Precision BioSciences, a Durham-based genome editing company, has reached two regulatory milestones for its potential therapy against multiple myeloma, a chronic cancer of white blood cells.

The U.S Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application and also granted the therapy Orphan Drug Designation, a status that gives companies tax reductions and other incentives to develop treatments for rare diseases.

The therapy, designated as PBCAR269A, is Precision’s third allogeneic chimeric antigen receptor (CAR) T cell therapy candidate.

“FDA acceptance of the IND for PBCAR269A further underscores the ongoing progress in our allogeneic CAR T pipeline,” said Matt Kane, co-founder and chief executive officer of Precision BioSciences. “We have now moved three CAR T programs from preclinical to clinical stage development since April 2019, and we look forward to continuing to advance our allogeneic CAR T portfolio to bring these novel therapeutic candidates to patients.”

Precision Bio photo

Matthew Kane

The company plans to begin a Phase 1 clinical trial of the therapy this spring at multiple sites using material produced at its own manufacturing facility in Durham. About 48 patients are expected to be enrolled.

For more information about the trial, visit, and enter study identifier number NCT04171843.

“In preclinical disease models, PBCAR269A has demonstrated no evidence of graft-versus-host disease at doses that resulted in potent anti-tumor activity,” said Chris Heery, M.D., chief medical officer of Precision BioSciences. “There remains significant unmet need in the treatment of relapsed/refractory multiple myeloma, and we are excited to begin clinical trials with an off-the-shelf CAR T therapy candidate in this setting.”

The IND for PBCAR269A builds on the initial clinical data Precision presented in late 2019 for its lead program, a CAR T therapy for treating non Hodgkins lymphoma and B-cell acute lymphoblastic leukemia, and the FDA’s acceptance of an IND for another CAR T therapy for treating non-Hodgkin’s lymphoma, chronic lymphocytic leukemia and small lymphocytic lymphoma.

Technology exploits T cells

Precision BioSciences harnesses T cells, a type of white blood cell that is vital to the adaptive immune system’s ability to identify specific antigens and destroy pathogens. Through the company’s CAR T technology, the T cells can be directed to kill cancer cells.

Precision produces the CAR T therapies by selecting T cells derived from healthy donors as starting material. Then, using its proprietary ARCUS genome-editing technology, the company modifies the donor T cells.

Scientists insert the CAR gene at the T cell receptor locus, enabling the T cell to target a specific marker on a cancer cell, while knocking out the T cell receptor to prevent the patient’s immune system from recognizing and attacking the T cells.

The company “optimizes” its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of certain types of CAR T cells throughout the manufacturing process and in the final product.

The process creates a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease, normally a major challenge when inserting foreign or altered cells or tissues into the body.

The company has posted a four-minute video on its website to explain CAR T therapy, using Samurai warriors as an analogy.

New manufacturing center open


Last July Precision opened its Manufacturing Center for Advanced Therapeutics (MCAT), the first in-house current Good Manufacturing Process (cGMP)-compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf chimeric antigen receptor CAR T cell therapy products.

Precision Bio photo

Precision Bio facility

“Given the potential output of our platform, we’ve known from the beginning that it was critical for us to address the need for scalable manufacturing of cell-therapy products in order to be able to effectively deliver them to patients,” Kane said when the facility opened. “In addition to our clinical work, it also has the potential to be a commercial launch facility with the capacity to generate up to 10,000 doses of CAR T cell therapies and 4,000 doses of gene therapies per year.”

The facility can produce three different drug substances: allogeneic CAR T cells, messenger RNA and adeno-associated viral vectors. It was designed to meet regulatory requirements in the United States, Europe and Japan.

Broader applications for ARCUS

In addition to health care, Precision’s ARCUS genome-editing platform has applications in food and agriculture.

In 2018 the company created a new name and brand identity, Elo Life Systems, for its food and agriculture business, previously known as Precision PlantSciences, based in Research Triangle Park.

Elo is using the ARCUS platform and other new technologies for applications in crop improvement, animal genetics, industrial biotechnology and sustainable agriculture.

Since it was spun out of Duke University in 2006, Precision raised about $300 million in venture capital, government grants and collaboration agreements. The company went public in March 2019, grossing $145.4 million in an initial public offering of stock.

The company’s shares are listed on the Nasdaq Global Select Market under the ticker symbol “DTIL,” shorthand for the company’s marketing tagline, “Dedicated to improving life.”

(C) N.C. Biotech Center