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.
Gemvax & Kael Co. Ltd. has identified peptides reported to be useful for the treatment of age-related macular degeneration (AMD), cerebral amyloid angiopathy and Alzheimer’s disease.
Corneal neovascularization (CNV) occurs when abnormal blood vessels grow into the cornea in response to ischemic or hypoxic conditions caused by infections, inflammation or chemical injuries.
Pulsesight Therapeutics SAS has submitted a clinical trial authorization (CTA) to the French authority ANSM seeking to conduct a first-in-human phase I trial of PST-611 for dry age-related macular degeneration (AMD)/geographic atrophy (GA).
The company is advancing a pipeline of therapeutics, including programs acquired from Denali Therapeutics Inc., focused on treating neurological, cardiometabolic and ophthalmic diseases.
Proper survival and regeneration of retinal ganglion cells (RGCs), which constitute the optic nerve, is crucial for managing traumatic or ischemic optic nerve injury, optic neuropathy, or optic pathway gliomas. Researchers from Wenzhou Medical University described the synthesis of novel spirocyclic chalcone derivatives designed to combat optic nerve damage.
Scientists at Admare Bioinnovations Inc., Kingston General Hospital (KGH) and Queen’s University have described cytochrome P450 26B1 (CYP26B1) inhibitors reported to be useful for the treatment of acne, dry eye, meibomian glands dysfunction, fibrodysplasia ossificans progressive, heterotopic ossification (HO) and multiple osteochondroma.
Sigma-2 receptor modulation using exogenous ligands reduces cellular damage associated with central nervous system degeneration in age-related pathologies such as Alzheimer’s disease, Parkinson’s disease or dry age-related macular degeneration. Researchers from Cognition Therapeutics Inc. described the neuroprotective effect of CT-2074, a sigma-2 receptor in preclinical models of open-angle glaucoma.
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