With >80% rare diseases having known monogenic (single-gene) cause. Traditional therapies for these diseases require frequent administrations and are only symptomatic. In contrast, gene therapy can correct the underlying genetic defects, offering a cure. Successful gene therapy may require only a single dose to confer lifelong improvement rather than requiring a lifetime of ongoing treatment. Even amid setbacks with regulatory authorities, companies have been investigating gene therapy approaches to treat rare diseases. In this digest, we bring you innovators and their journey in gene therapy research in different rare diseases like Hemophilia B, SMA, SCID, and Maple syrup urine disease, etc.