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 - Analyst
: Josh, to start out, could you provide a couple minutes of introductory comments about Dyne for investors new to the story?
Question: Keay Nakae - Chardan Capital Markets - Analyst
: Well, great. Thank you for that introduction, Josh. I think most of our audience is familiar with the huge value that the utilization of GalNAc paired
with the receptor on liver hepatocyte cells has had in advancing the field. So Oxana, what we find really attractive about your FORCE design platform
is that it leverages the selectivity of the antibody fragment as you are targeting moiety, with the precision of the oligonucleotide sequence payload,
along with the flexibility to deliver an siRNA or ASO when the target avails the opportunity. And this flexibility is key to I think moving -- again,
advancing the field and in your case initially targeting muscle. While all this may sound straightforward, can you talk about some of the key
challenges you've engineered solutions to for each of the components, as well as how to assemble them, which has really created the value of your
platform?
Question: Keay Nakae - Chardan Capital Markets - Analyst
: Yes, so we will get to that. But, Josh, when we think about using a conjugated antibody fragment versus a full monoclonal antibody, talk about the
advantages from your perspective of using the fragment.
Question: Keay Nakae - Chardan Capital Markets - Analyst
: So, Josh, with those advantages, from an IP perspective, what's defensible at this point with your proprietary FORCE design approach?
Question: Keay Nakae - Chardan Capital Markets - Analyst
: Okay, great. So, Oxana, going back to your initial targets, which are muscle disorders, one of your most advanced compounds is myotonic dystrophy
1; it's a CTG repeat expansion disorder. You've got this toxic mRNA that's in the nucleus, trapping MBNL, which is involved in appropriate splicing.
You're choosing to deliver an ASO payload conjugated to the Fab. Can you describe the advantages in this approach compared to other competitive
initiatives utilizing either an siRNA or a PNA oligonucleotide?
Question: Keay Nakae - Chardan Capital Markets - Analyst
: So digging in to the pre-clin data from the two different mouse models in the NHP, can you just walk us through, starting with the human cell data,
how all of this data is building upon itself to make the case of this is going to work, especially as you're getting closer and closer to advancing it
into the clinic?
Question: Keay Nakae - Chardan Capital Markets - Analyst
: Well, great. Yes, I think the whole field is anticipating you guys entering the clinic with that compound. Josh, your second program targets DMD,
and in this case you have the opportunity to build a franchise using, in this case, a morpholino ASO conjugated to the Fab. And obviously, the goal
here is to achieve exon skipping. You are targeting four different mutations starting with 51. And then how should we think about the cadence for
advancing the others in the clinic?
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OCTOBER 05, 2021 / 1:00PM, DYN.OQ - Dyne Therapeutics Inc at Chardan Genetic Medicines Conference
(Virtual)
Question: Keay Nakae - Chardan Capital Markets - Analyst
: Yes. No, I think that last point is really key, Josh. Well, in the last couple of minutes here, let me just quickly ask Oxana to touch on your third program
for FSDH (sic - FSHD) here knocking down the toxic DUX4 mRNA expression. What are the key preclinical findings here, Oxana?
Question: Keay Nakae - Chardan Capital Markets - Analyst
: Okay, great. So Josh, about a year ago you had a successful IPO. You had a successful follow-on. So quickly, your balance sheet, how much cash?
What's the length of the operating runway?
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OCTOBER 05, 2021 / 1:00PM, DYN.OQ - Dyne Therapeutics Inc at Chardan Genetic Medicines Conference
(Virtual)
Question: Keay Nakae - Chardan Capital Markets - Analyst
: So Josh, like you said, you did have a successful IPO. But what do you think investors are still possibly missing most or under appreciating about
the Dyne story?
Question: Keay Nakae - Chardan Capital Markets - Analyst
: Well, great. I'd love to spend more time talking with you today. Oxana will be on our panel later today talking about new delivery technologies for
RNA. And then obviously, as you mentioned, Josh, you'll have your R&D day coming up later this month. So I want to thank both of you for joining
us.
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