Startups, BioPharma

Using red blood cells as therapeutics: Flagship Ventures launches Rubius Therapeutics with $25M

The company plans to enter the clinic by the close of 2016 with a red blood cell-based therapy that treats phenylketonuria.

Red blood cells are known largely for their critical role in ferrying oxygen and carbon dioxide throughout the body – but they’re capable of so much more. That’s why this transportation mechanism has been harnessed by a cadre of Cambridge scientists, who have launched a compelling new startup: Rubius Therapeutics.

The company engineers red blood cells from hematopoietic stem cells, changing them into what Rubius calls “Red-Cell Therapeutics.” These RCTs are, in essence, modified RBCs that can transport therapeutic proteins throughout the body. They hold certain pharmacodynamic and pharmacokinetic advantages over traditional therapeutics, the company says – thanks in part to their natural ability to modulate the immune system and circulate in the bloodstream for a long time.

Cambridge investment firm Flagship Ventures has infused $25 million into the new startup, aiming to usher its RCTs into clinical trials by the close of 2016.

“This is very unique – we’re using tools from gene therapy and the genetic engineering world to create a broad new category of medicine,” CEO Avak Kahvejian said in a phone interview. “We can make RCTs with enzymatic capabilities, or with antibodies or antigens on the surface – containing any kind of protein we want to encode.”

These RCTs have applications in treating autoimmune conditions, metabolic diseases, cancer and other serious conditions, Kahvejian said. One major advantage of this modality is that RCTs have no nucleus, so can’t reproduce in the body – but can last for months in the body, delivering therapy as needed.

Rubius has already prototyped more than 50 different RCTs for many of these indications, he said – and plans to build out its “Rubius Erythrocyte Design platform” as well as grow a drug pipeline.

The first target Rubius will pursue is a treatment for phenylketonuria, or PKU – a metabolic disease caused by a genetic defect that prevents phenylalanine from being broken down in the body. Buildup of this amino acid causes severe developmental issues. Currently, PKU can only be controlled by diet. However, in preclinical models Rubius has created an RCT that can enzymatically lower phenylalanine levels in human serum.

Operating in stealth for the past 18 months, Rubius employs 13 and is on the prowl for more. Much of the technology was developed in-house, Kahvejian said, and a lot was invented by the innovation team at Flagship Venture Labs. It has a number of notable names on its board – including Dr. Robert Langer, the patent maven of MIT, and Merck Chief Medical Officer Dr. Michael Rosenblatt. The company’s scientific founder is Dr. Harvey Lodish, a professor of biology and bioengineering at MIT and a member of the Whitehead Institute.

“Red blood cells can now be produced in culture and engineered to possess enormous biotherapeutic properties,” Lodish said in a statement. “They spend as much as four months in circulation, providing an opportunity for long and tunable therapeutic treatments.”

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