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.

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

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

The "North America RNA Therapeutics Market, By Country, Competition, Forecast & Opportunities, 2019-2029F" report has been ...

With the new operations site and logistics center in Sydney, “Australian researchers and biotech companies will no longer ...

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 ...

Crispr has already secured a historic first approval for a CRISPR/Cas9 drug and has several more potential blockbuster ...