StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

By exploiting the genetic variation in cancer cells, an already approved cancer drug demonstrated enhanced effects against cancer cells in specific patient groups.

Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...

Tel Aviv University researchers have made a discovery that could enhance our understanding of genetic mutations and their ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The ...

The company is paying China-based biotech LaNova Medicines $588 million for the kind of dual-pronged antibody drug that ...

The development of gene therapy and mRNA vaccine technologies has been transformative, particularly during the COVID-19 ...

The Mass General Brigham Gene and Cell Therapy Institute, a hub of innovation dedicated to accelerating groundbreaking research, conducting clinical trials and developing FDA-approved treatments, ...

CAR-T therapy leverages the body’s immune system to target ... and can accurately quantify CAR gene copy number or mRNA ...