The first treatment for macular telangiectasia type 2 is set to enter the market following U.S. FDA approval of NT-501 (revakinagene taroretcel) from Neurotech Pharmaceuticals Inc., a privately held company that has been quietly advancing its encapsulated cell therapy platform for more than two decades.
Avirmax Biopharma Inc. has begun IND-enabling studies of ABI-201, a gene therapy for dry age-related macular degeneration (AMD). ABI-201 is an AAV vector that delivers three genes to correct the dysregulation of complement activation, to protect retinal pigment epithelia and photoreceptors, as well as to block retinal neovascularization.
A recent patent filing from Verily Life Sciences LLC seeks protection for the development of an ophthalmic drug delivery device with sensors and a camera which allow the delivery of a specific or controlled dose of a therapeutic agent in vapor form to a patient’s eye.
Inherited retinal dystrophies (IRDs), including retinitis pigmentosa and Stargardt disease, are a group of rare degenerative disorders of the retina with clinical and genetic heterogeneity. In a recent publication, researchers from the Institute of Ocular Microsurgery applied clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 and transcription activator-like effector nucleases (TALEN) gene-editing tools to precisely correct induced pluripotent stem cell (hiPSC) lines derived from IRD patients.
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.
Heterocyclic insulin-like growth factor 1 receptor (IGF-1R; CD221) inhibitors potentially useful for the treatment of thyroid-associated ophthalmopathy (Graves’ ophthalmopathy) have been disclosed in a Horizon Therapeutics Ltd. patent.
Olix Pharmaceuticals Inc. walked the talk in realizing a new $630 million licensing deal with Eli Lilly and Co. for its cardiovascular and metabolic disease asset, OLX-702A (OLX-75016), rallying stock by 30% after it had largely recovered from a terminated deal with France’s Théa Open Innovation last year.
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