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

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, ...

Find out what is driving the slowdown in gene therapy investment, as the sector continues to struggle with widespread layoffs ...

Good afternoon, ladies and gentlemen, and welcome, everyone, to the Maravai LifeSciences Third Quarter 2024 Earnings Conference Call. All lines have been placed on mute to prevent any background noise ...

An RNA-editing gene therapy has been developed that switches off the key driver of common eye conditions affecting diabetics ...