Weaponizing red blood cells to improve on CAR-Ts, treat cancers and rare diseases.
President: Torben Straight Nissen
Based: Cambridge, Massachusetts
Clinical focus: Cancer, rare and autoimmune diseases
The scoop: CAR-Ts have reinvigorated interest in cellular therapies and ratcheted up expectations for cancer outcomes. But the technology comes with some widely-acknowledged problems, from the relative lack of efficacy in solid tumors to the logistical headaches of harvesting, processing and administering cells.
Enter Rubius Therapeutics. Rubius is developing off-the-shelf red-cell therapeutics its president Torben Straight Nissen thinks will be “an improved and superior cellular therapy over the CAR-Ts.” And, buoyed by a $120 million round, the Flagship Pioneering-backed biotech is working on a broad pipeline that extends beyond cancer.
What makes Rubius Fierce: For many biotechs, improving on one of the hottest technologies to come around in years would be enough to keep them busy. For Rubius, pulling the rug out from under CAR-Ts is just one item on its to-do list.
Roughly following the playbook Flagship wrote with Moderna, Rubius raised a big preclinical round to exploit a platform with broad potential. Rubius is using the platform to turn hematopoietic stem cells from donors into enucleated red cells that express one or more proteins. And, critically for its drive to unseat CAR-Ts, Rubius will produce these off-the-shelf cells at large scale in bioreactors.
When expressed on the surface of the red cell, these combinations of proteins deliver the sorts of one-two hits that immuno-oncology companies are trying to engineer using cocktails of different drugs. A cell armed with a checkpoint inhibitor and costimulatory molecule could lower a tumor’s defense and dial up the immune response against it.
If the goal is to treat an enzyme-related disease, Rubius expresses proteins inside the cell. This hides the payload from the immune system inside a delivery vehicle Rubius thinks will persist for months after administration.
The potential of Rubius’ platform has enabled it to contribute to the migration of Big Pharma executives to biotech. Nissen left a senior position at Pfizer to take up the president post late last year. And former Novartis pharmaceuticals chief David Epstein arrived around the same time, initially to serve as chairman. Epstein added the “executive” prefix to his title in an expansion of his role in August.
In his new post, Epstein will spend more time with Rubius, specifically to work alongside Nissen to strike one or two partnering deals to expand use of the platform beyond the biotech’s bandwidth. The duo bring Big Pharma connections and, in the case of Epstein, a track record of identifying and advancing cell therapies to the task.
“David was really a driver and pioneer within Novartis in bringing in the CAR-Ts,” Nissen said. “He saw the potential [of CAR-T] very early on. He's seen the potential now in Rubius’ RCT platform ... and has seen that our platform is addressing a lot of the potential issues with the CAR-Ts. We think that validates our platform and will be hugely beneficial going forward.”
While Nissen and Epstein scout out deals, a growing team back at base is working to get drugs into the clinic next year and discover a pipeline of follow-on programs. Headcount now stands at 50, up from 40 at the time of the financing, and is on track to hit 100 by the middle of next year.
The new hires are bolstering Rubius’ development, bioprocessing and manufacturing groups, the importance of which will grow and grow as the company gets into the clinic and starts making its drugs at larger scale. And Rubius is adding further to its discovery and platform technology teams to ensure it squeezes the full potential out of an approach it sees unlocking a wide range of doors.
Investors: Flagship Pioneering and undisclosed institutional investors