Opus Genetics Inc. has secured U.S. FDA alignment on an eight-patient phase III trial of its lead gene therapy, OPGx-LCA5, for an ultra-rare form of inherited childhood blindness.
With the U.S. FDA go-ahead granted June 26 for Viridian Therapeutics Inc.’s IGF-1R antagonist Lumvoa (veligrotug-vvze, or “veli”) as a new thyroid eye disease (TED) therapy – due to launch immediately, the company said – Wall Street will be watching near-term payer dynamics. The drug is set to take on similarly targeted Tepezza (teprotumumab-trbw), owned by Amgen Inc. and approved in January 2020 to treat TED.
Viridian Therapeutics Inc.’s U.S. FDA clearance of Lumvoa (veligrotug-vvze) to treat thyroid eye disease (TED) includes labeling for chronic as well as active forms, and fewer infusions – plus fast, durable effects – should give the IGF-1R antagonist leverage in competing with similarly targeted TED drug Tepezza (teprotumumab-trbw), owned by Amgen Inc. and approved in January 2020.
The oversubscribed $330 million series B round secured by Ollin Biosciences Inc. marks more than another large venture round in ophthalmology. It also highlights an emerging biotech financing model in which Chinese pharma companies discover and clinically validate promising drugs before handing global development to well-capitalized U.S. startups backed by blue-chip venture investors.
Ethyreal Bio Inc. has come out of stealth mode in order to disclose preclinical data for ETHY-001, its monoclonal antibody targeting thyroid stimulating hormone receptor, at the Endocrine Society’s 2026 annual meeting on June 15.
Fresh off a deal with Travere Therapeutics Inc. for civorebrutinib worth more than $1.14 billion, Everest Medicines Ltd. went on a shopping spree, striking three deals in six days to expand its pipeline and geographic reach across Asia Pacific.
Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional Crispr-Cas9 editing.
The much-awaited phase III readout from Oculis Holding AG’s OCS-01 in diabetic macular edema (DME) fell short of expectations, delaying the possibility of a topical eye drop option for DME patients and prompting the company to shuffle its pipeline priorities, with a focus on late-stage programs targeting optic neuropathies and dry eye disease.
Daewoong Pharmaceutical Co. Ltd. announced after South Korean market hours May 21 that it gained successive rights to Turn Biotechnologies Inc.’s mRNA-based cellular rejuvenation platform from Hanall Biopharma Co. Ltd.
There are real world demonstrations of how autonomous artificial intelligence agents are poised to disrupt biomedical research, according to two papers published May 19 in Nature. Each describes an AI system that assists across the piece, from generating hypotheses to designing experiments, analyzing the data and refining hypotheses in the light of new data.