This Week in Rare Disease Breakthroughs š§¬ | Gene Therapies, Drug Approvals & Global Policy Shifts
From a $115M boost in PMM2-CDG therapy to the FDAās bold new pathway for ultra-rare disease approvals, this weekās roundup is packed with high-impact developments you wonāt want to miss. Whether you’re tracking gene therapies, regulatory milestones, or strategic biotech moves, we’ve got you covered.
š§ Hereās whatās inside this edition:
š¼ Spruce Biosciences pivots to rare neurology with its TA-ERT acquisition and eyes BLA by 2026.
š¤ Norgine strengthens its rare disease portfolio with the addition of six specialty drugs via the Theravia deal.
š§Ŗ Sarepta makes major progress across four LGMD gene therapy programs with a BLA filing expected in late 2025.
šļø Atsenaās ATSN-201 now holds four FDA designations as it advances gene therapy for XLRS.
š° Glycomine secures $115M in funding to launch a pivotal Phase 2b trial in 2025 for its first-in-class PMM2-CDG treatment.
š§¬ FDA Commissioner Makary introduces a āmechanism-drivenā approval pathway for ultra-rare diseasesāno RCTs required, with strong post-market oversight.
š§ Excellamol earns orphan drug status for its precision glioma therapy using sustained delivery tech.
š§« Tempest receives FDA orphan designation for TPST-1495, targeting FAP with immunotherapy backed by the NCI.
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