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Precision Bio Lands $110M as Gene-Edited Cell Therapy Nears Clinic

Xconomy Raleigh-Durham — 

While CRISPR has grabbed the lion’s share of gene-editing headlines, Precision BioSciences has used its own gene-editing technology to quietly build a pipeline of products for both agriculture and human health. The Durham, NC company is now preparing to start testing a gene-edited cell therapy for cancer in humans and it has raised $110 million to support its efforts.

Precision Bio said Wednesday that it will apply the new capital, a Series B round of funding, toward clinical trials testing its cell therapy. The company also says it will ramp up pre-clinical work on a gene therapy program, and bring a food program into field trials.

The latest financing comes two years after Precision Bio reached a deal with Baxalta that paid the startup $105 million initially, and up to $1.6 billion in additional payments, in a partnership to develop cancer immunotherapies. That partnership transferred to Shire (NASDAQ: SHPG), which acquired Baxalta in 2016. According to Shire’s securities filings, the Dublin, Ireland drug maker holds the exclusive option to pick up compounds from the partnership for late-stage clinical development and commercialization. Precision is paying for development costs up until Shire exercises its option. Shire’s partnership with Precision Bio will transfer to Takeda Pharmaceutical, which is in the process of closing its acquisition of Shire.

Precision Bio says its lead drug candidate is a type of cancer immunotherapy that involves removing T cells from the body and engineering them to better recognize cancer cells. Those cells are then infused into a patient to treat cancer. The company uses its gene-editing technology to delete genes that would cause the donor T cells to attack healthy tissue or prompt an immune response. The company also says it can insert genetic material in the T-cell that direct the cells to a particular “marker” on a tumor.

Last year, the FDA approved the first ever cancer treatments that involve modifying a patient’s own immune cells. Novartis (NYSE: NVS) won approval for tisagenlecleucel (Kymriah), a so-called CAR-T cancer immunotherapy to treat a severe form of leukemia. Gilead Sciences (NASDAQ: GILD) followed, winning approval for axicabtagene ciloleucel (Yescarta) as a treatment for serious cases of non-Hodgkin lymphoma. But this approach is expensive. Gilead’s treatment costs $373,000. The Novartis therapy costs $475,000. Also, clinical studies showed that not all patients responded to these treatments and in some cases, patients experienced severe side effects.

Rather than using a patient’s own T cells, Precision Bio works with T cells from healthy donors. The company says this “off-the-shelf” approach to treatment could be more consistent, scalable, and economical. The company also hopes its approach will expand both the number patients that respond to treatment, as well as the number of cancers that can be treated. Precision Bio has not said which cancers it plans to target, nor did the company say when it expects a clinical trial to start.

Precision Bio was founded in 2006 based on research licensed from Duke University. The company’s technology, which it calls Arcus, can modify genes in the cells of mammals or plants. Agriculture was Precision Bio’s first target and the company initially used its technology to support the research of partners. Disclosed partners include Syngenta, Agrivida, and Cargill. In 2012, Precision Bio split its plant research into a subsidiary called Precision PlantSciences. Earlier this month, Precision Bio renamed that business Elo Life Systems.

Precision Bio last raised money in 2015, when it closed a $26 million Series A financing round. At that time, CEO and co-founder Matt Kane told Xconomy that Arcus offers more precision and flexibility compared to CRISPR or TALENS, another gene-editing technology.

Arrowmark Partners led the Series B round of financing, joined by new investors 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.

Precision Bio said that earlier investors venBio, F-Prime, RA Capital management, Amgen Ventures, Osage University Partners, DUMAC, and the Longevity Fund also participated in the latest financing. In conjunction with the new funding, Precision Bio says Tony Yao of Arrowmark has joined the company’s board of directors.

Here’s more on Precision Bio’s approach to gene editing.

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