Gene Therapy

Regenxbio has initiated a second phase 3 pivotal trial investigating RGX-314 in patients with wet age-related macular degeneration, according to a press release.

The first patient has been dosed in a phase 1/2 clinical trial investigating 4D-150 for the treatment of wet age-related macular degeneration, according to a press release from 4D Molecular Therapeutics.

The top ophthalmology features in 2021 discussed champagne cork safety and the dangers of ocular exposure to hand sanitizer.

Novel therapies for patients with age-related macular degeneration as well as the impact of the COVID-19 pandemic on treatment for this population were popular topics at ophthalmology meetings in 2021.

Gene therapy could have the potential to revolutionize the treatment of many conditions, including age-related macular degeneration.

The first patients have been dosed in the phase 2/3 Sirius and Celeste clinical trials investigating QR-421a in patients with USH2A mediated retinitis pigmentosa and Usher syndrome, according to a press release from ProQR Therapeutics.

In this video, Matthew M. Heeney, MD, spoke with Healio about presentations on gene therapeutic options for hematologic diseases from ASH Annual Meeting and Exposition.

Patients with Leber hereditary optic neuropathy treated with Lumevoq experienced sustained efficacy and safety after 2 years as part of the phase 3 REFLECT clinical trial, according to a press release from GenSight Biologics.

Patients with transfusion-dependent beta-thalassemia who received betibeglogene autotemcel achieved durable transfusion independence with near-normal hemoglobin levels, according to study results with up to 7 years of follow-up.

The American Society of Hematology will present this year’s ASH Outstanding Service Award to Peter Marks, MD, PhD.