gene therapy

Sarepta’s Duchenne gene therapy shows promise in small study

The early peek at data from a Phase I/II study excited families, physicians, and investors 




gene therapy

Gene therapy start-up Freeline Therapeutics raises $116 million




gene therapy

Fetal gene therapy could treat genetic diseases in the womb

Researchers are working to fix genetic conditions like Gaucher disease before birth by testing experimental therapies on fetal animals