The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying ...

CSL and uniQure's Hemgenix shows sustained efficacy in hemophilia B, reducing bleeding rates by 90% and eliminating ...

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

According to research by nova one advisor, the global cell and gene therapy market size was valued at $18.13 billion in 2023 ...

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

The American Cancer Society estimates that 83,000 new cases of bladder cancer will be diagnosed this year, with nearly 17,000 ...

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

Researchers have developed a gene therapy that may provide a long-term treatment for hypophosphatasia. Unlike current ...

Sangamo (SGMO) reported positive updated data from a Phase 1/2 study of its gene therapy candidate ST-920 in the treatment of Fabry disease. Read more here.