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.
N4 Pharma plc’s subsidiary Nanogenics Ltd. has signed a contract to start the formulation and sequence selection work to prepare its ECP-105 product for testing 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.
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