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Gene Therapy

  • Central Nervous System,Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development

    CHMP recommends approval of NEZGLYAL for rare neurodegenerative disease

    The EMA’s CHMP has recommended approval of NEZGLYAL for boys [...]

    July 24, 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,Clinical & Regulatory,Drug Delivery & Formulation,Research & Development

    EpilepsyGTx partners with ClearPoint Neuro to support Phase 1 gene therapy trial

    EpilepsyGTx has partnered with ClearPoint Neuro to support the targeted [...]

    July 14, 2026
  • Advanced Therapies,Manufacturing & Supply Chain,Medical devices,Research & Development

    Bio-Rad launches Vericheck ddPCR kits for cell and gene therapy quality control

    Bio-Rad Laboratories has launched a portfolio of Vericheck ddPCR kits [...]

    July 8, 2026
  • Advanced Therapies,Genomics and sequencing,Research & Development,Therapeutic Areas

    Genespire reports preclinical gene therapy data for methylmalonic acidemia

    Genespire and researchers at the San Raffaele Telethon Institute for [...]

    July 8, 2026
  • Advanced Therapies,Biotech & Pharma Business,Clinical & Regulatory

    Cyllene Therapeutics raises €33M to advance EG110A into late-stage development

    Cyllene Therapeutics has raised €33 million in a Series C [...]

    July 7, 2026
  • Advanced Therapies,Clinical & Regulatory,Genomics and sequencing,Therapeutic Areas

    Atsena advances gene therapy ATSN-201 into Phase 3 trial for inherited blindness

    Atsena Therapeutics has dosed the first patient in the Phase [...]

    June 22, 2026
  • Advanced Therapies

    Houdini Bio develops DNA design platform targeting gene therapy silencing

    Houdini Bio has launched a machine learning-guided DNA sequence design [...]

    June 19, 2026
  • Advanced Therapies,Drug Discovery & Development

    Circio and GenAssist partner on next-generation gene therapies for muscle diseases

    Circio and GenAssist have announced a research collaboration aimed at [...]

    June 6, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Therapeutic Areas

    Life Biosciences raises $80M to advance gene therapy for optic neuropathies

    Life Biosciences has raised $80m in a Series D financing [...]

    May 5, 2026
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