The concept of turning a patient’s own immune cells into a cancer therapy has made strides, with the past year seeing FDA approvals of two such treatments for blood cancers. But these cell therapies don’t work for everyone, and they can cause dangerous side effects. They also haven’t yet worked on solid tumors.
RootPath wants to improve cancer cell drugs with an approach intended to be personalized to each patient’s disease, faster to develop than current cell therapies, and effective on solid tumors. The Cambridge, MA-based biotech is now peeking out from stealth with $7 million in venture funding to continue its research.
Sequoia China led the seed financing, which RootPath CEO Xi Chen says will support preclinical development of the technology. Other investors included Volcanics Venture, Baidu Ventures, and Nest.Bio Ventures.
Immunotherapy that uses a patient’s immune cells is called adoptive cell transfer. T cells, the frontline defenders of the immune system, are collected, engineered to better recognize and fight cancer, multiplied in a lab, and then re-infused back into the patient. Last year, Novartis (NYSE: NVS) and Gilead Sciences (NASDAQ: GILD) won FDA approvals for such cell therapies that treat advanced forms of leukemia. Both of these therapies are classified under a type of adoptive cell transfer called CAR-T cell therapy.
RootPath’s approach is similar to CAR-T up to a point, Chen says. T cells are collected, and they are ultimately infused back into the patient. But Chen says RootPath has developed technologies that enable it to analyze and sequence individual T cells, which helps identify the ones that are best suited to recognizing and fighting the tumor. Chen is keeping much of the detail about the technology under wraps for now, so it’s hard to independently assess the approach’s merits. But he says it could make a RootPath cell therapy more targeted to the tumor, which would in turn spare other tissue and avoid the side effects seen in CAR-T treatments.
Chen says RootPath’s cell therapies could also be more powerful. CAR-T therapies target a specific antigen on a tumor. By addressing a number of different tumor targets, a RootPath cell therapy could be effective on solid tumors, he says.
Speed is yet another advantage RootPath is aiming for. Chen claims that RootPath’s approach can expand, or multiply, the T cells needed to treat cancer—it requires hundreds of millions of them—at a much faster clip than other cell therapies. That means a RootPath therapy could reach the patient sooner.
A number of companies are opening their wallets in pursuit of ways to make cell therapies more effective and less toxic. Last year, Gilead acquired Cell Design Labs, a Santa Monica, CA, biotech that has researched ways to engineer cells to better recognize cancer and spare healthy tissue. In February, Gilead entered a partnership with Sangamo Therapeutics (NASDAQ: SGMO) aiming to develop next-generation cell therapies. And earlier this month, Bluebird Bio (NASDAQ: BLUE) inked a deal to develop new CAR-T therapies with Regeneron Pharmaceuticals (NASDAQ: REGN).
RootPath was formed last year and incubated within Nest.Bio labs in Cambridge and Hangzhou, China, Chen says. The company’s co-founders include Le Cong, who worked at the Broad Institute in the lab of Feng Zhang, a pioneer of CRISPR genome editing. Chen says RootPath’s approach uses CRISPR as a research tool, not in the actual therapy. But he adds that long term, it’s possible that gene editing could be used to knock out certain genes, which would then enable T cells to become more effective.
Chen says RootPath will pursue its research on its own, but would consider a partnership if a deal helps the company reach its goals faster. RootPath plans to continue developing its technology in the next three to six months, and then apply the remainder of the seed funding to animal tests lasting another six months. RootPath will need to raise additional cash in order to pursue its goal of human testing within the next two years, Chen says.