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Rare Diseases

  • Drug Development,Global health,Precision medicine,Rare Diseases,Research & Development,Technology and platforms

    First Arab human pangenome reveals millions of novel variants using long-read sequencing

    Study powered by PacBio HiFi sequencing offers new insight into [...]

    July 28, 2025
  • Clinical Trials,Global health,Oncology,Precision medicine,Rare Diseases,Regulatory Affairs,Research & Development

    CHMP backs Gilead’s twice-yearly lenacapavir for HIV prevention under accelerated EMA review

    Gilead Sciences has received a positive opinion from the Committee [...]

    July 25, 2025
  • Global health,Partnerships & Funding,Precision medicine,Rare Diseases,Research & Development,Technology and platforms

    PacBio brings isoform-level insights to 1000 Genomes Project with HiFi sequencing

    PacBio is contributing long-read transcriptomic data to the 1000 Genomes [...]

    July 25, 2025
  • Clinical Trials,Partnerships & Funding,Rare Diseases

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

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

    July 23, 2025
  • Biologics & Biosimilars,Clinical Development,Drug Delivery & Formulation,Global health,Partnerships & Funding,Rare Diseases

    New research partnership targets $1 malaria prevention shot using next-gen biologics

    Eyam Health, a Canadian biotech, teams with Medicines for Malaria [...]

    July 22, 2025
  • Rare Diseases,Research & Development

    Princess Royal opens new Insmed research facility in Cambridge focused on synthetic rescue

    Insmed has officially opened a new $10M, 17,000-square-foot research and [...]

    July 22, 2025
  • Clinical Trials,Drug Development,Rare Diseases,Research & Development

    DiaMedica reports positive phase 2 results showing DM199 reduces blood pressure in preeclampsia without placental transfer

    DiaMedica Therapeutics Inc has announced positive interim results from Part [...]

    July 18, 2025
  • Cell & Gene Therapy,Rare Diseases,Regulatory Affairs

    AAVB-039 Gains FDA clearance to advance treatment for Stargardt Disease

    AAVantgarde’s AAVB-039 gene therapy, targeting the root cause of Stargardt [...]

    July 17, 2025
  • Clinical Trials,Drug Development,Rare Diseases

    Cidara’s long-acting antiviral shows promise in Phase 2b flu prevention trial

    Cidara Therapeutics’ investigational long-acting antiviral, CD388, has achieved statistically significant [...]

    July 17, 2025
  • Cell & Gene Therapy,Clinical Trials,Rare Diseases

    Rare bone disorder: Novadip reports long-term success for pediatric patients treated under compassionate use programme

    Novadip Biosciences and Cliniques universitaires Saint-Luc have reported successful long-term [...]

    July 17, 2025
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