Precision BioSciences Doses First Patient in Phase 1 Allogeneic CAR T Clinical Trial of PBCAR19B Immune Evading Stealth Cell for Relapsed/Refractory Non-Hodgkin Lymphoma
“We are excited to have dosed the first patient in our Phase 1 trial of PBCAR19B, a next-generation, CD19-targeted candidate incorporating our immune evading stealth cell technology. This is our fourth CAR T program in the clinic, demonstrating the modularity of ARCUS for allogeneic CAR T therapies,” said
The Phase 1 study will be a non-randomized, open-label, single-dose, dose-escalation and dose-expansion study designed to evaluate the safety and clinical activity of PBCAR19B at flat dose levels beginning at 2.7 × 108 with the ability to dose up to 8.1 × 108 CAR T cells per patient, following standard lymphodepletion1 in patients with R/R NHL. The first dose level of PBCAR19B is comparable to dose level 3 of the PBCAR0191 CAR T. The primary objective of the study is to identify the maximum tolerated dose and any dose-limiting toxicities. Clinical trial material for this study is generated at the Company’s in-house
About PBCAR19B (Clinical Trials Study Identifier: NCT04649112)
PBCAR19B is a next-generation, immune evading stealth cell candidate for patients with CD19-positive malignancies such as R/R NHL. PBCAR19B is designed to improve the expansion and persistence of allogeneic CAR T cells following infusion by reducing rejection by T cells and NK cells. In addition to the CAR gene, the PBCAR19B stealth cell vector carries a short hairpin RNA that suppresses expression of beta-2 microglobulin, a component of
About Precision’s Allogeneic CAR T Platform
Precision is advancing a pipeline of cell-phenotype optimized allogeneic CAR T therapies, leveraging fully scaled, proprietary manufacturing processes. The platform is designed to maximize the number of patients who can potentially benefit from CAR T therapy. Precision carefully selects high-quality T cells derived from healthy donors as starting material, then uses its ARCUS genome editing technology to modify the cells via a single-step engineering process. By inserting the CAR gene at the T cell receptor (TCR) locus, this process knocks in the CAR while knocking out the TCR, creating a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease. Precision optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of naïve and central memory CAR T cells throughout the manufacturing process and in the final product.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development of PBCAR19B, the potential clinical benefit of our allogeneic CAR T product candidates and the further development of our ARCUS platform. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “believe,” “could,” “expect,” “should,” “plan,” “intend,” “estimate,” “target,” “mission,” “goal,” “may,” “will,” “would,” “should,” “could,” “target,” “potential,” “project,” “predict,” “contemplate,” “potential,” or the negative thereof and similar words and expressions.
Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators’ ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the
All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
1 Standard lymphodepletion = Fludarabine (30 mg/m/day for 3 days) plus cyclophosphamide (500 mg/m2/day for 3 days)
Chief Financial Officer
Senior Director, Corporate Communications