Precision BioSciences Inc JonesTrading Virtual Genetic Medicine Day Transcript

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

Question: Soumit Roy - JonesTrading - Analyst : So starting with Prime Medicine, one of the latest versions of gene editing, Allan, if you could provide us a simplified explanation of how prime editing works. And curious, do you spend a lot of time educating the investors on the mechanistic differences and the scope of your reach that has really expanded what base editing has shown?

Question: Soumit Roy - JonesTrading - Analyst : Thank you for touching on the bystander mutations. And we -- there is still an education process going on among the investors. And trying to understand have you -- is it just mechanistically impossible with your prime editing to have bystander exhaustively tested, looked upon [seekingly]?

Question: Soumit Roy - JonesTrading - Analyst : Picking the right indication, looking through the land itself, enrollment, marketize, path to registration seems to be the key deciding factor for success of a genetic company. You can't really run a basket tumor like oncology indications can do it. To Mike, however, you can answer the question. One is you have the four main mechanisms through which editing can work and you have the four products in there. You have insertion for OTC, editing for the mitochondrial disease, degrading the viral DNA for the lead program, chronic HPV, and excising DNA of a DMD asset there. How do you decide on the strategy? What should be the next asset to wholly develop versus maybe partner this out, outlicense that? And if you want to also address compare contrast between other editing technologies and how your stacks up. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 25, 2024 / 4:00PM, DTIL.OQ - Precision BioSciences Inc JonesTrading Virtual Genetic Medicine Day

Question: Soumit Roy - JonesTrading - Analyst : DMD space? That has really picked up in the last couple of months and huge investment interest RNA showing durable data. It got acquired Kate Therapeutics by Novartis and you have an asset. I'm thinking -- are you thinking about anything prioritizations, outlizing, what is?

Question: Soumit Roy - JonesTrading - Analyst : We're looking forward to your next move in that. Turning to Gabe and being iECURE being one of the leading companies in vivo-editing space and the only company to edit in neonatal. One is, what are your plans to monitor in the long term follow up to remove any concerns from the off-target edits? And second really is, we would love to get your perspective and also from Allan and Mike, the difference between dealing with ex-US and US authorities investigate other parents. What are the key questions you end up handling?

Question: Soumit Roy - JonesTrading - Analyst : I would love to get a sense like what's your sense from talking to US or ex-US, if there is any difference? What time period of data and safety they want to see before they actually approve this study? It's going to be two, three years, five years? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 25, 2024 / 4:00PM, DTIL.OQ - Precision BioSciences Inc JonesTrading Virtual Genetic Medicine Day

Question: Soumit Roy - JonesTrading - Analyst : Great. Thank you for that. Probably a question for Allan and Mike. Like to get any comments, like most often times we see CTS coming out before INDs. And curious to hear to provide any color on the FD interaction. What are they looking for in the IND package your experience? Compare contrast European Australian versus the US?

Question: Soumit Roy - JonesTrading - Analyst : Moving into the pipeline programs and really want, don't want to miss those to Allan on prime editing for Prime Medicine, CGD your lead asset, the lead, this indication. If you can share any color on the enrollment front, what are the key things you are -- challenges could be there/ And one this is being ex-vivo and taking some of the learnings from [Cas-KV] and editors on number of cycles you need for sale mobilization. That seems to be really, it could be three and four or it could be four to six and that could eat up six months, medium time to engraftment, especially in CGD cases. What of the key things that you're thinking of and --?

Question: Soumit Roy - JonesTrading - Analyst : I'm curious about your thinking, myeloablation and its associated risks even gentler mylo ablation, the one that Vanderbilt came out in the previous session we were discussing about it. Even though it's a gentler, it's still with a lot of risk. And that is turning a lot of companies towards the in-vivo approach, whether targeted LNP, whether virus like particles without any viral genome in it, just with capsids, you have X-CGD, new next asset coming out. Any thoughts moving towards the in vivo approach and completely circumvent the myeloablation, the mobilization, all these aspects?

Question: Soumit Roy - JonesTrading - Analyst : We'll see how Beam and CRISPR they are targeting the C117 and see how that turns out. Wilson's disease, Prime's next major push towards clinic. If you can just describe the approach for the disease. And primarily from the point of view of companies like Ultragenyx and Vivet Therapeutics, they are doing gene replacement. So they are just taking out putting in the X new gene, and that takes care of these multiple point mutations versus monogenic approach, which has been in CGD. And now you're expanding it to X-CGD. Is that going to be the approach for Wilson? Are you going to --?

Question: Soumit Roy - JonesTrading - Analyst : Looking forward to the IND CTL filing in 2026 for the Wilson?

Question: Soumit Roy - JonesTrading - Analyst : Turning to Mike. Chronic HPV, your lead asset, huge market. Really curious, one question is in case of an infectious disease, how are you thinking -- how does it, you're thinking this will your product will completely eradicate the chronic infection? It's going into the liver, large organ, LNP permeates to a certain cell depth. How does it work?

Question: Soumit Roy - JonesTrading - Analyst : So this viral particles are there like in 80% 90% of the liver cells. So it's really pockets of liver cells that the viral particles.

Question: Soumit Roy - JonesTrading - Analyst : What's the plan there? You want to get some of the clinical data from the European countries and then file the IND here or it's --?

Question: Soumit Roy - JonesTrading - Analyst : Any guidance on enrollment rate, data we expect --?

Question: Soumit Roy - JonesTrading - Analyst : Great. Turning to Gabe, OTC and pediatric and neonatal patients again. Expecting major initial state of the set of data first quarter or first half of '25 with ARCUS's platform editing platform. You already walked us through the initial natural history of the OTC patients, trying to understand at what stage do you expect most of these patients to opt in there? Just before liver transplantation, it increases the threshold to show clinical benefit.

Question: Soumit Roy - JonesTrading - Analyst : Do you find it easier to convince a parent of a neonatal or kid versus an adult.

Question: Soumit Roy - JonesTrading - Analyst : Find it easier to convince a parent like a newborn can be treated or is it an adult patient?

Question: Soumit Roy - JonesTrading - Analyst : So how many patient data are we going to see or first type or it's going to be --?

Question: Soumit Roy - JonesTrading - Analyst : Good luck. And thank you again. Last question for both Allan and Mike. Really, we're getting any thoughts on the RNA medicine space really, emerged as a key competitor and a lot of investors like it because it's kind of a drug like qualities. First, it's transient in nature. You can keep dosing, which I see negative but investors say, okay, these are long term safety concerns are not there; lower cost, like several folds, lower cost. When you talk to investors, how do you make your case to invest in Prime or ARCUS-based editing versus RNA companies Wave and Korro types?

Question: Soumit Roy - JonesTrading - Analyst : Mike.

Question: Soumit Roy - JonesTrading - Analyst : No, that's great. Thank you again, everyone for joining the panel and we're really looking forward to 2025. We have major data coming out from all three companies here; hopefully transformative. Our next panel will be with Dr. Kevin Alexander from Stanford University discussing ATTR space and [Intelius] data. Thank you again for joining us.