Below is a Q&A on the importance to Sarepta Therapeutics of...

Below is a Q&A on the importance to Sarepta Therapeutics of their PPMO program ( from Sarepta's Q1 Earnings Call on 10 May).
Just a re-fresh: Sarepta's PPMOs consist of a cell-penetrating peptide linked to a PMO (PMOs are a form of ASO). Sarepta have a PPMO in clinical trials and 5 in pre-clinical (targeting duchenne muscular dystrophy). The PPMO in clinical trials is using the PMO developed by Wilton and Fletcher.
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H.C. Wainwright and Company -- Analyst

So just as a follow-up on the PPMO program. Again, given the investment that you're seeing from peers, how important is this PPMO program to the overall franchise, especially focused on rescuing the non-ambulant patients?

Doug Ingram -- Chief Executive Officer

Yes, thank you. Well, we're very excited about the PPMO program. We don't -- we try not to talk a lot about it because we don't want to pump it right now. We're in the middle of a multi-setting dose.

We've had a very successful single-setting dose trial. We're starting our multi-setting dose at 4 mgs per k. We need to get a lot higher. We'd like to get a lot higher than that.

But one of the things we know in the PPMO is just to remind people -- I'll remind people [Inaudible] people are otherwise unaware, the PPMO program, which is our RNA therapy, it is the same back-up as PMO. We conjugated a peptide that allows, No. 1, the [Inaudible] becomes positively charged. It allows much greater penetration into the cell.

And in preclinical models, if we could get up to high enough doses, we can see as much as an order of magnitude, more expression and therefore, more benefit than our PMO technology. So we're really focused on that. And so it is a very big part of our thesis. It's a big part of our thesis for patients with Duchenne muscular dystrophy both, frankly, ambulatory and non-ambulatory patients, and it will be an important part of our platform going forward, beyond Duchenne muscular dystrophy.

One of the things that Dr. O'Neill and his team in research are working on is target selection, both from our PMO, our PMO plus and our PPMO programs, that the target selection beyond Duchenne in the areas where our RNA technology would make brilliant stands, sometimes even -- sometimes in ways that perhaps gene therapy couldn't actually address, places where, for instance, the gene is so large, it's not easily packaged in AAV and the like. So the PPMO program is important. We're tracking to get inside by the end of this year and in the first quarter of next year.

We have 6 constructs in the PPMO program. They cover about 43% of the Duchenne community, and we'll certainly provide an additional update on that at the end of this year, early into 2020.
https://www.**promotion blocked**/earnings/call-transcripts/2019/05/10/sarepta-therapeutics-srpt-q1-2019-earnings-call-tr.aspx
https://www.sarepta.com/our-pipeline