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.
Vigeneron GmbH has received IND approval from the FDA for VG-801, a novel mRNA trans-splicing gene therapy to treat Stargardt disease and other retinal dystrophies associated with mutations in the ABCA4 gene. IND clearance enables initiation of a phase I/II trial, with a clinical trial application (CTA) submission to the EMA also planned in the coming months.
Lento Bio Inc. has synthesized cysteine derivatives reported to be useful for the treatment of presbyopia. An exemplified compound (Cpd in row 4 in tbl1 pg 20, claim 18) decreased in lens stiffness in C57BL/6 mice (at 20 mM; as eye drops).
Splicebio S.L. has gained IND clearance from the FDA for its lead program SB-007, a protein splicing gene therapy to address the root genetic cause of Stargardt disease.
Researchers from the U.K. have analyzed whole-genome sequencing data from 7,276 cases and 236,741 controls in the UK Biobank to perform gene-level and a variant-level exome-wide association study analysis to identify variants related to retinal detachment.
WDR45 is located on the X chromosome. Its pathogenic variants are associated with various neurodegenerative disorders that are predominantly reported in females and present a wide range of clinical phenotypes, from early-onset developmental delay to neurodegeneration and multiple epileptic syndromes.
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