Precision BioSciences

Derek Jantz and Jeff Smith met as postdoctoral fellows at Duke University,[5] and in March 2006, they founded Precision BioSciences along with Matt Kane, a student at the Duke Fuqua School of Business at the time.[3] The company went through two rounds of early funding: a Series A round led by venBio to fund development of the genome editing platform,[6] and Series B financing to fund product development efforts.[7][8] The company completed its initial public offering in 2019, and trades under the Nasdaq ticker DTIL.[9][10]

Precision entered into a partnership with Eli Lilly in November 2020 to use ARCUS editing for up to six in vivo targets connected to genetic disorders,[11] beginning with Duchenne muscular dystrophy.[12] In September 2021, Precision announced two more collaborations, with UK biotechnology company Tiziana Life Sciences to explore using foralumab to aid chimeric antigen receptor (CAR) T cell therapy,[13] and with Philadelphia-based iECURE to advance candidates into clinical trials and investigate how ARCUS can help treat liver diseases.[11] Michael Amoroso, the former CEO of cell and gene therapy developer Abeona Therapeutics, succeeded Matt Kane as President and CEO in October 2021.[3] That December, Precision announced its entry into an agreement with a syndicate of investors led by ACCELR8 to spin off its subsidiary, Elo Life Systems, and create an independent company focused on food and agriculture business.[14]

Precision BioSciences' proprietary technology is the ARCUS platform and ARCUS nucleases.[4][8] ARCUS nucleases are based on a naturally occurring genome editing enzyme, I-CreI, a homing endonuclease that evolved in the algae Chlamydomonas reinhardtii[4][12] to make highly specific cuts and DNA insertions in cellular DNA.[15] The nuclease is able to deactivate itself once gene edits are made, which minimizes potential off-targeting.[12][16] An ARCUS nuclease is also much smaller in size than CRISPR spCas9.[17] It can use either adeno-associated virus (AAV) vectors or lipid nanoparticles (LNPs) for delivery to specific tissues and cells.[18] Precision has used ARCUS nucleases to develop multiple ex vivo allogeneic, "off-the-shelf" CAR T cell immunotherapies in early-stage clinical trials.[4][19] The company also uses ARCUS for in vivo gene editing programs,[4] some of which are in preclinical development as of May 2022.[20][18]

Similar to I-CreI, ARCUS nucleases generate a unique cleavage site in DNA that is characterized by four-base-pair, 3' overhangs.[4] ARCUS nucleases can perform a range of complex edits, including gene insertion, gene excision, and gene repair.[8][15] ARCUS nucleases are able to enact all editing operations in one step, which enables efficient multiplexing of edits.[19]

Precision has demonstrated some additional applications of the ARCUS platform, including treating ornithine transcarbamylase deficiency in newborn nonhuman primates and in the use of a LNP to treat chronic Hepatitis B.[15] The company is also pursuing PBGENE-PCSK9, a candidate to treat familial hypercholesterolemia, and PBGENE-PH1, a candidate to treat primary hyperoxaluria type 1.[21][22]

Precision is in the process of developing multiple candidates targeting non-Hodgkin lymphoma, acute lymphoblastic leukemia (ALL),[4][19] and multiple myeloma.[23] The company's lead candidate targeting CD19, PBCAR0191,[19] received orphan drug designation from the U.S. Food and Drug Administration for the treatment of ALL and mantle cell lymphoma, an aggressive subtype of non-Hodgkin lymphoma, as well as fast track designation for the treatment of B-cell ALL.[23] PBCAR0191 began its Phase 1/2a clinical trial of adult subjects in March 2019.[24][23] In June 2022, Precision reported a 100% response rate, a 73% complete response rate, and a 50% durable response rate, and the company sought to increase enrollment in the study.[25][26]

Precision is also developing PBCAR19B as an anti-CD19[26] stealth cell candidate that employs a single gene edit to knock down beta-2 microglobulin, for which a Phase 1 study began in June 2021.[27][28] The company is also conducting a Phase 1/2a clinical trial evaluating PBCAR269A, its investigational allogeneic B-cell maturation antigen-targeted CAR T cell therapy, for the treatment of multiple myleloma.[26] PBCAR269A began its Phase 1 trials in April 2020,[29] and as of July 2022 had moved onto recruitment for its Phase 1/2a study, which features PBCAR269A in combination with nirogacestat, a gamma secretase inhibitor.[26] In 2020, the FDA granted fast track designation to PBCAR269A for the treatment of relapsed or refractory multiple myeloma, having previously provided orphan drug designation.[23]

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