The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Well, great. Well, Oxana, really happy to have you here, and let's take advantage to talk about some of your design aspects. I mean, obviously, the
company was formed to be able to facilitate the delivery of oligos outside the liver. You guys are conjugating an antibody or antibody fragment.
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OCTOBER 03, 2023 / 3:00PM, DYN.OQ - Dyne Therapeutics Inc at Chardan Genetic Medicines Conference
So maybe talk about some of the advantages you see in your approach versus some of the other competitive approaches such as using a conjugated
protein or cell-penetrating peptide.
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Okay, and in terms of potential endosomal escape advantages of other targeting moieties, how would you address that from your perspective?
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Okay, great. And let's kind of expand a little bit on design considerations. For DM1, we're seeing a number of different approaches that I think
people appreciate. It is a repeat expansion disorder, results in the formation of this toxic DMPK in the nucleus, it binds important splicing proteins.
So different ways to kind of address that toxic DMPK, the foci.
So, in your approach, you're delivering an ASO; the goal is to kind of standard RNase H degradation. Others are choosing to address it using a risk.
Others are using to attack it via steric hindrance. So talk about the advantages you see with your approach.
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Okay. So investors are eagerly anticipating the presentation of your initial data from your first-in-human studies. Let's start with ACHIEVE for 101
for DM1. Maybe tee up, first of all, which -- the data for which patient cohorts we'll see, so let me ask you that first.
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Sure, absolutely.
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Okay, great. So you had been preceded by another company in terms of the splice correction data. I think it's well understood that the Thornton
panel 22 targets is a very good standard. So relative to that, how might your data be presented?
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Okay, great. And then just to touch on a point you just mentioned, the study is designed to be potentially registrational. So at this point, what is
your understanding of what it might take to have this be perhaps adjusted slightly to make it potentially registrational?
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Okay. Well, great. So again, everybody is looking forward to that data set; it will be great to see it. So let's move on to your second program targeting
DMD, specifically exon 51. So back to you, Oxana. With your PMO in this case, but delivered with your targeting moiety, help us think about your
preclinical data and really how it highlights the potential advantages that you may have versus a naked PMO.
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Okay. So again, here again, we're anxiously waiting to see these initial results. So can you tee up what we should expect to see you report on?
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OCTOBER 03, 2023 / 3:00PM, DYN.OQ - Dyne Therapeutics Inc at Chardan Genetic Medicines Conference
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Okay, great. So here again, the DELIVER study is designed to be potentially registrational. The huge benefit here is that we have kind of a well-defined
regulatory pathway. So let's assume that this data is very positive. How does the study then proceed from there to make it registrational?
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: And so my question, Josh, would be, again, let's assume we see really nice data here for DMD, why wouldn't you then kind of put the pedal to the
metal to advance your other targeted exon mutations that you mentioned and obviously finding a way to make that happen?
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: Okay. Well, great. And again, I know this data is coming, so it will be great to see it when it comes out. So maybe just a couple of questions left, one
would be on in terms of your cash. What length of runway does it currently support?
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: So maybe just a way to kind of tie this up. What do you feel that investors are either not fully understanding or underappreciating about the stock?
Question: Keay Nakae - Chardan Capital Markets, LLC - Analyst
: All right. Well, very good. Thank you so much for being with us today.
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