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Welcome to Discover Pharma! Today is : January 19, 2026

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Gene Therapy

  • Biotech,Cell & Gene Therapy,Clinical Development,Clinical Trials,Opthalmology,Rare Diseases,Research & Development

    AAVantgarde completes enrollment in LUCE-1 Phase 1 and Phase 2 trial of gene therapy AAVB-081 for Usher syndrome type 1B

    AAVantgarde Bio has completed enrollment in LUCE-1, a first-in-human Phase [...]

    January 16, 2026
  • Biomanufacturing,Cell & Gene Therapy,Clinical Development,Rare Diseases,Regulatory Affairs

    Imlifidase enables gene therapy for Crigler–Najjar patients with AAV immunity

    Genethon and Hansa Biopharma report GNT-018-IDES trial data showing imlifidase [...]

    October 12, 2025
  • Cell & Gene Therapy,Clinical Development,Research & Development,Technology and platforms

    Circio unveils data showing 40-fold increase in AAV gene delivery efficiency with circVec technology

    Circio Holding has presented new in vivo data showing its [...]

    October 8, 2025
  • Biotech,Cell & Gene Therapy,Clinical studies,Clinical Trials,Drug Development,Opthalmology

    Complement Therapeutics receives FDA IND clearance for CTx001 to enter Opti-GAIN trial in geographic atrophy

    Complement Therapeutics GmbH (CTx) has received clearance from the FDA [...]

    October 8, 2025
  • Biotech,Genomics and sequencing,Partnerships & Funding,Rare Diseases,Research & Development

    Chiesi and Arbor partner on rare disease gene editing programmes targeting PH1 and other liver disorders

    Chiesi Group has entered into an exclusive global partnership with [...]

    October 7, 2025
  • Cell & Gene Therapy,Clinical Trials,Drug approval,Rare Diseases,Research & Development

    AAVantgarde secures FDA orphan drug designation and UK trial approval for Stargardt gene therapy

    AAVantgarde Bio has received two regulatory milestones for its investigational [...]

    October 2, 2025
  • 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
  • Biomanufacturing,Biotech,Cell & Gene Therapy,Clinical Development,Pharmaceuticals and therapeutics,Research & Development

    BioGrad unveils state-of-the-art Liverpool bio-manufacturing centre to drive cell and gene therapy growth in the North

    BioGrad has announced plans for a new Centre of Bio-Manufacturing [...]

    September 26, 2025
  • Cell & Gene Therapy,Central Nervous System,Clinical Trials,Neurosciences,Pharmaceuticals and therapeutics,Research & Development

    Gene therapy AMT-130 slows Huntington’s disease progression in early trial

    A gene therapy developed by uniQure appears to slow progression [...]

    September 25, 2025
  • CDMOs & Manufacturing,Cell & Gene Therapy,Pharmaceuticals and therapeutics,Supply Chain & Logistics,Technology and platforms

    Merck opens climate-neutral €150 million filter manufacturing facility in Ireland

    Merck has inaugurated its first climate-neutral manufacturing facility in Ireland, [...]

    September 23, 2025
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