RMAT designation enables intensified FDA engagement and potential accelerated pathways, implying preliminary clinical evidence for RTx-015 despite absent peer-reviewed efficacy details, endpoints, and ...
A groundbreaking gene therapy tested through a UCLA-led clinical trial has received approval from the U.S. Food and Drug ...
A gene therapy that targets a form of retinitis pigmentosa showed a signal to improve retinal function for up to 3 years after treatment, according to a small clinical trial. The phase 2 SKYLINE trial ...
Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...
Opus Genetics has shared data on its gene therapy in children with an ultrarare eye disease, setting the biotech up to talk to the FDA about the next steps for the program. North Carolina-based Opus ...
Yvette Crawley, 71, from Apex, N.C., started noticing changes to her vision around the time she turned 60. She described it as if she were “looking through a dirty window.” She was diagnosed with a ...
Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...
A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...
In a phase 2 trial, OCU410, a modifier gene therapy for geographic atrophy secondary to dry age-related macular degeneration, reduced lesion growth and size while slowing ellipsoid zone loss, ...
Opus Genetics Inc. will be sitting down with the U.S. FDA to talk about positive three-month data from the pediatric cohort of its ongoing phase I/II trial called OPGx-LCA5-1001 – partially funded by ...
Researchers at NYU College of Dentistry's Pain Research Center have developed a gene therapy that treats chronic pain by indirectly regulating a specific sodium ion channel, according to a new study ...
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