Studies in non-human primates demonstrated nearly 100% gene editing and knockout of endogenous RHO gene and more than 30% replacement protein levels using a dual vector AAV approach…
• The primary outcome of the MGT009 study is safety, and botaretigene sparoparvovec treatment was found to be generally safe and well-tolerated • Significant improvements were…
Retinitis pigmentosa, a degenerative genetic disease of the eye, is characterized by progressive vision loss, usually leading to blindness. In some patients, structural defects in the…
Studies in non-human primates demonstrated nearly 100% gene editing knockout of endogenous RHO gene and more than 30% replacement protein levels Treated eyes showed morphological and…
Researchers from the University of California, Irvine have discovered that the absence of Adiponectin receptor 1 protein (AdipoR1), one of the principal enzymes regulating ceramide…
4D-125 was well tolerated in all patients treated to-date (n=8), with no dose-limiting toxicities, no serious adverse events and no chronic inflammationClinical activity observed through…