DTIL - Precision BioSciences 22:53 PM - Aug 16 2021
by: Maverick

Precision BioSciences - DTIL - DD - August 2021

OS: 58M
Float: 52M
Cash: $300M (runway into 2023)

Website:
precisionbiosciences.com


https://www.benzinga.com/pressreleases/21/08/b22462343/precision-biosciences-reports-second-quarter-2021-financial-results-and-provides-business-update

ARCUS Gene Editing: precisionbiosciences.com/our-a...

Catalysts

Precision expects to provide an interim update for PBCAR20A in 2021.
Precision expects to provide an interim update on the monotherapy arm of the study in 2021 (PBCAR269A: Phase 1/2a study of PBCAR269A targeting B-cell maturation antigen (BCMA) for patients with R/R multiple myeloma.)

(Main One - for me)
Gene Editing Event: Precision will host its first in vivo focused gene editing event on Thursday, September 9, 2021. The virtual event will include presentations on the Company's in vivo gene editing business strategy, including pipeline development plans and timelines to the clinic for certain gene editing programs such as its wholly-owned PH1 program. The virtual event is expected to last approximately two hours and will be a live video webcast available through the Company's website. Additional details for the event will follow.

Technology:

Well written article (note it's 2 years old)

fool.com/investing/2019/06/11/...

It did have an important note of caution:

Investors should also know that the ARCUS platform shares one significant drawback with CRISPR systems: it works by cutting DNA. While that's kind of the point behind first-generation gene editing, making it possible to cut out faulty DNA and even insert new genetic material, it also triggers natural DNA repair mechanisms that can lead to complex side effects. Researchers are beginning to think that will make cutting tools poor options for directly editing cells in the body (called "in vivo"), which is why next-generation gene editing platforms avoid cutting altogether.

Buried note about human trials coming up?

investor.precisionbiosciences....

“This is the first time ARCUS has been used to edit outside the nuclear genome and has done so with encouraging safety and efficacy in this mouse model,” said Derek Jantz, Ph.D., co-author of the paper and Chief Scientific Officer at Precision BioSciences. “We continue to see promising results in preclinical studies suggesting that ARCUS could potentially effectively edit mutant mtDNA in vivo in human clinical trials. I congratulate Carlos and his team on this research and look forward to further work on this program.”

The good/bad of all gene editing tech is still very fluid, new and up for debate:

twitter.com/Chipoo86/status/14...

The reason I'm buying/holding:

healio.com/news/hematology-onc...

“I jokingly call it CRISPR 2.0,” Shah told Healio. “The ability to carry this payload of genetic information [and] put it where you want it is a huge plus. Moving forward, this is not going to be restricted to CAR T-cell therapy.”

“The best way to think about it is to imagine a more efficient form of CRISPR,” Shah said. “ARCUS, like CRISPR, can selectively drop a DNA message wherever you want to place it.”

The platforms diverge in the tool that is used to deliver information about where to cut and deliver the genetic payload, Shah said.

Another strength of ARCUS is its ability to deliver multiple payloads of genetic information compared with CRISPR, Shah said.

The platform allows researchers to make all alterations required in a gene-edited CAR T cell with the same tool — and all in one step. It allows them to do disrupt or knock out genes, and it also allows for the delivery of new genetic information, Shah said. The single-step gene editing allows for significant improvements in efficiency, he added.

Read the entire article.

Fun fact:  The ticker DTIL stands for "Dedicated To Improving Life"

Summary:
Two Phase 1/2 data updates by end of year (multiple myleoma and CLL)
Gene editing event on September 9, 2021
I'm expecting them to release some pretty lofty pipeline plans for gene editing at the event and a nice run in the days preceding the event.  I might hold a bit through the event since it's not a data release, but more of a corporate pipeline update.

I jumped in too early on this one (11.29 average right now), but I think I'll be ok in a few weeks.
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