Lyora Therapeutics Inc. has launched with seed funding and a focus on genetic medicines targeting the root cause of inherited retinal diseases. The company has acquired advanced preclinical assets, which it plans to optimize and advance to be delivered locally and remain durable for life.

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.

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.

Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional CRISPR/Cas9 editing.

Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional Crispr-Cas9 editing.

Axovia Therapeutics Inc. has been awarded a new $1.0 million grant by the nonprofit organization A Race Against Blindness to support the clinical development of AXV-101, an investigational gene therapy aimed at combating childhood blindness due to retinitis pigmentosa caused by Bardet-Biedl syndrome 1 (BBS1).

Aussie gene therapy company Mirugen Pty Ltd. has emerged from stealth mode from the Center for Eye Research Australia in Melbourne, toting a AU$4.5 million (US$2.9 million) seed round that will see it optimize its lead candidate to treat retinitis pigmentosa.

Aussie gene therapy company Mirugen Pty Ltd. has emerged from stealth mode from the Center for Eye Research Australia in Melbourne, toting a AU$4.5 million (US$2.9 million) seed round that will see it optimize its lead candidate to treat retinitis pigmentosa.

Variant SAS and the Rare Ocular Diseases Center at the University of Campania Luigi Vanvitelli (UCLV) have received positive feedback from the EMA for VAR-002, a recombinant AAV vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations.