AAVantgarde Bio SrL raised €61 million (US$65 million) in series A funding to take forward two novel approaches to gene therapy that aim to overcome the packaging limits of adeno-associated virus (AAV) vectors. The company plans to move its lead program, in retinitis pigmentosa associated with Usher syndrome type 1b, into the clinic later this year. A second program, in Stargardt disease, is a couple of years behind it.
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.
Coave Therapeutics aims to move its lead gene therapy program, CTx-PDE6b, for a form of retinitis pigmentosa (RP) into a pivotal trial in 2025 on the back of a phase I/II study, which uncovered a preliminary efficacy signal in one patient subgroup.
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.
On the strength of upside in best corrected visual acuity (BCVA) in patients with geographic atrophy (GA), Annexon Inc. officials will meet with the U.S. FDA to talk about what’s next for ANX-007, after the C1q inhibitor missed the phase II trial’s primary endpoint: mean rate of change, or slope, in the GA lesion area compared to sham.
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).
RSS
