Rubius goes after genetically re-engineered red-cell drugs with $25M from Flagship

Rubius CEO Avak Kahvejian

For the past 18 months, Rubius Therapeutics has been quietly stitching itself together into a new biotech looking to pioneer a radically new technology: turning hematopoietic stem cells into red blood cells and "functionalizing" them with built-in therapeutic proteins that can address dozens of potential drug targets.

Today, Rubius is coming out of stealth mode with a $25 million Series A bankrolled entirely by Flagship Ventures, an early-stage investor which fostered the biotech in its prolific VentureLabs. 

Rubius is pursuing a drug technology that was hatched in the lab of MIT's Harvey Lodish, professor of biology and bioengineering at MIT and a member of the Whitehead Institute. Up front and center is Rubius's initial target: phenylketonuria (PKU), a genetic condition that blocks the breakdown of an amino acid, allowing phenylalanine to accumulate, with potentially catastrophic results that includes brain damage. 

After that first target, though, the startup says that there are dozens of potential programs to ramp up. "Red cells also have profound effects on the immune system and may ultimately transform the way we treat autoimmune diseases and allergies," notes Lodish.

Engineering new red blood cells from hematopoietic stem cells has been a big focus in biotech. But CEO Avak Kahvejian says that "we're the only company making these red cells but also genetically engineering these cells" into a therapy.

Rubius has been testing this approach--using a lentiviral delivery vehicle to insert a gene that encodes for the protein into the cell--in animals as well as in vitro, with encouraging results, according to Kahvejian. And there are 13 staffers now at work in a 6,400-square-foot facility on Memorial Drive in Cambridge, MA.

There are about 300 new cases of this rare disease each year in the U.S., with an estimated 15,000 to 20,000 cases. Many of these patients control the disease through a rigid protein-free diet with supplements. A few months ago, BioMarin ($BMRN) bought the global rights to Kuvan, which is used to lower levels of phenylalanine in a PKU patient's blood.

That drug, says the CEO, is only mildly effective on a subset of patients, leaving the road clear for Rubius to go after a much more effective treatment. 

This first program will be in the clinic next year, says Kahvejian. And he expects to follow up with more, doubling the staff in the next year while exploring new collaborations with biopharma companies.

Lodish and Flagship's Noubar Afeyan are both on the board, along with some familiar faces from the Cambridge biotech hub. The board includes investor MIT's Robert Langer, Seres ($MCRB) CEO Roger Pomerantz, Jim Gilbert, a healthcare investor and former partner and director at Bain & Co., Merck's ($MRK) Michael Rosenblatt and lawyer Peter Hutt.

This is the third new biotech from VentureLabs this year, with Rubius following Evelo Therapeutics and Codiak BioSciences, which combined a VentureLabs company with IP from the University of Texas. A Flagship spokesman says that the venture group has now launched 36 companies from VentureLabs. 

- here's the release

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