Gene Therapy

Targeting human retinal progenitor cell injections for RP

Investigators find approach improved vision and visual fields in some patients.

Unraveling the genetics of glaucoma

Details enhance knowledge of biological pathways that contribute to disease pathogenesis.

Investigators start pivotal subretinal gene therapy trial

RGX-314 eyed by investigators as a therapeutic option for exudative AMD.

Oyster Point Pharma unveils preclinical study results for Enriched Tear Film Gene Therapy to target ocular surface diseases

June 03, 2021

According to the company, preclinical study results from a 42-day proof-of-concept in vivo study demonstrated a single, intralacrimal gland injection of an adeno-associated virus containing the NGF gene produced statistically significant increase in NGF in tear film, as compared to placebo, as early as Day 7.

Results unveiled from phase 2/3 gene therapy study for XLRP

May 14, 2021

While Biogen’s XIRIUS study did not meet its primary endpoint of demonstrating a statistically significant improvement in treated eyes, positive trends were observed across several clinically relevant prespecified secondary endpoints.

Gene therapy prompts foveal morphologic changes in LCA

May 10, 2021

Friederike Kortuem, MD, MSc, discusses how treatment with voretigene neparvovec-rzyl led to a short-term change in the foveal morphology in a patient with visual impairment that included nyctalopia and decreased visual acuity in early childhood.