Precision BioSciences Files for $100 Million IPO to Fund Off-the-Shelf CAR-T Trials

Durham, NC-based Precision BioSciences filed on Friday with the U.S. Securities Exchange Commission for an initial public offering (IPO). The company, which focuses on gene-edited CAR-T cell therapies for cancer, plans to raise $100 million.

Precision’s technology is based on its ARCUS tech platform. ARCUS is a fully synthetic enzyme much like a homing endonuclease. A homing endonuclease is a naturally-occurring DNA-cutting enzyme found in the genomes of many eukaryotic lifeforms. ARCUS is an improvement on nature, with high specificity that can be customized to recognize a DNA sequence inside any target gene.

In late-November, the U.S. Food and Drug Administration (FDA) accepted Precision and Servier’s Investigational New Drug (IND) application for PBCAR0191. This is an allogeneic anti-CD19 CAR-T therapy for B-cell acute lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma (NHL). It is Precision’s first clinical-stage product candidate.

The companies claim PBCAR0191 is the first gene-edited allogeneic CAR-T product candidate in human trials for NHL. The IND submission included in-depth analysis of off-target editing data and evidence to support the elimination of graft versus host reactions.

Precision’s CAR-T product candidates use T-cells from qualified donors. They are then manufactured in large batches and cryopreserved in order to be shipped, stored, and used in an off-the-shelf manner.

So far, CAR-T products on the market are engineered and manufactured on a one-on-one basis, unique to each individual patient. That’s extremely expensive and time-consuming. The ability to have a single product available for appropriate patients would be a significant advance in the CAR-T market.

In December, Precision brought Shalini Sharp on to its board of directors. Sharp is the chief financial officer and executive vice president of Ultragenyx Pharmaceuticals.

“I am excited to work with Precision as the company develops new therapies based on its next generation gene editing technology, ARCUS, which has the potential to help patients achieve better outcomes,” Sharp stated at the time. “I have also been impressed by the scaled manufacturing process Precision has developed, which is a critical and sometimes overlooked factor.”

And on January 16, Precision formed its Scientific Advisory Board (SAB). The four appointments to the SAB were Kenneth C. Anderson, program director of the Jerome Lipper Multiple Myeloma Center and LeBow Institute for Myeloma Therapeutics at the Dana-Farber Cancer Institute and Kraft Family Professor of Medicine at Harvard Medical School; Hagop Kantarjian, chair of the Department of Leukemia at The University of Texas MD Anderson Cancer Center; Raymond Schinazi, the Frances Winship Walters Professor of Pediatrics and director of the Laboratory of Biochemical Pharmacology at Emory University; and Cameron Turtle, Associate Member at Fred Hutchinson Cancer Research Center and Associate Professor at the University of Washington.

“We are thrilled to have attracted some of the world’s leading experts in immuno-oncology and infectious disease to join our SAB,” stated Matt Kane, Precision’s chief executive officer. “At Precision, we have found that by committing to excellence, we’ve cultivated a support network of extraordinary people who are as enthusiastic about the possibilities of ARCUS genome editing as we are, especially in areas of significant unmet medical need.”

Precision plans to list its IPO on the Nasdaq under the symbol DTIL.