Gene Therapy

KOLOA, Hawaii — In this Healio Video Perspective from Retina 2023, Lejla Vajzovic, MD, associate professor of ophthalmology at Duke University, discusses advances in gene therapy.

Neurogene Inc. announced the FDA has granted clearance to its investigational new drug application for NGN-401, an adeno-associated virus gene therapy candidate for treating Rett syndrome in female pediatric patients.

KOLOA, Hawaii — In this Healio Video Perspective from Hawaiian Eye 2023, Allen C. Ho, MD, discusses gene therapy for wet and dry age-related macular degeneration and diabetic retinopathy.

Myrtelle Inc. announced updated findings of the open-label phase 1/2 clinical trial of its recombinant adeno-associated virus vector-based investigational gene therapy for Canavan disease, a fatal genetic disorder in children.

This year’s ASH Annual Meeting and Exposition spotlighted practice-changing research in malignant and benign hematology.

Infants with Artemis-deficient severe combined immunodeficiency improved after corrected genes in their stem cells enabled them to grow their own T cells and B cells, according to a study published in The New England Journal of Medicine.

The FDA lifted its partial clinical hold on bluebird bio’s sickle cell disease studies evaluating lovotibeglogene autotemcel among patients aged younger than 18 years, according to the agent’s manufacturer.

NEW ORLEANS —One-time treatment with the gene therapy betibeglogene autotemcel continued to demonstrate long-term efficacy among patients with transfusion-dependent beta-thalassemia, study results showed.

Gene therapy could represent an equitable therapeutic strategy for sickle cell disease in the United States, according to study results presented at ASH Annual Meeting and Exposition.

In this Healio Video Perspective, Lejla Vajzovic, MD, FASRS, discusses promising 6-month data from the ALTITUDE trial investigating RGX-314 gene therapy for the treatment of diabetic retinopathy.