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Welcome to Discover Pharma! Today is : April 27, 2026

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Genetic Diseases

  • Biotech,Funding,Genetic Diseases,Mergers & Acquisitions,Research & Development,RNA Therapeutics

    Circio raises NOK 250M in oversubscribed private placement to advance circRNA gene therapy platform

    Circio has raised NOK 250 million in a significantly oversubscribed [...]

    April 8, 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
  • Cell & Gene Therapy,Clinical Development,Genetic Diseases,Research & Development

    Scribe Therapeutics secures milestone $1.5BN collaboration with Eli Lilly for in vivo CRISPR program

    Scribe Therapeutics has achieved a second success milestone in its [...]

    February 18, 2026
  • Cell & Gene Therapy,Genetic Diseases,Research & Development,RNA Therapeutics

    SynGenSys launches Liver.SET library for liver-specific gene expression in in vivo therapies

    SynGenSys has launched Liver.SET, a library of liver-specific synthetic promoters [...]

    February 5, 2026
  • Clinical Development,Drug approval,Genetic Diseases,Rare Diseases,Regulatory Affairs

    Pegunigalsidase alfa every four weeks dosing backed by CHMP for adult Fabry patients

    The European Medicines Agency’s Committee for Medicinal Product for Human [...]

    February 2, 2026
  • Biotech,Genetic Diseases,Healthcare leadership,Movers & Shakers,Pharmaceuticals and therapeutics,Precision medicine

    Enhanced Genomics appoints Katerina Leftheris to board of directors

    Enhanced Genomics has appointed Katerina Leftheris to its board of [...]

    January 14, 2026
  • Biotech,Cell & Gene Therapy,Genetic Diseases,Genomics and sequencing,Rare Diseases,Research & Development,Technology and platforms

    PacBio HiFi adopted to study sudden unexplained death in childhood and advance precision therapies for rare diseases

    PacBio is supporting two research initiatives that use its HiFi [...]

    January 13, 2026
  • Biologics & Biosimilars,Clinical Development,Genetic Diseases,Rare Diseases,Research & Development,RNA Therapeutics

    Tevard Biosciences shows full-length protein rescue in Duchenne muscular dystrophy and dilated cardiomyopathy models

    Tevard Biosciences has presented new preclinical data demonstrating the potential [...]

    October 2, 2025

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