Gene Therapy

Applied Genetic Technologies exceeds enrollment target for SKYLINE trial of AGTC-501

AGTC-501 is a recombinant AAV vector-based gene therapy developed for the treatment of X-linked retinitis pigmentosa.

4D Molecular Therapeutics announces first patient dosed in Phase 1/2 Clinical Trial of 4D-150

According to the company, 4D-150 is a dual-transgene, intravitreal gene therapy designed to inhibit four distinct angiogenic factors to prevent angiogenesis and reduce vascular permeability for the treatment of wet AMD.

UCI investigators use cryo-electron tomography to reveal molecular mechanisms underlying mutations within the eye that lead to blindness

Team at University of California, Irvine provides an understanding of the mechanisms underlying the pathologies of certain gene mutations.