Gene therapy
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Sarepta Therapeutics Pauses ELEVIDYS Shipments in the U.S.
Sarepta Therapeutics is voluntarily pausing ELEVIDYS shipments in the U.S. to facilitate information exchange with the FDA and finalize safety labeling. This gene therapy treats Duchenne muscular dystrophy by delivering a micro-dystrophin gene. The pause will take effect July 22, 2025. The company emphasizes patient safety and maintaining a productive relationship with the FDA. ELEVIDYS is approved for Duchenne patients aged 4+ with confirmed DMD gene mutations.
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Genflow Announces Grant Progress
Genflow Biosciences (GENFF) secured a €4 million non-dilutive grant from Belgium’s Wallonia Region to advance GF-1002, its gene therapy candidate for Metabolic Associated Steatohepatitis (MASH). The grant covers a three-year development program aligned with Genflow’s clinical roadmap. While initial disbursement awaits administrative procedures, the GF-1002 program continues without interruption. Genflow highlights that all project-related expenses incurred in 2025 will be eligible under the grant’s terms.
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以下是几个可能的英文标题： * Taysha Gene Therapies Announces Pricing for Public Offering of Common Stock and Pre-Funded Warrants * Taysha Gene Therapies Sets Pricing for Public Offering of Common Stock and Pre-Funded Warrants * Taysha Gene Therapies Reveals Pricing of Public Offering for Common Stock and Pre-Funded Warrants
Taysha Gene Therapies (TSHA) announced a public offering to raise approximately $200 million. The offering includes common stock and pre-funded warrants, priced at $2.75 and $2.749 per share respectively. This capital will advance Taysha’s CNS-focused gene therapy programs, targeting diseases like Rett syndrome with its AAV-based therapies. The deal is expected to close around May 30, 2025.
