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.
Mutations in the CEP290 (NPHP6) gene cause severe cilia formation defects and a wide range of ciliopathies, ranging from non-syndromic Leber congenital amaurosis (LCA10) to Meckel syndrome (MKS). Researchers from Medetia SAS and affiliated organizations recently presented the discovery and preclinical evaluation of a novel prostaglandin-E2 receptor agonist, MDT-110, as a potential treatment of NPHP6/CEP290-associated phenotypes.
It has been previously demonstrated that IL-17A plays a proinflammatory role in autoimmune diseases, and it has also been reported that IL-17A may take part in the occurrence and development of neurodegenerative disorders. Due to its association with both immunomodulation and inflammation, in a recent study, researchers from Shanghai Jiao Tong University aimed to investigate the role of IL-17A in the pathological process of glaucoma neuropathy.
Charcot-Marie-Tooth disease (CMT) is a group of neuropathies characterized by sensory and motor dysfunction that progress with aging. It is considered that about 60% of the axonal forms of the disease, such as CMT2, remain genetically undiagnosed.
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.
Mutations in the RPGRIP1 gene are associated with rare retinal dystrophies and most commonly with Leber congenital amaurosis (LCA) type 6, which is characterized by vision loss, among other symptoms.
Researchers from Skyline Therapeutics (Shanghai) Co. Ltd. presented preclinical data for the new recombinant AAV vector therapeutic SKG-0106, being developed for the treatment of neovascular (wet) age-related macular degeneration (AMD).
Pharmabcine Inc. has received IND approval from the Korean Ministry of Food and Drug Safety (MFDS) to initiate a phase I trial of PMC-403 in Korea in subjects with neovascular (wet) age-related macular degeneration (AMD).
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