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FDA grants uniQure’s Huntington’s gene therapy Priority Review

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The FDA has accepted uniQure’s application for its experimental Huntington’s disease gene therapy, AMT-130 (ifezuntirgene inilparvovec), and granted it Priority Review. The FDA’s target action date is May 1, 2027. Priority Review speeds the review timetable; it does not guarantee approval. The application is being considered under the Accelerated Approval pathway. The FDA said it is tentatively planning an advisory committee meeting, but has not set a date. If approved, AMT-130 could become the first disease-modifying treatment for Huntington’s disease. The therapy is designed to reduce production of the huntingtin protein by delivering a gene-silencing treatment directly into the brain. UniQure says it has aligned with the FDA on key elements of a planned confirmatory study of about 200 patients. The company plans to begin screening by the end of 2026; the study’s primary measure is total functional capacity at 36 months. The milestone follows a difficult regulatory period f...

Pfizer beats Q3 estimates and raises 2026 outlook

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Novartis ends BioArctic neuroscience deal worth up to $802 million

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Midstream Health raises $20 million to improve hospital finances

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CinRx and Graviton form CinRock to pursue ROCK2 therapies for heart and lung conditions

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FDA partial hold on Bright Minds’ BMB-101 trial sends shares sharply lower

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Karyopharm’s proposed Ayrmid deal is part of a Chapter 11 restructuring

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Merck to sell U.S. CDMO business to Lonza

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InventisBio reports Phase III psoriasis win for nomelcitinib

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Eos reports preliminary PTC-2105 results in aged rats

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Tiziana reports mixed-positive Phase 2a results for intranasal foralumab

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FDA grants accelerated approval to Inovio’s VOCERYQ for rare HPV-related airway disease

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Rare kidney disease: closing the gap between discovery and care

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