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.
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Including upfront payments, half of development costs for SRP-9001, $1.7bn in milestone fees and royalties on sales outside the US, Sarepta pegs deal’s value at $10bn-plus.
Ongoing trial is intended to compare the newly approved steroid Emflaza with prednisone in DMD for efficacy and safety.
Aduro Biotech discusses what it looks for in an academic collaboration. Plus, Sarepta adds gene therapies to its Duchenne pipeline from Nationwide Children's Hospital and Columbia University out-licenses IP to a pair of biotechs.