Natural History Study Launches for LCA Caused by Specific Mutation in CEP290

Audio version:
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.

FFB-CRI Investing $7.5 Million in Emerging Therapy for USH2A

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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.

AGTC Launches XLRP Gene Therapy Clinical Trial at Five Sites in U.S.

Audio version:
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.

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