Sarepta Therapeutics

Aug 20, 2024

Sarepta Therapeutics, a leader in precision genetic medicine for rare diseases, has recently achieved a significant milestone with the FDA's approval of AMONDYS 45 (casimersen). This antisense oligonucleotide, developed from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, is now approved for treati...

Aug 20, 2024

A rare muscle disorder, Duchenne Muscular Dystrophy (DMD), approximately affects 1 in 3,500 male births globally. In 2023, the total number of prevalent cases of Duchenne Muscular Dystrophy in the 7MM (The US, EU5, and Japan) was around 31,400. The United States encompasses the highest prevalent population of Duche...

Jan 17, 2024

From January 8th to 11th, 2024, the 42nd Annual J.P. Morgan Healthcare Conference (JPM24) took center stage in San Francisco, CA, USA. Spanning four dynamic days, this conference saw the active participation of prominent figures from major pharmaceutical, biotechnology, Medtech, HealthTech entities, and emerging fa...

Jun 30, 2023

Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease that affects 1 in every 3,500 to 5,000 male neonates worldwide. It is caused by mutations in the gene encoding the protein dystrophin. These genetic changes emerge as developmental delays and, in more severe types of DMD, limb weakness, loss of indep...

Jun 27, 2023

FDA Approves Jardiance for the Treatment of Type 2 Diabetes in Children 10 Years and Older Boehringer Ingelheim and Eli Lilly and Company announced that the FDA has approved Jardiance® (empagliflozin) 10 mg and 25 mg tablets to decrease blood sugar together with diet and exercise in children 10 years and older w...

May 16, 2023

Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy Sarepta Therapeutics, Inc., a pioneer in precision genetic medicine for rare diseases, announced that the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in favor of...

Jun 23, 2020

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...

Dec 24, 2019

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...

Apr 09, 2019

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...

Feb 19, 2019

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...