Precision BioSciences

Precision BioSciences, Inc.

Company type	Public
Industry	Biotechnology
Founded	March 2006 (2006-03)
Founder	Derek Jantz, Jeff Smith, Matt Kane
Headquarters	Durham, North Carolina , United States
Key people	Michael Amoroso (President and CEO)
Products	Gene editing treatments using ARCUS genome editing technology
Website	www.precisionbiosciences.com

Precision BioSciences, Inc. is a clinical-stage gene editing firm based in Durham, North Carolina, publicly listed in the United States. Established in 2006, the company specializes in developing both in vivo and ex vivo gene editing treatments by utilizing its exclusive "ARCUS" genome editing technology.

History

Precision BioSciences was established in March 2006 by Derek Jantz and Jeff Smith, who were postdoctoral researchers at Duke University, along with Matt Kane, a student at Duke's Fuqua School of Business. The company initially raised capital through a Series A funding round led by venBio to support the development of its genome editing technology, followed by a Series B round aimed at advancing its pipeline. Precision went public in 2019 and is traded on the Nasdaq exchange under the symbol DTIL.

In November 2020, Precision entered into a collaboration with Eli Lilly to apply its ARCUS platform to as many as six in vivo genetic disorder targets, beginning with Duchenne muscular dystrophy. By September 2021, the company had formed partnerships with UK-based Tiziana Life Sciences to investigate foralumab for chimeric antigen receptor (CAR) T cell therapy applications, and with Philadelphia-based iECURE to progress liver disease programs and clinical studies utilizing ARCUS technology. Michael Amoroso, previously CEO of Abeona Therapeutics, was appointed President and CEO in October 2021. In December of that year, Precision agreed with investors led by ACCELR8 to spin off its Elo Life Systems subsidiary into an independent entity focused on food and agriculture.

ARCUS genome editing

The proprietary ARCUS platform and ARCUS nuclease enzymes developed by Precision BioSciences are derived from the naturally occurring genome editing enzyme I-CreI, a homing endonuclease found in the algae Chlamydomonas reinhardtii. These nucleases facilitate precise DNA cleavage and insertion events while uniquely self-inactivating post-editing to minimize off-target activity. Compared to CRISPR spCas9, ARCUS nucleases are more compact, enabling delivery via single adeno-associated virus (AAV) vectors or lipid nanoparticles (LNPs).

Precision utilizes ARCUS to create multiple ex vivo allogeneic "off-the-shelf" CAR T cell therapies currently undergoing early clinical evaluation, alongside several in vivo gene editing projects in preclinical development. The ARCUS nucleases generate distinctive DNA breaks with four-base-pair 3' overhangs and support complex modifications such as gene insertion, deletion, and repair—all achievable in one step to enable efficient multiplex editing.

The company has demonstrated ARCUS’s application in treating ornithine transcarbamylase deficiency in newborn nonhuman primates and targeting chronic Hepatitis B through LNP delivery. Its pipeline includes PBGENE-PCSK9 for familial hypercholesterolemia and PBGENE-PH1 addressing primary hyperoxaluria type 1. Collaborations like the one with iECURE bolster Precision’s capacity for advancing gene editing therapeutics.

Clinical trials

Precision is advancing several candidates aimed at treating non-Hodgkin lymphoma, acute lymphoblastic leukemia (ALL), and multiple myeloma. Its lead program, PBCAR0191 targeting CD19, has received orphan drug designation from the U.S. FDA for ALL and mantle cell lymphoma indications, as well as fast track status for B-cell ALL. Initiated in March 2019, Phase 1/2a trials of PBCAR0191 reported by June 2022 a complete response rate of 73%, an overall response rate of 100%, and a durable response rate of 50%, leading to plans for expanded patient enrollment.

The firm is also progressing PBCAR19B, an anti-CD19 stealth cell candidate engineered with a single gene edit to reduce beta-2 microglobulin expression; Phase 1 trials commenced in June 2021. Additionally, Precision is conducting Phase 1/2a studies for PBCAR269A, an allogeneic CAR T therapy targeting B-cell maturation antigen for multiple myeloma treatment. This candidate began Phase 1 testing in April 2020 and is currently being evaluated alongside nirogacestat, a gamma secretase inhibitor. The FDA has granted both fast track designation and orphan drug status for PBCAR269A in relapsed or refractory multiple myeloma.

These clinical efforts underscore Precision’s strategic emphasis on developing off-the-shelf CAR T therapies designed to capture market opportunities within hematologic cancers through advanced gene-editing platforms combined with accelerated regulatory pathways.