Precision BioSciences, a genome-editing company based in Durham, has received authorization from the U.S. Food and Drug Administration to begin a study of its first clinical-stage product candidate, a cancer immunotherapy.
The FDA accepted Precision’s Investigational New Drug (IND) application for PBCAR0191, an allogeneic anti-CD19 CAR T therapy for two types of blood cancer: B-cell acute lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma (NHL).
“Our allogeneic CAR T program has exceeded all expectations, moving from concept to IND acceptance in under three years,” Matt Kane, Precision’s chief executive officer, said in a statement. “We are thrilled to have received authorization to initiate clinical studies for a therapy that we believe could be transformative for patients suffering with NHL and B-ALL.”
Precision’s PBCAR0191 is positioned to be the first gene-edited allogeneic CAR T product candidate in human clinical trials for NHL.
Data provided to the FDA included evidence supporting the elimination of graft versus host interactions, a serious complication of cell-transplant therapies for blood cancers.
Precision said it also provided the FDA manufacturing data supporting the scalability and reproducibility of the T cell genome-editing production process. The company is currently producing clinical trial material at full scale with its manufacturing partner.
Engineering T cells from donors
To fight cancer in a new way, T cells – a type of immune system cell that recognizes invading germs or cancer cells – are engineered to carry a tumor-targeting chimeric antigen receptor (CAR). These engineered cells have the potential to save the lives of many patients unresponsive to traditional chemotherapy and radiation regimens.
Precision’s allogeneic CAR T product candidates use T cells derived from qualified donors. The T cells are manufactured in large batches and are cryopreserved for shipment, storage and off-the-shelf use.
These allogeneic CAR T product candidates rely on Precision’s ARCUS genome-editing platform to remove the T cell receptor in order to prevent graft versus host disease without the need for donor-patient matching. ARCUS editing also enables targeted insertion of the CAR gene into a single, specific location in the T cell genome for more controlled, consistent expression.
Precision is partnering on the PBCAR0191 CAR T therapy with Servier, an international pharmaceutical company based in Suresnes, France, and governed by a non-profit foundation. The independent company, which has a presence in 149 countries and 21,700 employees worldwide, had revenue of over 4 billion euros in 2017.
Servier reinvests 25 percent of its revenue, excluding generic drugs, in research and development and uses all its profits for development. The company focuses on cardiovascular, immune-inflammatory and neuropsychiatric diseases, cancer and diabetes, as well as high-quality generic drugs.
Growth, awards and funding
Precision’s mission is “to cure genetic disease, overcome cancer and feed the planet” using ARCUS, a therapeutic-grade, naturally derived genome editing platform. The company, spun out of Duke University in 2006, has grown to have 132 employees – called “precisioneers” – and has posted openings for 14 jobs on its website.
Earlier this month Precision won recognition at the SEBIO Investor and Partnering Forum in Atlanta for best venture funding deal after securing $110 million in Series B financing this summer.
The Series B financing was led by ArrowMark Partners and included Franklin Templeton Investments, Cowen Healthcare Investments, Brace Pharma Capital, Pontifax AgTech, OCV Partners, Adage Capital Management, Cormorant Asset Management, Gilead Sciences, Vivo Capital, Alexandria Venture Investments, Ridgeback Capital, Agent Capital, and entities affiliated with Leerink Partners. Previous investors venBio, F-Prime, RA Capital Management, Amgen Ventures, Osage University Partners, DUMAC, and the Longevity Fund also participated.
Including the Series B financing, Precision has raised over $270 million through partnerships and private financing rounds.
Also this summer, the company created a new name and brand identity for its food and agriculture business, Elo Life Systems. The business will use Precision’s ARCUS platform and other new technologies for applications in crop improvement, animal genetics, industrial biotechnology and sustainable agriculture.
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