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BioWorld - Sunday, July 19, 2026

Rare disease

Home » Topics » Disease categories and therapies » Rare disease
  • Blue dollar sign on white background
    July 14, 2026
    By Tamra Sami

    Drug Farm bags $55M series D to advance AI-developed ALPK1 drug

    Drug Farm Biotechnology Co. Ltd. closed a $55 million series D round to advance its AI-developed alpha-protein kinase 1 (ALPK1) inhibitor, DF-003, in a phase III trial for ROSAH syndrome, a rare genetic disease that can lead to blindness.
  • Eya and dna illustration
    July 7, 2026
    By Marian (YoonJee) Chu

    Opus aligns with FDA on rare eye disease gene therapy trial

    Opus Genetics Inc. has secured U.S. FDA alignment on an eight-patient phase III trial of its lead gene therapy, OPGx-LCA5, for an ultra-rare form of inherited childhood blindness.
  • Digital ma screen
    July 7, 2026
    By Karen Carey

    Endocrinology becomes Vertex’s fifth pillar with $10B Crinetics buy

    Representing the fourth largest biopharma M&A announced in 2026, Vertex Pharmaceuticals Inc. offered $85 per share in cash, or about $10 billion, to buy endocrine specialist Crinetics Pharmaceuticals Inc., adding the acromegaly drug Palsonify and a phase III asset, atumelnant, for congenital adrenocorticotropic hyperplasia (CAH).
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