Retina treated with gene therapy
Notable industry executive brings more than 15 years of ophthalmic experience and award winning achievement to FFB’s mission.
The Massachusetts Eye and Ear Infirmary (MEEI) and other centers in the US and Europe are conducting a natural history study for people with Leber congenital amaurosis 10 (LCA10) caused by a mutation referred to as “intron 26 c.2991+1655A>G” in the CEP290 gene.
The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) has entered into a partnership with ProQR to develop a retinal therapy for people with Usher syndrome type 2A (USH2A) caused by mutations in exon 13 of the USH2A gene. FFB-CRI will be investing up to $7.5 million in milestone-based funding to advance the treatment, known as QR-421a, toward a Phase 1/2 clinical trial during 2018. ProQR plans to issue the initial data report for the clinical study in 2019.
Applied Genetic Technologies Corporation (AGTC) is now recruiting for its Phase 1/2 gene therapy clinical trial for males with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. Approximately 15 patients will be enrolled in the study, which is primarily evaluating safety. Three doses of the gene therapy will be tested. The trial is taking place at five sites in the U.S.
Your browser does not support the audio element.
Audio Version:Your browser does not support the audio element.
Jason Morris, Chief Administrative Officer at TKC Holdings, has been appointed to the Foundation Fighting Blindness Board of Directors, the Foundation announced today.