Precision BioSciences Inc at Stifel Tailoring Genes: Genetic Medicines Day (Virtual) Transcript

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

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Excellent. Okay. Well, I think I want to dive into each one of those programs. But I think to start, can you talk about where ARCUS came from? And I guess, specifically, why is this better than CRISPR cash or some other [algorithm] that might be out there?

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Fantastic. Okay. Just a couple more questions just on the technology. So I guess, what is known -- you talked about specificity a little bit her. Is the off-target profile and the specificity profile, is that different for each candidate, or is that intrinsic within this class of editing? And then the other thing I wanted you to touch on if possible is how easy is it to manufacture ARCUS? Is it more or less similar to a small CAR T manufacturing?

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Excellent. There's a couple of programs that we'll talk about. But I guess as you think about putting a package together for the FDA liquidity, given that this is a unique editor relative to maybe some of the packages that they're seeing, what do you need to put together? Like, what does the FDA ask in terms of an IND and getting this okay in people? Is it all about specificity and -- yeah.

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Okay. Very cool. Let's start with the hemophilia B program. I think you guys have made some progress on this recently. Maybe first off, what is -- what do you see as the role of gene editing in hemophilia B? And I guess maybe another way to ask this is why haven't these existing drugs solve this challenge so far?

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Hepatitis B. Thank you.

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Let me ask that again. So I guess regarding the hepatitis B program. So what do you see as the role for gene editor in the treatment landscape?

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Excellent. This program has been in the works for few years now. I guess what is -- has its recent inflection point? I guess what's driving the momentum?

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Excellent. Okay. I feel like a lot of us are pretty familiar with hepatitis B and this treatment landscape. But could you maybe just talk about the hepatitis B surface antigen. What level do you need to get that down to be excited and what editing efficiency gets you to that level?

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Excellent. Okay. And this is maybe early, but do you have a sense for -- is there a segment of the patient population you want to focus on initially in the clinic? And do you have a sense for the timeframe for moving towards the clinic?

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Okay, fantastic. Next question I want to ask you about is about durability. This is broadly, I think, applicable to all the genes -- in vivo gene-editing programs. But how do you think about durability in that? You did get a lot of interesting data in non-human primates I think maybe a couple of different disease setting. But talk about what you're seeing in durability and your expectations for that.

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Great. Okay. Okay, fantastic. So it's more than five years -- at least five years in non-human primates thus far.

Question: Ben Burnett - Stifel Financial Corp. - Analyst : I want to come back to this with regards with the DMD program. But I think, Cassie, we had talked about in the past the importance of getting it deployed to potent cells as well to ensure that durability. But real quick, though, before we do that, you have a couple of different collaborations. Walk us through the Novartis collaboration. What are the unique challenges of editing a hematopoietic stem cell?

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Okay. Awesome. In the last couple of minutes, let's talk about the Duchenne muscular dystrophy program. I guess from a technology perspective, how does this differ from the other gene editors that you have?

Question: Ben Burnett - Stifel Financial Corp. - Analyst : That's great. You touched one of the important things I wanted to ask you about. The muscle is a different --it's different tissue, but there's similar -- there's different groups of muscles that you would need to target cardiac, skeletal, for example. What are you seeing in your preclinical data? And what are your expectations for targeting the various muscle tissues that are in the body?

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Okay. That's great. That's exactly what I'm going to ask, the level of editing that's needed. I do want to ask in the last minute or two, there's a lot of programs, there's lot of versatility with the ARCUS platform. I guess, Michael, how do you allocate resources and time to various programs? But

Question: Ben Burnett - Stifel Financial Corp. - Analyst : Okay, okay. Well, that's great. Thank you, guys, very much for the conversation. I think we're right at time. So we'll end the panel.