Sarepta Therapeutics, Inc.
NEWS
The U.S. FDA approved Sarepta Therapeutics’ Amondys 45 (casimersen) for patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation amenable to exon 45 skipping.
The new year began with a fairly low level of clinical trial news. Here’s a look.
Sarepta Therapeutics announced topline results from Part 1 of Study SRP-9001-102 for its gene therapy SRP-9001 for Duchenne muscular dystrophy.
Sarepta Therapeutics entered into a research alliance with Codiak BioSciences to design and develop engineered exosome therapeutics for neuromuscular diseases.
It was a fairly busy week with clinical trial updates and announcements. Here’s a look.
On Monday, Sarepta Therapeutics announced positive results from a small study of SRP-9003, its gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E).
Dyno says that with its research-and-development and collaboration deals with biopharma companies, it is potentially eligible for more than $2 billion in upfront payments, research support, and various milestones and option fees.
As the biopharma industry responds to the COVID-19 pandemic, some of the area’s companies are at the very frontlines of drug development against the disease, while others play supporting roles for the entire industry. Here’s a look.
The CRL not only brings up the potential toxicities of the drug, but also raises questions about the drug’s efficacy.
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