12:00 AM
Jan 07, 2016
 |  BC Innovations  |  Product R&D

Notorious RBC

Rubius reaps the rewards of RBC advances and advantages

Rubius Therapeutics Inc. emerged from stealth mode last month to announce the first candidate from its engineered red blood cell platform. Behind that program, the company has an arsenal of tailored "red-cell therapeutics" that capitalize on the unique biology of RBCs and co-founder Harvey Lodish's discoveries of how to manipulate and culture them efficiently.

"We can put antibodies on the surface and bind all kinds of nasty things from the blood; we can put many kinds of enzymes on or in red cells. There's a huge number of possible applications, and we're working on many of them," said Lodish, who is also a founding member of the Whitehead Institute for Biomedical Research and professor of biology and bioengineering at the Massachusetts Institute of Technology.

The technology allows the company to add proteins of choice to RBCs and produce a new form of therapeutic that's a cross between a sophisticated liposome and a standard cell therapy. Because the cells persist in the circulation for up to four months, they can have more potent or longer-lasting effects than liposomes and soluble proteins. But because they have no nucleus, they avoid the risk of oncogenic proliferation - one of the primary drawbacks of standard cell therapies.

Lodish told BioCentury that the U.S. Defense Advanced Research Projects Agency (DARPA)'s interest in blood replacement products for battlefield use helped lay the scientific foundation for the system by driving research to overcome limitations in in vitro blood production.

"About five years ago, DARPA contacted me because they had contracts out to companies to make red cells in culture for transfusion, and there was a bottleneck," Lodish said. "A lot of groups could take bone marrow or cord blood stem cells and put them in culture, and they would go much of the way to make red cells. The cells would induce hemoglobin and turn red, but they never finished the differentiation process" by losing their nucleus, he said.

"What we proceeded to do is develop a very robust culture system for converting hematopoietic...

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