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.
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.
Childhood-onset neurodegeneration with cerebellar atrophy (CONDCA) is an autosomal recessive disorder that causes progressive motor and cognitive impairment in children. The disease arises as a result of inactivating mutations in cytosolic carboxypeptidase 1 (CCP1), leading to excessive polyglutamylation of tubulin in the brain. Researchers at Shimane University have shown in a mouse model that delivering a truncated form of CCP1 into the brain can substantially mitigate Purkinje cell degeneration and improve motor function.
CLN3 disease, also known as juvenile neuronal ceroid lipofuscinosis, is a rare neurodegenerative disorder affecting neuronopathic lysosomal storage that severely impacts the central nervous system while also inducing notable peripheral neuromuscular symptoms. Researchers from Washington University School of Medicine have demonstrated the potential of gene therapy for CLN3 disease.
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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)")
