With the safety of gene therapies thrown into the spotlight by a series of FDA clinical holds, Astellas has said that an interim readout in a phase 1/2 trial of its Pompe disease candidate
NHS England has agreed a confidential deal with Orchard Therapeutics that will allow access to Libmeldy – a gene therapy for a rare childhood disease that is thought to be the most expensiv
The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file fo
Gyroscope Therapeutics and its gene therapy for the sight-robbing disease geographic atrophy (GA) has been acquired by a big pharma company – but likely not the one you would expect.
Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient.
Dutch biotech uniQure and partner CSL Behring are planning to file for approval of their gene therapy for haemophilia B, armed with new trial data that they say answers earlier concerns abo