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).
At the recent ASGCT meeting, researchers from Exegenesis Bio Inc. presented preclinical data for EXG-102-031, a novel recombinant adeno-associated virus (rAAV)-gene therapy being developed for the treatment of neovascular age-related macular degeneration (AMD), also called wet AMD (wAMD).
MicroRNA-184 (miR-184) mutations have been previously tied to inherited anterior segment dysgenesis, potentially causing cataracts and keratoconus. To elucidate the involvement of miR-184 in ocular disorders, Chinese researchers have developed a miR-184 knockout zebrafish model. Investigators used CRISPR-Cas9 technology to delete two miR-184 paralogs (dre-mir-184-1 and dre-mir-184-2) in zebrafish, which are highly conserved between human and fly.
Azura Ophthalmics Ltd. has divulged codrugs consisting of an immunomodulator moiety and a keratolytic agent reported to be useful for the treatment of eye and dermatological disorders.
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