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: Maury Raycroft - Jefferies Group LLC - Analyst
: So maybe to start off, if -- for those who are new to the story, if you can provide a one-minute intro into Precision.
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. And I think that's a good overview. And Michael, maybe if you can give us some perspective on why you decided to join Precision and what
your takeaways are after your first 10 days on the job.
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. That's great. Should we expect any changes in strategy or focus going forward?
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it.
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. And a question came in for you, Michael, too. What do you see as your first and most important order of business as CEO?
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. And so maybe if you guys could deep dive into your ARCUS gene editing technology and talk about some of the key preclinical data you
have reported that investors should know about, particularly, data that helps investors understand differentiation versus other gene editing
approaches.
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. That's really helpful. And we just had a conversation with an expert in the space and he talked about the advantages of nucleases that aren't
around for a long period of time. And Michael, I think you alluded to this a little bit, too. For your ARCUS enzyme, how does that work?
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Interesting. And maybe for your lead in vivo program for PCSK9, can you recap your strategy and timelines for that program and talk about how
your approach is differentiated from Verve and others that are pursuing this target?
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. And that makes sense. And your technology was recently validated by a research collaboration with Eli Lilly. Can you talk about the progress
so far on that in DMD, and any other status updates on the other targets at this stage? And can you say anything about timelines as well?
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. Okay.
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Okay. And then, after your PCSK9 program, you have identified PH1 as the next in vivo program that you are going to advance into the clinic. What
delivery are you pursuing for that one and how do you see this therapy in comparison to Alnylam's approach for PH1?
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. Okay. And earlier this year, you announced a publication, showing for the first time that ARCUS has been used to edit outside the nuclear
genome targeting mutant mitochondrial DNA in vivo. And this showed encouraging safety and efficacy in a mouse model. Can you elaborate on
a -- further work that needs to be done to advance this program into the clinic?
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. But a lot of potential with that technology and that approach.
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. And also wanted to ask about how Intellia's clinical data that they reported recently where they used CRISPR/Cas9 in LNPs, in ATTR. How
that has informed your in vivo development strategy?
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. Okay. And you mentioned the iECURE collaboration earlier. Can you talk a little bit more about the gene insertion data that Dr. Jim Wilson
shared during your gene editing event? What was the target and how efficient was the editing with the market?
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. And maybe 15 years to your off-the-shelf CAR T programs, which we have followed pretty closely over the last couple years -- maybe talking
about the CD19 one first and just wondering if you plan on sharing some additional data this year for that program.
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Got it. And maybe last question: you guys have the in vivo programs and a technology potential there, and then also the clinical programs, and
several INDs planned out over the next couple years. I guess what's the market missing with the stock?
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OCTOBER 27, 2021 / 5:30PM, DTIL.OQ - Precision BioSciences Inc at Jefferies Gene Therapy/Editing Summit
(Virtual)
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Great. Well, Michael, Derek, thanks so much for joining us today. Great speaking with you both.
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