Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the ...

Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first ...

Scientists looked at multiple techniques used to measure the modified viruses deployed in some gene therapy research and treatments. One technique, known as SEC-MALS, was the most precise and ...

With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now found a commercialization partner for both the U.S. and Asian markets.

Sangamo Therapeutics (NASDAQ:SGMO) reported positive updated data from a Phase 1/2 study of its gene therapy candidate isaralgagene civaparvovec, or ST-920, in the treatment of Fabry disease. The ...

The University of Southern California (USC) has received a $6m grant from the California Institute for Regenerative Medicine ...