Gene therapy

Regeneron’s DB-OTO gene therapy showed significant hearing improvements in children with OTOF-related hearing loss in the CHORD trial. 11 of 12 participants demonstrated clinically meaningful hearing gains, with 3 achieving normal hearing. The trial met its primary endpoint, and improvements were durable up to 72 weeks. DB-OTO was generally well-tolerated. Regeneron plans to file for U.S. regulatory approval later in 2025, pending FDA discussions. DB-OTO holds Orphan Drug and other FDA designations.

Sensorion reported positive H1 2025 results, highlighting progress in gene therapies. The Audiogene Phase 1/2 trial for SENS-501 (OTOF-GT) showed a positive safety profile and early hearing improvement signs. Enrollment in SENS-401’s Phase 2a Cisplatin-Induced Ototoxicity study is complete, with topline data expected in H2 2025. GJB2-GT is on track for a Q1 2026 CTA filing. The company’s cash position of €57.1m funds operations into Q3 2026. R&D expenses increased slightly, reflecting intensified clinical trial activities. Net loss was -€16.0 million.

REGENXBIO (Nasdaq: RGNX) will participate in several investor conferences in September 2025, including events hosted by Wells Fargo, Morgan Stanley, Baird, and H.C. Wainwright. The company will engage in one-on-one meetings and fireside chats to discuss its strategic vision, clinical progress, and the potential of its AAV gene therapies for rare and retinal diseases, including RGX-202 for Duchenne muscular dystrophy and surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy. Webcasts will be available on REGENXBIO’s website.

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.

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.

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.