gene therapy
Rett Syndrome Patient Dies in Neurogene Phase I/II Trial After High Dose of Gene Therapy
The patient died from complications linked to a rare hyperinflammatory syndrome associated with exposure to high doses of the AAV therapy delivery vehicle.
Novartis Acquires Kate Therapeutics in $1.1B Deal
The acquisition is poised to strengthen Novartis' position in the gene therapy market for neuromuscular conditions.
Voyager Therapeutics Selects Tau-Silencing Gene Therapy Candidate
The company said it expects to seek regulatory permission to begin clinical testing of VY1706 in the US and Canada in 2026.
Regenxbio Treats First Patient in Pivotal Stage of Duchenne Muscular Dystrophy Gene Therapy Trial
The company expects the trial to support the submission of a biologics license application with the US Food and Drug Administration in 2026.
Medera, CADENCE Launch Heart Failure Gene Therapy Trial in Singapore
In the trial, researchers will test how well Medera's SRD-001 gene therapy improves symptoms in heart failure patients with reduced ejection fraction.
Nov 7, 2024
Ocugen Obtains $30M Credit Facility
Nov 4, 2024