Researchers at Carl von Ossietzky Universität Oldenburg generated retinal pigment epithelial cell lines lacking three RPGR isoforms and examined whether they showed the defects in ciliary structure and actin turnover known to occur in retinitis pigmentosa.
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.
Inherited retinal dystrophies (IRDs), including retinitis pigmentosa and Stargardt disease, are a group of rare degenerative disorders of the retina with clinical and genetic heterogeneity. In a recent publication, researchers from the Institute of Ocular Microsurgery applied clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 and transcription activator-like effector nucleases (TALEN) gene-editing tools to precisely correct induced pluripotent stem cell (hiPSC) lines derived from IRD patients.
After raising AU$40 million (US$25.9 million) earlier this year to advance lead candidate VP-001 for treating retinitis pigmentosa type 11, PYC Therapeutics is now advancing PYC-003 to the clinic for polycystic kidney disease.
Retinitis pigmentosa (RP) is the most common hereditary degenerative eye disease that leads to progressive vision loss, primarily caused by retinal degeneration.
Skyline Therapeutics (Shanghai) Co. Ltd.’s SKG-1108, a novel one-time intravitreally delivered gene therapy, has been awarded U.S. orphan drug designation for the treatment of retinitis pigmentosa.
Rznomics Inc. has received clinical trial notification (CTN) from Australia’s Therapeutic Goods Administration (TGA) for the initiation of a phase I/IIa trial evaluating RZ-004, a gene therapeutic candidate for autosomal dominant retinitis pigmentosa with rhodopsin mutation.
-and-Raymond-Wong-(R).webp?height=488&t=1755204408&width=650 "Mirugen co-founders Keith Martin (L) and Raymond Wong (R)")
