Sarepta Therapeutics and Alnylam Pharmaceuticals have announced the pricing of $1 billion and $900 million in convertible senior notes respectively.
The FDA removed the clinical hold on Sarepta Therapeutics’ investigational Duchenne muscular dystrophy (DMD) therapy Tuesday after the company agreed to adjust its clinical trial protocols to include expanded monitoring of urine biomarkers.
The nonprofit biotech Cure Rare Disease (CRD) is headed to the clinic with a CRISPR therapeutic aimed at halting the progression of Duchenne muscular dystrophy in single-patient dosing.
One of the tenets of “personalized medicine” is that medications will be tailored to individuals based on their personalized genetics. Last year, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research approved 50 drugs. A new study found that 33, or 66% of them, were supported with genomic data.
Sarepta Therapeutics announced Thursday that the U.S. Food and Drug Administration has put the Phase II trial of its Duchenne muscular dystrophy (DMD) candidate SRP-5051 (vesleteplirsen) on clinical hold following a serious safety signal.