Sarepta Therapeutics

Sarepta Therapeutics, a leading player focused in developing precision genetic medicines for rare diseases with more than 25 programs ongoing, has recently announced the submission of New Drug Application (NDA) to the US FDA for Casimersen (SRP-4045) for the treatment of Duchenne muscular dystrophy (DMD) with a gen...

Sarepta Therapeutics has announced a research and option alliance with Codiak BioSciences to design and develop engineered exosome therapeutics for neuromuscular diseases The engineered exosome technology is believed to improve the delivery of delivering gene therapy, gene editing and RNA technologies for ne...

The Duchenne Muscular Dystrophy Market Size (DMD Market) in the 7MM was found to be USD 266.06 Million in 2017, during the study period (2017-2030). A rare muscle disorder, Duchenne Muscular Dystrophy (DMD), approximately affects 1 in 3,500 male births globally. Moreover, Duchenne Muscular Dystrophy prevalence ...

Roche has entered into a licensing partnership with Sarepta Therapeutics worth USD 1.15 Billion.  Roche has agreed to license in Sarepta’s gene therapy ‘SRP-9001’ for Duchenne muscular dystrophy (DMD), though which Roche will gain access to exclusive rights to it globally except in the US.  As per t...

Anylam, Regeneron to broaden the scope of R&D in Pharma Alnylam in collaboration with Regeneron is expanding its research on NASH. Regeneron with the capability of generating various targets via antibody approach is going to get infused with USD 800 million upfront. The joint deal will help in expanding R&am...

CRISPR treatment delivers benefits in mice The researchers from Duke University along with drugmaker Sarepta Therapeutics published a report stating that a single change in the genetic system of Duchenne muscular dystrophy (DMD) affected mice, using CRISPR gene editing tool resulted in increasing their life spa...

PMV Pharma Raises $74M, Aiming to Hit Cancer’s Hard Target- P53 PMV Pharmaceuticals is the latest startup to load up with cash in an attempt to home in on one of the toughest targets in cancer biology: the tumor suppressor protein known as p53. The company raised a $74 million Series B round from Topspin Biotech Fun...

The Food and Drug Administration approved the recent controversial drug to treat Duchenne muscular Dystrophy, which is a rare disease that confines boys to wheelchairs and condemns them to an early death. The decision was made after months of debate between the Agency and Sarepta Therapeutics, regarding the evidence...