My guess is that it may be something around NS Pharma's NDA for exon 53. Haven't heard whether their NDA was accepted and I think that deadline was in December for the FDA. I just wonder if Sarepta has something in motion to assert "orphan drug" protection? Perhaps that would be a question the DMD community would want answered too.
Also you might recall Wave LifeScience said it would provide the results of their trial exon 51 trial and even though they didn't show measurable dystrophin at 48 weeks, neither did AVI at the time. At least one of the boys had biopsies taken by Sarepta and by Wave, and I imagine it might be interesting to see how those biopsies compare.
Some tweets from last week from "oligogirl" on twitter who was attending the ParentProjectMD summit on gene therapy in San Diego.
Edited to add the tweets I had left out.
Tweets by "oligogirl" Jan. 24
"Lee Sweeney asks about treating older patients. Note that only a limited number of patients have been treated so - the field learns with each extra patient treated to also provide input to work towards treating older patients. pfizer is planning a NA trial.
@Sarepta is also considering a gene therapy trial for older patients. They are working on improving manufacturing. Both on scale and improving amount of virus per production run. Panel stressed that there is a lot that we do not know yet about benefits of young and old patients
Also a lot we do not know about risks in young and older patients - short term and long term. More information will come but important to be aware we are just getting started.
Panel agrees on trying to limit muscle biopsies as much as possible. However biopsies provide critical information on whether microdystrophin is expressed and the duration of expression. See also our paper on this topic - effort led by patient community
Treating older patients brings extra challenges though: production (older patients are bigger and need more virus), immunity (older patients may be more prone to have an immune response). Manufacturing needs to be improved to ensure costs do not increase when more virus is needed
Also a lot we do not know about risks in young and older patients - short term and long term. More information will come but important to be aware we are just getting started."