Gene Therapy

Lindsey A. George, MD, discussed “exciting” preclinical data on gene editing for hemophilia B, which was presented in a session at ASH Annual Meeting and Exposition.

The first two female pediatric patients with Rett syndrome have been dosed in an ongoing phase 1/2 trial of investigational AAV9 gene therapy NGN-401, according to the manufacturer.

Alexis Thompson, MD, MPH, is an optimist.

SAN DIEGO — One-time treatment with lovotibeglogene autotemcel gene therapy conferred “transformative” benefit to patients with sickle cell disease, according to a presenter at ASH Annual Meeting and Exposition.

Health Canada has authorized an amendment to the ongoing REVEAL phase 1/2 trial evaluating an AAV9 investigational gene transfer therapy that expands eligibility to patients with Rett syndrome aged 12 years and older.

Fewer than half of those dealing with Parkinson’s disease in the United States are able to gain clinical control of symptoms without experiencing side effects, according to results of a new study.

A nonprofit focused on treatments and cures for Charcot-Marie-Tooth disease will partner with a biotechnology firm to advance a novel intrathecal miRNA therapy to address CMT type 1a in adults with the condition.

Individuals with severe sickle cell disease showed willingness to accept the risks associated with emerging gene therapy treatments, according to survey results published in Blood Advances.

Receipt of 4D-710, an aerosolized gene therapy, resulted in high cystic fibrosis transmembrane conductance regulator protein expression levels in patients with cystic fibrosis, according to a company press release.

Healio and Cardiology Today have compiled a list of the most-read cardiology news of October 2023.