Sarepta Snaps Up Gene Therapy Approaches to DMD

Sarepta may have launched its first therapy for Duchenne muscular dystrophy in the US, but it is facing potential competition from a plethora of late-stage DMD therapies; it is backing research that could lead to new gene therapies able to treat most affected patients, rather than a subgroup of individuals with a particular gene mutation.

Sarepta Therapeutics Inc. has licensed rights to two gene therapy research programs underway at the Nationwide Children’s Hospital, Columbus, Ohio, adding new projects to its growing Duchenne muscular dystrophy pipeline to complement its recently launched lead DMD product Exondys 51 (eteplirsen).

Marketing a range of therapies for a specific disease or condition is a well-established strategy for pharmaceutical companies, particularly in a disease like DMD that is mediated by a number of different genetic mutations

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