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Rare Diseases

  • Clinical Trials,Rare Diseases,Regulatory Affairs

    Cereno Scientific submits Phase 2b trial protocol for CS1 in pulmonary arterial hypertension to FDA

    Cereno Scientific has submitted the clinical trial protocol for its [...]

    November 11, 2025
  • 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
  • Biomanufacturing,Pharmaceuticals and therapeutics,Rare Diseases,Regulatory Affairs

    Grifols’ Biotest launches Yimmugo in United States after FDA approval

    Grifols has expanded its portfolio of plasma-derived therapies in the [...]

    October 12, 2025
  • Biologics & Biosimilars,Clinical Development,European biotech,Global health,Hematology,Rare Diseases,Technology and platforms

    Terumo BCT and Hemex Health join forces to improve outcomes for sickle cell patients

    Terumo Blood and Cell Technologies (Terumo BCT), a global medical [...]

    October 10, 2025
  • Biologics & Biosimilars,Clinical Development,Clinical Trials,Drug Development,European biotech,Pharmaceuticals and therapeutics,Rare Diseases,Regulatory Affairs

    Orexa announces positive interim data for Phase 2 trial in prevention of Post-Operative Ileus

    Orexa B.V., a Dutch life sciences company focused on improving [...]

    October 9, 2025
  • Central Nervous System,Drug Development,Pharmaceuticals and therapeutics,Rare Diseases

    FDA authorises Emalex Biosciences’ Expanded Access Program for Tourette syndrome drug

    Emalex Biosciences has received FDA authorisation for an Expanded Access [...]

    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
  • Biotech,Clinical Development,Clinical Trials,Drug Development,Global health,Partnerships & Funding,Rare Diseases,Research & Development

    SeaBeLife secures €2 million to advance dual-targeted therapies for dry AMD and severe acute hepatitis

    SeaBeLife, a biotechnology company developing novel drug candidates to block [...]

    October 1, 2025
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