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rare disease

  • Biotech & Pharma Business,Drug Discovery & Development,Therapeutic Areas

    Elixirgen signs worldwide option deal with Nippon Shinyaku for Duchenne therapy

    Elixirgen Therapeutics has signed an option agreement with Nippon Shinyaku [...]

    June 29, 2026
  • Clinical & Regulatory,Pharmaceuticals and therapeutics,Therapeutic Areas

    Cereno reports positive 12-month CS1 safety data ahead of Phase 2b PAH trial

    Cereno Scientific has reported encouraging 12-month safety and clinical observations [...]

    June 29, 2026
  • Advanced Therapies,Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development

    Chiesi and Arbor win EU orphan status for PH1 gene editing therapy

    The European Commission has granted orphan drug designation to Chiesi [...]

    June 28, 2026
  • Clinical & Regulatory,Pharmaceuticals and therapeutics,Therapeutic Areas

    Grifols launches Fesilty in US for congenital fibrinogen deficiency

    Grifols has launched Fesilty in the USA, making the fibrinogen [...]

    June 16, 2026
  • Clinical & Regulatory,Drug Delivery & Formulation,Pharmaceuticals and therapeutics,Therapeutic Areas

    FDA grants orphan drug designation to Tris Pharma’s TRN-257 for idiopathic hypersomnia

    FDA has granted orphan drug designation to Tris Pharma’s investigational [...]

    June 11, 2026
  • Central Nervous System,Clinical & Regulatory,Drug Discovery & Development

    CONNECTA starts Phase 2a trial of CTH120 for fragile X syndrome

    CONNECTA Therapeutics has started a Phase 2a clinical trial of [...]

    June 10, 2026
  • Advanced Therapies,Drug Discovery & Development,Pharmaceuticals and therapeutics,Therapeutic Areas

    Skyhawk reports encouraging 12-month Huntington’s disease data for SKY-0515

    Skyhawk Therapeutics has reported encouraging 12-month results from its ongoing [...]

    June 6, 2026
  • Advanced Therapies,Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

    Intellia reports first positive Phase 3 in vivo CRISPR data in hereditary angioedema

    Intellia Therapeutics has reported positive Phase 3 results for lonvoguran [...]

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

    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 [...]

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

    Pharvaris raises $175 million to advance oral therapy for hereditary angioedema

    Pharvaris has secured approximately $175 million through a public offering [...]

    May 5, 2026
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