Dyne Therapeutics Inc at Morgan Stanley Global Healthcare Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Jason Russell - Morgan Stanley - Analyst : <_ALACRA_META_ABSTRACT>But first, let's just do a quick refresher on the platform. Josh, maybe you just start back with the FORCE platform, you've got some competitors and what I'll call the antibody directed oligo space approaches vary, can you just help elucidate for us your approach. Why is it different? Why do you feel like it's a very unique platform that gives you advantages for your candidate?

Question: Jason Russell - Morgan Stanley - Analyst : Great. Maybe just a couple of follow ups. So TfR1 or transferrin receptor, what's the typical role of that in normal biology? You're almost hijacking it with your approach for the FORCE platform, so just baselines for our audience.

Question: Jason Russell - Morgan Stanley - Analyst : Great. And Wildon, then maybe just a follow up because it has a role of managing iron uptake, what are the concerns if any, from a safety, tolerability perspective as you prepare to give us your first clinical data here around the corner?

Question: Jason Russell - Morgan Stanley - Analyst : Okay, great. Well, obviously encouraging to hear that so far, so good. And yeah, I think that's a great transition to your, I'll say, one of your lead programs from myotonic dystrophy 1 or DM1, you've got DYNE-101 to address. So let's start with DM1, many people may not be familiar with it, but it's actually a huge disease, no therapies, just touch on the disease for a minute, Josh.

Question: Jason Russell - Morgan Stanley - Analyst : Okay. A couple of follow-ups there, you've covered off some things I was planning plan to cover. Maybe just going to back to the population really quick. You touched on some numbers characterizing that population, what are the analogs for that population? I mean, what's something that -- DMD is something we're all familiar with and there have been therapeutics, but where does DM1 sit?

Question: Jason Russell - Morgan Stanley - Analyst : Those are big numbers.

Question: Jason Russell - Morgan Stanley - Analyst : Okay. And then just on the -- sticking on the patient population for a moment, identification of these patients, I mean, again, take DMD, as an example, identified young, these patients, I believe are adults when they're identified, but there's the family relationship. How does that all work?

Question: Jason Russell - Morgan Stanley - Analyst : Great. And maybe last one on DM1 and then we can move on just on the patients again, is there a big registry of these patients, is there big foundations that you work with and have relationships with?

Question: Jason Russell - Morgan Stanley - Analyst : Okay, great. And I assume that was not my last question on DM1. One more if I could. You talked about some of the data that we're going to see, I guess two-part question, one, I believe the public guidance, it's second half of this year, any further tightening to that or that's what it is right now?

Question: Jason Russell - Morgan Stanley - Analyst : Okay, will leave it there. And then on the types of information, we're going to get splicing. Help contextualize for us what good looks like. And I know you might not be setting exact benchmarks out there, but just help frame how investors should start to think about the data that you're going to be putting out.

Question: Jason Russell - Morgan Stanley - Analyst : And I think the safety and tolerability points are going to be really important one, just given some of the other developments in the space. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 11, 2023 / 8:15PM, DYN.OQ - Dyne Therapeutics Inc at Morgan Stanley Global Healthcare Conference

Question: Jason Russell - Morgan Stanley - Analyst : That's great. Thank you. Let's shift gears to your next program. I would venture to say on a similar timeline, DYNE-251 for DMD patients. These are exon 51 or patients amenable to exon 51 skipping. Before we jump to the study, there are a number of exon-skipping therapeutics. You get this question all the time. There's a gene therapy. There are other companies. So talk to us about the need here with these [boys]. Why is this program interesting?

Question: Jason Russell - Morgan Stanley - Analyst : Okay, great. And I think you largely touched on this, but for the data that will come at the end of the year, anything else you -- frame that data, how we should expect that data in the year.

Question: Jason Russell - Morgan Stanley - Analyst : And no safety tolerability issue so far that you've observed in this trial.

Question: Jason Russell - Morgan Stanley - Analyst : Okay. Wonderful. So a couple of minutes left, you've obviously got a lot on your plate and your focus is where it should be for the rest of the year. But let's fast forward a little bit from, say 101, 251, both, you know, hit or surpassed your expectations, so a great place to be, what's next? Give us a head of where you might go and where the more next steps would be.

Question: Jason Russell - Morgan Stanley - Analyst : Amazing; exciting times. Any closing remarks. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 11, 2023 / 8:15PM, DYN.OQ - Dyne Therapeutics Inc at Morgan Stanley Global Healthcare Conference