Cell replacement therapy is currently being investigated to restore the retinal pigment epithelium (RPE) layer and treat vision decline due to dry age-related macular degeneration (AMD). The Neural Stem Cell Institute and collaborating institutions developed an approach to RPE replacement using an adult RPE stem cell source derived from the RPE layer of donated cadaver eyes.

Directed evolution has become a central pillar in gene therapy. This engineering strategy enables the generation of more efficient variants of genetic editors and delivery vectors. Molecular diversification methods are increasingly sophisticated and are now accelerated by machine learning and AI tools, as showcased at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) held in Boston this week.

Researchers from Beacon Therapeutics Holdings Ltd. reported the development of BTX-001, an intravitreal gene therapy designed to target the complement pathway through delivery of a C5 inhibitor.

Curacle Co. Ltd. inked a potential $1.07 billion deal with U.S.-based “newco” Memento Medicines on May 10, granting the latter exclusive global rights to develop and commercialize preclinical TIE2/VEGF-directed bispecific antibody MT-103 for retinal disorders.

Curacle Co. Ltd. inked a potential $1.07 billion deal with U.S.-based “newco” Memento Medicines on May 10, granting the latter exclusive global rights to develop and commercialize preclinical TIE2/VEGF-directed bispecific antibody MT-103 for retinal disorders.

Researchers at Opus Genetics Inc. reported the efficacy of OPGx-BEST1, an AVV-based gene therapy developed to deliver a functional BEST1 transgene to retinal pigment epithelium (RPE) cells to re-establish normal BEST1 expression and activity.

Curacle Co. Ltd. inked a potential $1.07 billion deal with U.S.-based “newco” Memento Medicines on May 10, granting the latter exclusive global rights to develop and commercialize preclinical TIE2/VEGF-directed bispecific antibody MT-103 for retinal disorders.

In its first substantial M&A transaction since 2021, Bayer AG is buying Perfuse Therapeutics Inc., a firm that has operated quietly, though it reported positive midstage data last year for PER-001, an endothelin antagonist, in glaucoma and diabetic retinopathy.

The classic origin story for a biotech startup is that of a scientist who nurtures his work out of a university and to commercial success. For Link Biologics Ltd. and its TSG6-based pipeline, the story is the other way around; it began with now-CEO Reuben Dawkins meeting University of Manchester scientists Tony Day and Caroline Milner while he was on the lookout for “great science that needs help to make it to patients.” The three are now co-founders of Link, which spun out of the University of Manchester in 2021 and has four programs in three indications, all based on TSG-6 biology.

Top-line data from the Reveal-2 phase III trial of elegrobart to treat chronic thyroid eye disease (TED) drove Viridian Therapeutics Inc.’s stock up by 33.6%, positioning the company for a BLA filing in the first quarter of 2027. The trial met both primary endpoints at week 24: proptosis responder rate, as measured by exophthalmometry, required for a filing with the U.S. FDA; and the overall responder rate, required for an EMA filing. Doses every four weeks or every eight weeks achieved 50% and 54% PRR, respectively, compared to 15% for placebo. The doses also achieved 47% and 54% ORR vs. 15% for placebo. ORR included those with both proptosis and Clinical Activity Score response, defined as no worsening from baseline in the study eye, without deterioration in the fellow eye.