Gene therapy specialist Meiragtx Holdings plc is heading for its first marketing approval following the successful treatment of 11 children with Leber amaurosis, a severe form of congenital retinal dystrophy that rendered them blind at birth. The 11 children, aged between 1 and 4 years old, all gained visual acuity following a single delivery of a correct version of the AIPL1 (aryl-hydrocarbon interacting protein-like1) gene, which codes for a photoreceptor protein in the cones and rods.
Perceive Pharma Inc., a spin out from Perceive Biotherapeutics Inc., has closed a $15 million series A funding round to help it advance first-in-class therapies to prevent vision loss in glaucoma, and additional undisclosed disease areas.
Fudan University has disclosed phosphatidylcholine:ceramide cholinephosphotransferase 2 (SGMS2; SMS2) inhibitors reported to be useful for the treatment of cancer, atherosclerosis, dry eye, pulmonary edema, fatty liver, obesity, type 2 diabetes and Alzheimer’s disease.