world evidence,Research & Development,Technology and platforms

    Nautilus and the Allen Institute partner to map tau proteoforms in Alzheimer’s disease research

    Nautilus Biotechnology has announced a new collaboration with the Allen [...]

    August 4, 2025
  • Biologics & Biosimilars,Clinical Development,Drug Development,Rare Diseases,Regulatory Affairs

    FDA grants fast track to Nektar’s rezpegaldesleukin for severe alopecia areata

    Nektar Therapeutics has received FDA Fast Track designation for its [...]

    July 29, 2025
  • Drug Development,Patient Centricity,Rare Diseases,Regulatory Affairs

    MHRA approves Biogen’s tofersen for ultra-rare genetic form of motor neurone disease

    The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has [...]

    July 28, 2025
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