To clarify, I am both a long term investor and strong advocate for ANP and ATL1102.
As for the 13% amenable population - I’ve seen figures ranging anywhere from 13-20%, so I picked the most generous figure for argument’s sake and linked the source.
In regards to the use of the term ‘Gene Therapy’ vs ‘mRNA’:
As far as I understand, these terms are somewhat interchangeable in this context. Using AO’s such as Exondys-51 interferes with the pre-mRNA process by affecting E49-E51 reading on the DMD gene. As this process affects how the DMD gene is read and affects the subsequent mRNA created, ‘Gene therapy’ seems an appropriate term, as would ‘mRNA technology’. If I’ve missed a gap in understand this and there’s an important functional distinction then I’d love to understand this further.
I’d note I’m a tad confused as you initially state Sarepta’s drug is not gene therapy, but go on to say their gene therapy program delivered terrible results.
Regardless of semantics or terminology, I’m in complete agreement with you that ATL1102 looks to hold a much stronger position in the DMD therapeutics market than Sarepta’s drugs (existing and in pipeline).
For the record, I’m not sure what the need for your tone is here. I would think the whole point of this discussion thread is for enthusiastic investors to critically analyse ANP’s drug and the competitors in the space. I’m absolutely behind ANP and look forward to Phase 2B trial approvals being released.
Thanks for highlighting the issues they’ve encountered with the FDA and subsequent caution they’ll be using when evaluating future gene therapies/whatever you’d like to call it.
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