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.
Researchers from Huazhong University of Science and Technology have described the potential use of RBM47 as a therapeutic target for thyroid-associated ophthalmopathy, an autoimmune disorder characterized by proptosis, lid swelling, diplopia and compressive optic neuropathy.
Inmed Pharmaceuticals Inc. has selected an intravitreal (IVT) formulation for INM-089 as a drug candidate to be utilized in the company’s ongoing development program targeting the treatment of dry age-related macular degeneration (AMD).
Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon 6, c.768G>T. Due to its high prevalence, c.768G>T is an interesting therapeutic target for STGD1. Researchers from Radboud University developed a new antisense oligonucleotide (AON) therapy, designed to rescue the splicing defect caused by this variant.
Stargardt disease, the most common form of juvenile macular degeneration, results from biallelic mutations in the ABCA4 gene. In a recently published study, researchers at the Institute of Molecular and Clinical Ophthalmology Basel and collaborators proposed a novel dual AAV vector system to deliver a split-intein adenine base editor for precise correction of the most common Stargardt mutation, c.5882G>A (p.Gly1961Glu).
Sitala Bio Ltd. has prepared new indole derivatives acting as complement factor B (CFB) inhibitors. They are thus reported to be useful for the treatment of age-related macular degeneration, schizophrenia, atypical hemolytic uremic syndrome, membranous nephropathy, myasthenia gravis, paroxysmal nocturnal hemoglobinuria, diabetic retinopathy and rheumatoid arthritis, among others.
Tikun Therapeutics Inc. has obtained U.S. orphan drug and rare pediatric disease designations for its programs in familial dysautonomia, namely its rAAV2-U1a-hELP1 gene replacement therapy for the treatment of optic neuropathy in familial dysautonomia and BPN-36964 for systemic treatment of familial dysautonomia.
Inherited retinal diseases (IRDs) are a group of monogenic disorders caused by mutations in more than 280 genes, and are characterized by progressive degeneration of photoreceptor cells in the retina.
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