4DMT Molecular Therapeutics Inc. is looking ahead to phase III as positive data continue to roll out for gene therapy candidate 4D-150 in wet age-related macular degeneration (AMD), with the results from the phase II portion of the phase I/II Prism trial showing the single-shot treatment significantly reduced the need for chronic anti-VEGF injections. Presented over the weekend at the Angiogenesis, Exudation, and Degeneration 2024 Conference, results from the 24-week dose-expansion cohort, which comprised wet AMD patients with severe disease and high treatment burdens, showed patients receiving the high dose (3E10 vg/eye) had a 90% reduction in annualized anti-VEGF injection rates.

Kiora Pharmaceuticals Inc.’s development and commercialization deal with Théa Open Innovation (TOI), a sister company of Laboratoires Théa, for KIO-301 in degenerative retinal diseases “takes a bit of an industry-standard type of approach” in terms of structure, said CEO Brian Strem.

Vision Care Group CEO Masayo Takahashi led the world's first clinical study of a retinal cell transplant derived from induced pluripotent stem cells (iPS cells) in 2014 when she led the Laboratory for Retinal Regeneration at Japan’s Riken Center for Biosystems Dynamics Research. In 2019, she founded Vision Care and subsequently founded two subsidiary companies dedicated to developing cell and gene therapies.

Ascidian Therapeutics Inc. has received FDA clearance of its IND application for ACDN-01, an RNA exon editor targeting the genetic cause of Stargardt disease.

Bluerock Therapeutics LP, a wholly owned, independently operated subsidiary of Bayer AG, has exercised its option to exclusively license OpCT-001, an induced pluripotent stem cell (iPSC) derived cell therapy candidate for the treatment of primary photoreceptor diseases, from Fujifilm Cellular Dynamics Inc. and Opsis Therapeutics LLC.

Researchers from Broad Institute and Harvard University presented the discovery of all-in-one virus-like particles (VLPs) designed to deliver prime editor (PE) ribonucleoprotein (RNP) complexes into mammalian cells.

The hearing has returned for the first person who has received gene therapy for treating genetic hearing loss in the U.S. Initial results from Akouos Inc.’s phase I/II study showed that within 30 days of receiving AK-OTOF-101, pharmacologic hearing was restored to an 11-year-old who had profound hearing loss from birth.

Kriya Therapeutics Inc.’s unveiling of its new gene therapy program for thyroid eye disease (TED), KRIYA-586, added yet another player to the burgeoning space, where a handful of developers have reached the phase III stage.

E-Therapeutics plc has offered a pipeline update, following the nomination of novel target genes, which have yielded promising results in preclinical studies.