So here’s how Blackstone is spending its $4.6 billion biopharma pot.
The private equity firm announced Tuesday they were teaming with the CRISPR biotech Intellia and the little-known German CAR-T startup GEMoaB to launch a new — and so far unnamed — CAR-T company. Blackstone, the sole investor, will pour $250 million into the joint venture and take a third ownership. Intellia and Cellex, GEMoaB’s parent company, will each take another third.
The company will focus on building off-the-shelf CAR-Ts that can be manipulated and controlled even after they’re administered to patients — two of the major goals that other CRISPR and cell therapy companies are now trying to attain.
“This launch represents the first of many steps to addressing the various limitations that currently exist in the cell therapy space,” Intellia COO Andrew Schiermeier, who will become president and CEO of the new company, said in a statement.
Olivier Brandicourt, the former Sanofi CEO who joined Blackstone as an advisor this year, will chair the board.
The new company will start out with two programs already in the clinic. A subsidiary of Cellex, GEMoaB already put two controllable CAR-Ts in Phase I: one for leukemia and one for prostate cancer.
Both programs rely on GEMoaB’s controllable CAR-T technology. Instead of arming T cells with receptors that bind to proteins on cancer cells, they put on receptors that bind to a “targeting module.” When the modules are infused into a patient, they bind to a protein on cancer cells and the CAR-Ts bind to the modules. They then, hopefully, destroy the cancer cells. If the modules are ever taken away, GemOAB says — for example, because the patients are starting to show major side effects — the CAR-Ts stop working within four hours.
The approach, they say, allows them to go after targets that would prove toxic under traditional CAR-T approaches. That includes CD123, a promising leukemia target that other developers avoided because it also sits on healthy cells. Carl June’s Tmunity Therapeutics saw two deaths last month going after the same target, PSMA, that GEMoaB is pursuing in cancer.
It’s a similar approach to the one Arcellx is now pursuing preclinically and the one now-defunct Unum pursued disastrously before its collapse. But GEMoaB distinguished itself with proof-of-concept human data at ASH last month.
Those programs both rely on a patients’ own cells, but the new company will also look to develop off-the-shelf therapies. There are few details yet for the rest of the company’s approach, except that it could also cover autoimmune diseases as well as cancer. The new company will co-develop a CAR-T for cancer with Intellia and has an option to co-develop a second one for a different indication.
Intellia has been largely tight-lipped around its cancer pipeline so far, disclosing only one of several therapies they’re developing. But the deal comes at a time when more CRISPR companies are pushing into CAR-T. Largely, they’ve used CRISPR to engineer cells that can persist longer or that can be given off-the-shelf.
After making progress on their sickle cell therapies, CRISPR Therapeutics’ next major batch of data will come from a pair of cancer cell therapies. In March, Caribou Biosciences raised $115 million as they pushed to develop CAR-Ts for the first time.
For Blackstone, the deal represents one of their largest investments in a startup since they closed their $4.6 billion fund last year, although they do not have a perfect scorecard on their previous bets. Ferring, a gene therapy developer they gave $400 million, imploded this year.