Gene Therapy

A natural history study shows patients who have Leber’s hereditary optic neuropathy with the ND4 mutation have a dramatic and irreversible decline in visual acuity, a press release from GenSight Biologics said.

ORLANDO, Fla. — In this video at the ASH Annual Meeting and Exposition, Steven W. Pipe, MD, professor of pediatrics and pathology at the University of Michigan, reviews recent innovations in hemophilia and incorporating gene therapy into practice.

A patient at France’s National Eye Hospital of the Quinze-Vingts has been granted temporary authorization for use of Lumevoq, GenSight Biologics’ gene therapy for the treatment of Leber hereditary optic neuropathy, according to a press release.

SAN FRANCISCO — RGX-314 gene therapy demonstrated meaningful reductions in anti-VEGF injections and increases in visual acuity and central retinal thickness among patients with wet age-related macular degeneration, according to an update presented at Retina Subspecialty Day at the American Academy of Ophthalmology annual meeting.

Wills Eye Hospital’s Ophthalmologist-in-Chief Julia Haller, MD, was recently selected to be one of 100 new members of the National Academy of Medicine, which recognizes individuals who have demonstrated outstanding professional and academic achievement and commitment to service.

SAN FRANCISCO — In the OPTIC trial, patients with wet age-related macular degeneration who were treated with ADVM-022, an intravitreal gene therapy, did not require additional injections following treatment, according to a presenter at the American Academy of Ophthalmology annual meeting.

The first patient has been dosed in the third cohort of the OPTIC phase 1 clinical trial of ADVM-022, an intravitreal gene therapy for the treatment of wet age-related macular degeneration, according to a press release from Adverum Biotechnologies.

SAN FRANCISCO — A gene therapy treatment candidate for Leber hereditary optic neuropathy bilaterally improved best corrected visual acuity during a phase 3 trial, according to a speaker here.

SAN FRANCISCO — Patients with wet age-related macular degeneration in the OPTIC trial treated with one in-office injection of ADVM-022, a gene therapy specifically designed for long-term intraocular VEGF suppression, maintained best corrected visual acuity and experienced improved central subfield thickness from baseline through 24 weeks’ follow-up, according to a speaker here.

Ocugen announced a strategic partnership with CanSino Biologics for gene therapy candidates and also the completion of its merger with Histogenics.