Researchers at Sanofi SA have developed a promising gene therapy approach targeting the microtubule-associated protein tau (MAPT) for the treatment of Alzheimer’s disease.
Remedium Bio Inc. has entered into a multitarget research and development collaboration with Eli Lilly & Co. to advance gene therapies for type 2 diabetes and obesity using Remedium’s Prometheus dose-adjustable gene therapy platform.
Myotonic dystrophy type 1 (DM1) is a rare, progressive genetic disease that causes severe muscle weakness and other debilitating symptoms, such as compromised respiration and cardiac conduction abnormalities. No disease-modifying therapy exists for DM1, so care focuses on managing symptoms like arrhythmia, myotonia, hypertension, cataracts, respiratory issues and sleep disorders.
Axovia Therapeutics Inc. has been awarded a new $1.0 million grant by the nonprofit organization A Race Against Blindness to support the clinical development of AXV-101, an investigational gene therapy aimed at combating childhood blindness due to retinitis pigmentosa caused by Bardet-Biedl syndrome 1 (BBS1).
Adeno-associated virus (AAV)-based gene therapy is considered a promising strategy to treat hearing loss. However, its clinical application is limited by the genetic heterogeneity of hereditary hearing loss, requiring gene-specific analysis and approach optimization for broader treatment applications.
Editas Medicine Inc. has nominated a lead in vivo development candidate, EDIT-401, a potential one-time therapy designed to significantly reduce LDL cholesterol (LDL-C) levels. The in vivo gene editing medicine is designed to treat hyperlipidemia by directly editing the LDLR gene to increase LDLR protein expression and reduce LDL-C levels.
With a few tweaks to the protocol, Rocket Pharmaceuticals Inc.’s phase II trial testing RP-A501 in Danon disease is expected to resume following the lifting of the clinical hold, issued by the U.S. FDA in May in response to the death of a patient in the pivotal gene therapy study.
Aussie gene therapy company Mirugen Pty Ltd. has emerged from stealth mode from the Center for Eye Research Australia in Melbourne, toting a AU$4.5 million (US$2.9 million) seed round that will see it optimize its lead candidate to treat retinitis pigmentosa.
Opus Genetics Inc. has obtained IND clearance from the U.S. FDA application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related inherited retinal disease (IRD).
The U.S. FDA has given a swift and full approval to Precigen Inc.’s gene therapy, Papzimeos (zopapogene imadenovec), for treating adults with recurrent respiratory papillomatosis (RRP), a rare and chronic disease characterized by benign tumors in the respiratory tract.
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-and-Raymond-Wong-(R).webp?height=488&t=1755204408&width=650 "Mirugen co-founders Keith Martin (L) and Raymond Wong (R)")
