Sarepta Therapeutics, Inc.
NEWS
The World Muscle Society 2022 Congress is ongoing in Halifax, Canada, with numerous companies presenting cutting-edge research in neuromuscular diseases. Here’s a look.
The FDA had a busy week, approving drugs, greenlighting clinical trials and other regulatory activities for Immuneering, Amylyx, Sarepta, Sanofi, and Regeneron and more.
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’s investigational treatment for certain types of DMD after the company agreed to adjust its clinical trial protocols.
CRD’s CRD-TMH-001 is expected to upregulate an alternate form of the dystrophin protein with CRISPR technology and stabilize or reverse the symptoms associated with DMD.
In its Q2 conference call, Sarepta Therapeutics indicated plans to accelerate its timeline for SRP-9001 a gene therapy for Duchenne muscular dystrophy (DMD).
Last year, the FDA’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 and its partner, Roche, presented promising functional data from several trials of its gene therapy for Duchenne muscular dystrophy.
The FDA lifted the clinical hold on Dyne Therapeutics’ clinical study of DYNE-251 in Duchenne muscular dystrophy (DMD) patients amenable to skipping exon 51. A Phase I/II trial is expected by mid-year.
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