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Precision Biosciences Overview

|Includes: Adaptimmune Therapeutics plc (ADAP), ATRA, CELG, CLLS, CRSP, EDIT, GILD, NTLA, NVS, SGMO
Summary

Precision Bio is a private gene editing company who just raised $110m.

Baxalta, a Shire company has a collaboration agreement with Precision for allogeneic CAR-T cell therapy.

Baxalta is now part of Shire who has increasingly shown signs of prioritizing gene therapies.

Precision signed an oncology allogeneic CAR-T collaboration with Baxalta worth up to $1.7b.  Baxalta was acquired by Shire in 2016 which puts this partnership in the Shire family.

Precision just raised a B round of $110m.  Gilead (GILD) was a new investor.  Amgen ventures continued their participation.  The funding will allow them to take their lead off the shelf cell therapy product into the clinic and advance their lead in vivo gene therapy program into IND-enabling studies.

Precision come out of Duke University.  Their conmpany statement:

Precision BioSciences is dedicated to improving life. Our mission is to cure genetic disease, overcome cancer, and feed the planet. We are striving to achieve this goal with ARCUS, our therapeutic-grade, naturally-derived genome editing system that combines both specificity and efficacy to help overcome life’s greatest genetic challenges. For additional information, please visit www.precisionbiosciences.com

Their ARCUS gene editing platform is based upon a synthetic homing endonuclease.

Homing endonucleases

Homing endonucleases are nature’s genome editing system. They are site-specific DNA-cutting enzymes encoded in the genomes of many eukaryotic species. Homing endonucleases have the unusual ability to precisely recognize long DNA sequences (12-40 base pairs) that are typically rare enough to occur only once in a complex genome. These non-destructive enzymes trigger gene conversion events that modify the genome in a very precise way, most frequently by the insertion of a new DNA sequence. The ability to target a single DNA break in a complex genome and to achieve gene modification without random off-targeting makes homing endonucleases the ideal starting material for a therapeutic-grade genome editing technology.

The single DNA break contrasts with the double strand break used by CRISPR, TALENs and ZFN which have been been the subject of concerns relating to the p53 gene.