Skyline Therapeutics (Shanghai) Co. Ltd. has received FDA clearance for its IND application for a phase I/IIa trial of SKG-0106, a one-time intravitreally delivered recombinant adeno-associated virus (AAV) gene therapy for the treatment of neovascular age-related macular degeneration (wet AMD). A global phase I trial will be initiated soon.
Intergalactic Therapeutics Inc. has announced promising preclinical data with IG-002, its lead program addressing all forms of ABCA4-related retinopathies. Intergalactic’s nonviral gene therapy platform is designed to overcome limitations of standard adeno-associated virus (AAV) gene therapy technologies, including vector capacity limitations and vector-related safety concerns.
Benitec Biopharma Inc. has received FDA clearance of its IND application for BB-301, its silence and replace gene therapy for the treatment of oculopharyngeal muscular dystrophy (OPMD)-related dysphagia.
Barring truly major surprises, exagamglogene autotemcel (Exa-cel, Vertex Pharmaceuticals Inc.) is on track to become the first approved CRISPR-based gene editing therapy. It is partly in expectation of Exa-cel’s approval that the European Hematology Association (EHA) and the European Society for Bone Marrow Transplantation hosted a session on “transplantation versus gene therapy in sickle cell disease.”
Currently, there are no treatments to reverse or prevent genetic hearing loss, which affects 1 in 500 newborns. Several gene replacement and overexpression preclinical studies targeting genetic hearing loss have shown success, as the inner ear can be accessed safely by local injection. However, all these gene therapy studies have been performed in neonatal animals, except one in the Otof gene; therefore, the suitability of the approach in the fully mature adult inner ear remains to be elucidated.
Researchers from Seelos Therapeutics Inc. presented the discovery and preclinical evaluation of a gene therapy candidate, SLS-009, for the treatment of Huntington’s disease (HD).
With overuse of opioids – the standard of care for many chronic pain cases – becoming something of an epidemic in the U.S., the availability of an alternative, non-opioid analgesic is a big draw. Established in 2021, Adolore Biotherapeutics Inc. is one company that could provide the answer, with its locally and long-acting gene therapies potentially providing a breakthrough that “knocks everybody’s socks off.”
Bietti’s crystalline corneoretinal dystrophy (BCD) is an autosomal recessive inherited disease caused by mutations in the cytochrome P450 (CYP) family 4 subfamily V member 2 (CYP4V2) gene, which encodes a polyunsaturated fatty acid (PUFA) hydroxylase dominantly expressed in retinal pigment epithelium (RPE) cells.
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