The biotech Sarepta has had a complicated go of it with the FDA sometimes related to their Duchenne muscular dystrophy (DMD) gene therapy efforts.
In late 2016 I wrote about how there was some controversy as the FDA approved the Sarepta drug eteplirsen (Exondys 51) also for DMD, going against an advisory panel that had recommended against approval.
Now STAT reports that an internal FDA group was going to reject the firm’s drug SRP-9001, but that CBER Director Peter Marks decided to have it get a look by an advisory panel, which will happen in mid-May.
There were earlier reports that Sarepta had originally thought the FDA was going to proceed with review without even requiring an advisory meeting at all.
DMD patients need new options so I wish Sarepta well and hope they get excellent data. You can see a video below that is a kind of clinical trial update on SRP-9001 from Sarepta within a webinar.
What is DMD?
From the Muscular Dystrophy Association, DMD is a “genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin.”
My lab does epigenomics research on stem cells and childhood cancers so I’m often on the lookout for new epigenome papers. This week there were some good ones.
What is the epigenome? One way to think about it is as the intersection of epigenetics with the genome. Epigenomics research focuses on defining mechanisms regulating the epigenome and its functional outputs to cell behavior.
- DNMT3A-coordinated splicing governs the stem state switch towards differentiation in embryonic and haematopoietic stem cells, Nat. Cell Bio. This is a cool paper led by Peggy Goodell.
- Dynamic antagonism between key repressive pathways maintains the placental epigenome, Nat. Cell Bio. Another cool Nat. Cell Bio paper, this one from a team led by Alexander Meissner.