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 heterogeneous nature of DBA mutations makes developing a single therapy challenging. Moreover, current gene therapy ...

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

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

After a research note raised potential safety concerns with Amgen's lead obesity candidate, the company defended its drug and ...