Three draft recommendation documents published on Wednesday are intended to guide drug sponsors and accelerate the ...

After three patients died, the maker of an investigational gene therapy stopped shipments of the drug after it refused to ...

Scientists from Tel Aviv University and Boston Children's Hospital use a optimized virus to deliver a corrected gene into the ...

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct ...

A patient has died in a clinical trial evaluating a Rocket Pharmaceuticals gene therapy shortly after complications emerged that led the FDA to pause the study. An inquiry into the fatality is ...

UCLA researchers have made strides in developing a stem cell gene therapy for alpha thalassemia major, a severe, inherited ...

Gene therapies are unlocking new drug targets that were previously thought undruggable. Covering modalities like ASOs, siRNA, saRNA, mRNA, and ADARs, each with different mechanisms of action, gene ...

The first participant in an investigational gene therapy trial of STXBP1 encephalopathy died, Capsida Biotherapeutics said. The death occurred days after dosing, according to information posted on ...