Sarepta Therapeutics

Licensing agreements for CNS-targeted oligonucleotide therapeutics increased 339% from 2023 to 2024, reaching $2.81 billion in total deal value according to GlobalData's analysis.

The FDA has granted Sarepta Therapeutics a platform technology designation for its rAAVrh74 viral vector, marking one of the first such awards since the program launched in 2024.

The FDA has placed a clinical hold on Rocket Pharmaceuticals' Phase II gene therapy trial for RP-A501 after a patient developed capillary leak syndrome and subsequently died.

Satellos Bioscience's investigational treatment for Duchenne muscular dystrophy (DMD) has demonstrated encouraging efficacy signals in adult patients, offering potential new options in a challenging therapeutic area.

• Comprehensive analysis of 700+ pharmaceutical and biotechnology partnering deals with disclosed royalty rates from 2010-2025 has been published by ResearchAndMarkets.com. • The report provides unprecedented access to actual contract documents submitted to the SEC, offering detailed insights into royalty rate structures and their integration with other financial terms. • Key industry players including AbbVie, Pfizer, Novartis, and Bristol-Myers Squibb are featured among the 50+ companies actively disclosing royalty rates in their partnership agreements.

• Pacira BioSciences has reached a settlement agreement with Fresenius, Jiangsu Hengrui Pharmaceuticals, and eVenus Pharmaceuticals regarding patents for its pain management drug Exparel, protecting market exclusivity until 2030. • Under the agreement, Fresenius will begin limited-volume sales of generic bupivacaine liposome injectable suspension in early 2030, with gradual market share increases reaching the high thirties percent by the final years of the agreement. • The settlement strategically preserves Pacira's market dominance for its lead drug Exparel for nearly a decade before allowing controlled generic entry, significantly extending protection beyond potential patent challenges.

Sarepta Therapeutics and Roche have paused their Elevidys (delandistrogene moxeparvovec) clinical trials in Europe after a patient death, while investigations into the cause are underway.

Peter Marks, head of FDA's Center for Biologics Evaluation and Research since 2016, has resigned, leaving cell and gene therapy developers without their biggest regulatory advocate during a challenging investment period.

Gene therapy is transforming treatment for neuromuscular diseases, with AAV-mediated approaches offering multiple strategies including adding, silencing, or editing genes depending on disease mechanisms.

Eteplirsen treatment significantly reduced the risk of cardiac function decline in patients with Duchenne muscular dystrophy, with treated patients showing a 78% lower risk of reaching left ventricular ejection fraction below 55% compared to controls.