Researchers at Duke University have demonstrated that retinal endothelial cells (RECs) generated from human pluripotent stem cells can restore retinal vascularization in a preclinical model of ischemic retinal injury.

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.

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.

Lu-AG-22515, also known as velaprumig, is a recombinant fusion protein that targets CD40 ligand (CD40L) and human serum albumin (HSA), thereby blocking the binding between CD40 and CD40L, a known signaling pathway involved in several autoimmune diseases. Researchers at H. Lundbeck A/S evaluated the pharmacokinetic (PK) and pharmacodynamic (PD) profile of Lu-AG-22515 in cynomolgus monkeys, as well as the impact of its inhibitory effect in the preclinical setting of thyroid eye disease (TED).

Deficiencies of the enzyme β-N-acetylhexosaminidase (Hex) cause rare, autosomal recessive, fatal, neurodegenerative lysosomal storage disorders called GM2 gangliosidoses, including Tay-Sachs disease (TSD) and Sandhoff disease. Hex enzyme is a heterodimer encoded by HEXA (α subunit) and HEXB (β subunit), whose mutations result in TSD and Sandhoff disease, respectively.

Retinitis pigmentosa (RP) is an inherited retinal dystrophy that causes loss of vision. Pathogenic variants in proteins involved in RNA splicing are the second most common cause of autosomal dominant RP, with mutations in PRPF31 being the most prevalent. Additionally, mutations in spliceosomal small nuclear RNAs (snRNAs) U4 and U6 have recently been linked to RP.

Memento Medicines Inc. has announced its launch with a $93 million series A financing and the licensing of a lead program for retinal diseases.

Opus Genetics Inc. is advancing a pipeline of gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases, with three programs expected to enter clinical testing over the next 12-18 months.