But I think the key is, and I think it’s very important to say that we are focused on building out a therapeutic portfolio in the area that we’ve outlined, particularly using our RNA editing capability. And that’s where we’re going to put our internal resources. Collaborations are great resources that we have in research payments that we can use into exploiting and exploring these are the capabilities that our chemistry can present. Your last question, I would want a little bit of clarity because I think there are 2 different components to it, which is where do we see N531 splicing and really exon skipping as we move it forward relative to the newly approved gene therapy or whether or not it’s potentially combined — whether you can work in combination.
So, I think it’s important as we think about demonstrating best-in-class editing, being able to deliver a highly specific, stable, durable skipping agent. That doesn’t require conjugates to get into cells to generate substantial amounts of exon skipping. I think we’ve got a very clear path in the exon that we hope just given our franchise. And I think seeing that translate and building that franchise is important. We know that gene therapy currently is only approved for 1 year of age for 4 to 5-year olds. So, within that range. And only in the U.S. under an accelerated approval. So it’s — we’re watching, we’re paying attention to where that data is. There’s, I know, data forthcoming at the end of this year, really on the translation of that microdystrophin.
So I think like everybody else, while we all saw that recent accelerated approval. I think there’s a lot to continue to follow on the benefit of microdystrophin and whether or not it’s functional. So I think there, we’re not going to take our eye off the ball and be distracted on not delivering functional dystrophin protein that we believe has based on our preclinical data based on following the Becker protein, being able to ultimately see that this type of protein when you create functional protein demonstrates a benefit. Importantly, as well as we think about the benefit of our skipping capability comes down to that question around our chemistry, which is different than others. I think sometimes within the enigma of talking about these companies, we think about our all exon-skipping being kind of fairly similar where it can go on a distribution basis.
And there have been a number of companies trying to drive better distributions in scalar muscle. What’s important is that our construct preclinically, both in our double knockout model as well as non-human primate, actually have higher levels of exon skipping in the heart and the diaphragm. So as we think about ultimately being able to out — change the outcome for these boys lives, it’s not only by repairing and fixing muscle and improving emulation. What’s most important to us is making sure we protect the heart and protect respiratory function. So, as we think about the totality of what we can bring forward potentially for exon skipping, we’re excited about what we can do with N531 and beyond, and we’ll continue to watch the field evolve in terms of microdystrophin.
Operator: Our next call comes from the line of Salim Syed from Mizuho.
Salim Syed: Great. Thanks for the questions, guys. Paul, just a few quick ones from us. Just more clarification than anything here. So with the CTAs now being imminent, I’m sure you have some idea when we’re getting data, and I think folks have tried to ask it on the call, but I’ll try to ask it maybe differently. Is the baseline thinking here that we’ll be able to get data in 2024? And also just curious when you’ll be able to — is the Phase 1 trial design being shared this year or are we getting that also in ’24? On DMD, the functional endpoints that you’ve mentioned, do those have any bearing on whether this Phase 2 is registrational? Or is it just going to be solely dependent on dystrophin levels? And then on 003, the wording in the press release, it seems like it changed a little bit versus the first quarter.
So I’m just curious, I think this time you guys added the word available multi-dose data versus last press release, the word available wasn’t in the press release. This press release was additional single dose and any available multi-dose, last press release was additional single and multi-dose, so curious what exactly has changed there in the multidose side?
Paul Bolno: Excellent. All right. So if we start, and thanks for the question, Salim. So we think about, let me just start write down in order AATD, DMD, HD. So as it relates to AATD time lines, as we said, the CTA submissions are imminent. Once we begin the trial, we can give a further update in terms of the expectations for data. I think it’s always important to see that trial kick-off. I think, it’s obviously safe to assume that data won’t be in 2023 for that study. But looking forward, I think when and what data we’ll have as we move into 2024 is something that we can provide updates on once that trial initiates. As it relates to DMD, you brought up a question on differentiation of filing based on dystrophin and what functional data we have.
