Gene Therapy

INDIANAPOLIS — A single in-office treatment of ABBV-RGX-314 was well-tolerated and improved severity of diabetic retinopathy, according to 1-year study results presented at Academy 2024.

The FDA granted investigational drug clearance to HG202, a CRISPR/Cas13Y RNA-editing therapy for neovascular age-related macular degeneration, according to a press release from HuidaGene.

CHICAGO — In this Healio Video Perspective from Eyecelerator@AAO, Sam Barone, MD, of Nanoscope Therapeutics discusses the mutation-agnostic gene therapy MCO-010.

Topline data from a phase 2 clinical trial of an investigational gene therapy for Parkinson’s disease showed that a higher dose of the medication significantly improved disease-specific and quality of life measures by week 26.

In this video, Alokkumar Jha, PhD, MSc, BE, spoke with Healio about key presentations from the American Neurological Association annual meeting.

Bluebird Bio’s lentiviral vector gene therapy Skysona met its primary efficacy endpoint of survival without major functional disability in 81% of boys with early cerebral adrenoleukodystrophy, a recent analysis showed.

Patient readiness will greatly affect the success of gene therapy for sickle cell disease at the individual and collective levels, according to a consensus statement published in JAMA Network Open.

In this video, Barbara Blodi, MD, discusses advancements in gene therapy for the treatment of age-related macular degeneration.

In this video, Donald J. D’Amico, MD, discusses data from the phase 1/2 PRISM trial of 4D-150 in patients with neovascular age-related macular degeneration.

In this video, Donald J. D’Amico, MD, discusses data from the phase 2 LUNA trial of Ixo-vec intravitreal gene therapy for neovascular age-related macular degeneration.