Rock star researcher licenses out new CAR-T platform to upstart

Boston Harbor

PureTech has launched a preclinical biotech today that begins life with a new CAR-T platform licensed from Pulitzer Prize-winning oncologist Dr. Siddhartha Mukherjee.

Boston, MA-based PureTech--which works as a hybrid venture fund and R&D biotech--has launched the startup “Vor BioPharma,” which will focus on developing a new chimeric antigen receptor T-cell therapy (CAR-T) platform. Vor is a wholly owned subsidiary of the company. The company has not released any preclinical data from the platform and financial details of the licensing deal were not disclosed. 

Its tech has been licensed from the lab of Vor’s scientific co-founder, Dr. Siddhartha Mukherjee--assistant professor of medicine at Columbia University, and the Pulitzer Prize-winning author of The Emperor of All Maladies: A Biography of Cancer

At the heart of the platform is CAR-T, which many Big Pharma and smaller biotechs are hopeful will be the next big thing in cancer research.

T-cell therapy--which modifies the body’s own immune cells (T cells) to recognize and kill cancer cells--has already shown some stellar results from patients with advanced B-cell leukemias, using the antigen CD-19--a protein expressed on the cell surface of B-cell lymphomas and leukemias.

Kite Pharma ($KITE), Novartis ($NVS) and Juno Therapeutics ($JUNO) are the big players in this emerging field and all have posted some startling results in certain blood cancers--but there have been limitations outside of B-cell leukemias and especially in solid tumors.

Speaking to FierceBiotech, Aleks Radovic-Moreno, senior associate of PureTech and co-founder of Vor, said that the new platform from Mukherjee--which has not yet produced any drugs--will look to go beyond the B cell and aim for cancers with limited treatments.

Radovic-Moreno said: “CAR-T is really a medical revolution and the data have been fantastic--in B-cell malignancies. When we looked at this space we thought wow, wouldn’t it be great if we could have the technology that could replicate the success we’ve seen in B-cells to other types of malignancies. When you look at the current CAR-T field, it’s been a challenge replicating that.

“In the new platform we have from Sid [Mukherjee], we believe can go well beyond the B-cell. This is a platform technology but it has the potential to create a pipeline of new products. It’s very early-stage and we can’t really talk about timelines, but we’re discussing right now what avenues we want to go down. But when you look at where the low-hanging fruit is--and where there is still great unmet medical need--we think that’s a great place to start to really demonstrate the power of the platform.”

He said that he didn’t want Vor to be the eleventh company to develop a CD-19 CAR-T: “We’re going after indications which really are not well addressed, we're not just following the pack.”

He explained that the platform and the biotech’s method is a new way of targeting cancer. “We think that there are certain cell populations that when you target them effectively, it leads to a more durable treatment effect. We’re essentially developing this platform to help eliminate these specific cell populations. Interestingly, by targeting these cell populations there is also the possibility of reduced resistance emerging--and that’s actually something we’re starting to see right now [resistance in the CD-19 CAR-Ts], so this is something we’re keeping in mind with our tech.”

Looking down the road, could there be tie-ups with other CAR-T players--or even those researching checkpoint inhibitors? This is something that’s already happening, with Roche ($RHHBY) and Kite Pharma announcing back in March that they would test the Swiss company’s new PD-L1 drug atezolizumab alongside Kite's CAR-T treatment KTE-C19 in patients with refractory, aggressive non-Hodgkin lymphoma. Its rival Juno signed a similar deal last year to test a combination its CAR-T candidate with AstraZeneca's ($AZN) PD-L1 blocker, also in NHL.

Radovic-Moreno said: “We’re exploring and prioritizing the pipeline as quickly as we can in the preclinical setting, but at the moment, we’re focused on doing our own thing--which is essentially to make sure we’re going to get the most out of our platform. But having said that, we are very open to collaborations and we think there is a lot of potential to work with partners in this space, and we will do so at the appropriate time.”

On the safety side, he said: “The CD-19 CAR-Ts have resulted in profound B-cell aplasia and the complete loss of B-cells [although not life-sustaining tissues, this is still a severe toxicity] and that’s actually a biomarker of success in patients. So, we are very aware of that, and we think our targeting will not have that affect.”

Can Vor’s CAR-T platform get around this, and perhaps other toxicities seen with current CAR-T research? “We think our approach will have differentiating aspects as regards safety. Of course, you have to go to the clinic to get the definitive answer, but from what we’ve seen so far, we think there is a high potential for us to have a better safety profile, at least on the cell preservation side.”

On the business side, the company works in-line with PureTech’s new hybrid strategy that will see it develop its own medicines and also help fund smaller upstarts, of which it will typically own the majority stake. Daphne Zohar, PureTech’s CEO, said that Vor is “100% owned by us at this point,” so it doesn’t currently see the need for any external VC funding rounds.

Zohar said that in terms of gaining access to the platform, her company focuses on a specific research theme or area and then spends time working with people involved in that field.

“I think that allows us to hear about things earlier than others, and even before they’re published or out in the public domain. Sid actually has strong working relationships with other key oncology researchers who we’ve worked with before--or helped set up companies with them in the past--so we could see just a little bit past the horizon with this platform, which allowed us to get involved at this early stage.”

She added that the biotech will work with “leading oncologists and immunologists” in developing its pipeline. It’s also set up a team of scientific founders and a Scientific Advisory Board, which includes Joseph Bolen, Vor’s acting CSO, who was formerly president and CSO of Moderna Therapeutics, and Sanjiv Sam Gambhir, a professor of radiology, materials science and engineering, and bioengineering at Stanford University, who has co-founded several startups and is an adviser to several other biotechs.

David Steinberg, executive VP at PureTech, will be the acting CEO for Vor. This is the typical PureTech approach: to have members of their management lead new companies for a time when launched. Vor will be based with PureTech in Boston. 

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