BioPharma, Pharma

Intellia, Cellex form CAR T cell therapy newco backed by $250M from Blackstone

Limitations to cell therapies include the types of cancers they can address and the dangerous side effects that these treatments can cause. Gene-editing biotech Intellia sees technology from clinical-stage GEMoaB as a potential solution, and it is forming a new company that will pair their respective platforms. Blackstone Life Sciences has committed $250 million to the new venture.

T-cells attacking cancer cell illustration of microscopic photosT-cells attacking cancer cell illustration of microscopic photos


Gene-editing biotech Intellia Therapeutics is joining forces with Cellex Cell Professionals, launching a new startup that will develop immunotherapies for cancers and inflammatory diseases. That new company will be backed by up to $250 million from Blackstone Life Sciences.

This new company aims to improve on the type of cancer immunotherapy called chimeric antigen receptor T cells, or CAR Ts, which are made by engineering a patient’s own immune cells into a living drug. These therapies are so far approved only for treating blood cancers and they introduce the risk of sparking dangerous immune responses. Cambridge, Massachusetts-based Intellia believes that combining its technology with that of a Germany-based biotech can overcome those limitations.

Intellia brings its expertise in in CRISPR gene editing, which it uses to develop allogeneic, or off-the-shelf CAR T therapies from donor cells. Cellex offers its manufacturing capabilities. Cellex subsidiary, GEMoaB, will contribute technology that makes CAR T “switchable,” turning it on or off in order to improve how long the therapy is effective, as well as its safety. Speaking on a Tuesday conference call, Intellia CEO John Leonard said that if a cancer and normal tissue share a target, GEMoaB’s technology can withdraw the cell therapy if there is toxicity—a “reversability” that offers an advantage compared to other CAR T approaches.

“We think we’ll have access to a leading CAR T platform that essentially will leapfrog the other modalities that are out there,” he said. “It’s a powerful way to proceed.”

The yet-to-be-named company stems from an alliance that Intellia began last year with GEMoaB. The goal was to discover and develop “next-generation” cellular immunotherapies for difficult-to-treat cancers and inflammatory diseases. Leonard said that as the collaboration made progress, the partners came to realize that consolidating their respective technologies could move more quickly and efficiently. This new company, which will develop both autologous and allogeneic therapies, will have an exclusive license to combine Intellia’s technology platform with GEMoaB’s two T-cell platforms, called UniCAR and RevCAR.

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While the new company will become a separate entity, it will still keep close ties to the firms that formed it. Intellia has the right to share in the development and funding of an allogeneic universal CAR T-cell product that the new company develops for an immuno-oncology indication intended for co-commercialization in the U.S. and select European countries. Intellia also has an additional option to enter into a second co-development, co-funding agreement from selected allogeneic universal CAR T-cell therapies.

Intellia further benefits by addressing its manufacturing needs. An agreement with Cellex gives the Intellia access to the German company’s cell therapy manufacturing site and allogeneic cell donations, according to an investor presentation. That “preferred relationship” will support Intellia’s wholly owned pipeline of ex vivo cell therapies while also giving it the capacity to expand that pipeline. The new company will also have access to Cellex’s manufacturing capabilities.

The most advanced Intellia program, NTLA-2001, is being developed in partnership with Regeneron Pharmaceuticals as a treatment for the rare disease transthyretin amyloidosis. Interim Phase 1 data are scheduled for presentation on June 26 during the annual meeting of the Peripheral Nerve Society. GEMoaB has developed its own pipeline of preclinical and clinical-stage drugs for blood cancers and solid tumors. That pipeline includes wholly owned assets as well as a prostate cancer program in Phase 1 testing under a partnership with Bristol Myers Squibb.

Phase 1 tests of GEMoaB’s lead universal CAR T therapies for blood cancers and solid tumors are ongoing. Interim data published earlier this year in the journal Blood showed that the lead blood cancer program, UniCAR-T-CD123, was well tolerated by patients and demonstrated early signs of efficacy in treating relapsed or refractory acute myeloid leukemia. That program and others in the pipeline will stay with GEMoaB as it becomes a subsidiary of the new company.

The new company will draw its leadership team from both Intellia and GEMoaB. Andrew Schiermeier, currently executive vice president and chief operating officer of Intellia, will be the new company’s president and CEO. Gerhard Ehninger, CEO of Cellex and GEMoaB’s chief medical officer, will become chief medical officer of the new company. GEMoab Chief Scientific Officer Armin Ehninger will take on the same role at the new company. The new company will be based in Cambridge but maintain labs in Dresden, Germany. Leonard said that the bulk of the research will be done in Dresden, but Schiermeier will also build a team in the U.S.

“The idea is to have the company have a German background with a strong American accent, if you will,” Leonard said. “It’s a way of accessing the American markets, having a clinical presence here in the U.S., and moving forward as it progresses.”

The deal is expected to close in the third quarter of this year. When it does, Blackstone, Intellia, and Cellex will have equal ownership of the new company.

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