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rare diseases

  • Advanced Therapies,Biotech & Pharma Business,Clinical & Regulatory,Pharmaceuticals and therapeutics,Therapeutic Areas

    Hans Schambye appointed as CEO of BOOST Pharma as BT‑101 advances toward Phase 3

    BOOST Pharma has appointed Hans Schambye as its new chief [...]

    May 5, 2026
  • Advanced Therapies,Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

    iXCells Biotechnologies partners with Rosebud Biosciences to advance organoid models for rare diseases

    iXCells Biotechnologies and Rosebud Biosciences have announced a partnership to [...]

    May 5, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Therapeutic Areas

    Veristat names Kim McLean Boericke as CEO to drive next phase of clinical research growtheristat names Kim McLean Boericke as CEO to drive next phase of clinical research growth

    Veristat, a global clinical research organization (CRO) and consultancy specializing [...]

    May 5, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

    Minovia secures two new US patents for mitochondrial augmentation therapy platform

    Minovia Therapeutics has been granted two new US patents covering [...]

    May 5, 2026
  • Advanced Therapies,Clinical & Regulatory,Therapeutic Areas

    EMA grants orphan drug designation to Kedrion treatment for congenital aceruloplasminemia

    Kedrion has received orphan drug designation from the European Medicines [...]

    May 5, 2026
  • Drug Discovery & Development,Therapeutic Areas

    Successful treatment for ALS could deliver billions in global value

    New research commissioned by Challenge Works and conducted by the [...]

    May 5, 2026
  • Advanced Therapies,Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Opinion & Events,Therapeutic Areas

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

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

    May 5, 2026
  • Biotech & Pharma Business,Drug Discovery & Development,Genomics and sequencing,Therapeutic Areas

    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 [...]

    May 5, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Opinion & Events,Therapeutic Areas

    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 [...]

    May 5, 2026
  • Advanced Therapies,Biotech & Pharma Business,Manufacturing & Supply Chain,Opinion & Events,Pharmaceuticals and therapeutics,Therapeutic Areas

    Er-Kim to commercialize Nulibry and Zokinvy across Central and Eastern Europe, Mediterranean and Eurasia

    Er-Kim has signed an agreement with Sentynl Therapeutics to distribute [...]

    May 5, 2026
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