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.

Diabetic retinopathy and diabetic kidney disease are frequent microvascular complications of diabetes, both related to exacerbated vascular permeability coming from microvascular barrier malfunctioning.

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.

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

Investigators from Proqr Therapeutics NV have tested their product QR-1011, an antisense oligonucleotide designed to correct splicing abnormalities within the ABCA4 gene that affect protein expression levels, in Stargardt disease.