Flagship launches Rubius to develop functionalized red blood cells for treatment of serious diseases

Flagship Ventures, a leading innovation and venture firm focused on healthcare and sustainability, announced it has launched Rubius Therapeutics, to develop functionalized red blood cells for the treatment of autoimmune conditions, metabolic diseases, cancer, and other serious diseases. Flagship has made an initial capital commitment of $25 million to enable Rubius to enter clinical testing of its novel therapeutic modality.

Rubius is pioneering Red-Cell Therapeutics™ (RCTs). RCTs are a new treatment modality and possess pharmacodynamic and pharmacokinetic advantages over traditional therapeutics, in part due to their natural ability to engage and modulate the immune system and circulate for extended periods in the body.

"Working together with our collaborators, technology advances made at Flagship's innovation foundry, VentureLabs®, led to the creation of a completely new therapeutic modality," said Noubar Afeyan, Ph.D., senior managing partner and CEO of Flagship Ventures and a director of Rubius. "We believe Red-Cell Therapeutics will become a new standard of care in many areas of high unmet medical need."

One of the diseases Rubius intends to initially target is phenylketonuria (PKU), a disease of metabolism that affects as many as one in 13,000 births in the U.S. and is responsible for about 300 new cases each year. People born with PKU have a genetic defect that leaves them unable to break down a particular amino acid, one of the building blocks of proteins. Left untreated, the amino acid, phenylalanine, builds up in the body and causes severe developmental problems and long-term health issues. Newborns are routinely tested for this genetic disease within days of birth.

Currently, the most effective treatment for PKU is a strict diet that lowers the amount of phenylalanine in the body. Rubius has developed and pre-clinically validated a Red-Cell Therapeutic capable of enzymatically reducing phenylalanine levels in human serum. By continually lowering phenylalanine levels in the bloodstream, RCT therapy promises to allow PKU patients to live normally without extremely restrictive diets.

"Red blood cells can now be produced in culture and engineered to possess enormous biotherapeutic properties," said Harvey Lodish, Ph.D., professor of biology and bioengineering at MIT and a member of the Whitehead Institute, who is a scientific founder of Rubius and has joined the firm's board of directors. "They spend as much as four months in circulation, providing an opportunity for long and tunable therapeutic treatments. Red cells also have profound effects on the immune system and may ultimately transform the way we treat autoimmune diseases and allergies."

"Through its rapid prototyping capability, Rubius has generated and tested over 50 different RCTs for a wide array of indications including autoimmunity, oncology and infectious disease," said Avak Kahvejian, Ph.D., founding CEO of Rubius and Flagship VentureLabs partner. "The company is now poised to drive lead programs into the clinic, build a drug pipeline and further develop the Rubius Erythrocyte Design™ (RED) platform. We are expanding our R&D and management teams, and setting the stage for our next phase of growth."

Besides Dr. Afeyan and Dr. Lodish, the Rubius board of directors includes:

• Jim Gilbert, a healthcare investor and former partner and director at Bain & Co., who serves on several boards, including the board of Nestle Health Sciences. Jim serves as chairman of Rubius.
• Robert S. Langer, Ph.D., Institute Professor at MIT and the holder of more than 1,000 patents
• Dr. Roger Pomerantz, chairman and CEO of Seres Health and former worldwide head of licensing and acquisitions, senior vice president at Merck & Co. Inc.
• Dr. Michael Rosenblatt, executive vice president and chief medical officer of Merck & Co. Inc., and former dean of the Tufts University School of Medicine.
• Peter Hutt, senior counsel at Covington & Burling and former chief counsel of the U.S. Food and Drug Administration.

Since emerging from Flagship VentureLabs 18 months ago, Rubius has been developing its RCT platform in stealth mode. The company has established a broad patent estate covering its platform and therapeutic products. Rubius is currently testing RCTs in animal models of disease, and plans to initiate human clinical trials in the very near future.