Rubius Therapeutics Springs Out of Flagship VentureLabs to Take on PKU with Red Cell Therapeutics

Rubius Therapeutics Springs Out of Flagship VentureLabs to Take on PKU with Red Cell Therapeutics
December 9, 2015
By Alex Keown, BioSpace.com Breaking News Staff

CAMBRIDGE, Mass. – Rubius Therapeutics, a company focused on developing engineered red cell treatments for multiple medical conditions, including autoimmune conditions, metabolic diseases and cancer, launched today with $25 million in backing from Flagship VentureLabs.

The new company, based in Cambridge, Mass., specializes in engineering red blood cells to have medicinal purposes. The company’s therapeutic program turns stem cells into red blood cells and then introduces genetic material into the cells to express specific proteins, which will make them uniquely suited for treating a variety of diseases, including infections, cancer and metabolic disorders. The engineered blood cells will remain in the body for up to four months, which gives them advantages in treating these various diseases over traditional therapies, the company said.

“What has us so excited is the broad applicability of red cell therapeutics. There is applicability in cancer therapy, autoimmune diseases and allergies. It’s a real versatile canvas to develop many new medicines,” Avak Kahvejian, chief executive officer of Rubius, told BioSpace in an exclusive interview Tuesday.

Engineered cells as a treatment, such as chimeric antigen receptor T cell (CART) therapies, are already being used by multiple companies. However, Kahvejian said T cells have a “highly specific function,” while the engineered red blood cells “have a variety of therapeutic properties to address a wide array of diseases.” Kahvejian said Rubius’ red cell therapeutics will allow diseases to be treated in broad and new ways that will have positive impacts on the lives of patients who face multiple diseases.

But first, Rubius has its sights set on developing a treatment for phenylketonuria (PKU), a metabolic disease 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. Most PKU patients maintain a strict diet in order to lower the amount of phenylalanine in the body. Kahvejian said Rubius’ red cell therapy should have a broad application in the treatment of PKU and allow patients to live more normal lives without having to endure a restrictive diet.

Kahvejian told BioSpace that Rubius’ experimental therapy should be more broadly accessible to PKU patients than existing PKU treatment, BioMarin ’s Kuvan, which is used with a subset of PKU patients.

“Ideally this will serve a wider number of patients and will liberalize them from having a more restrictive diet,” Kahvejian said.

For the past 18 months Rubius has been operating under the auspices of Flagship VentureLabs. Kahvejian said he anticipates the $25 million in funding will take the company through its first human trials, which are anticipated for the end of 2016.

Kahvejian said the company will also explore how its red cell therapeutics will also impact allergy research. He said his son has nut allergies, which for many people can be quite deadly, and the company’s therapy, when combined with a particular antigen, can change the way the body reacts to certain allergens.

“This is just a really exciting time and the potential is great,” Kahvejian said.

Rubius currently has 13 employees and has taken over 6,500 square feet of laboratory space in the thriving Kendall Square area of Cambridge. Kahvejian said the company will continue to hire over the next few months, and although he did not disclose a number of expected hires, he said it is conceivable the company could double in size.

This is the third company spun out of Flagship VentureLabs in the past month, Jeffrey Kasner, Flagship’s senior director of communications, told BioSpace. In November VentureLabs spun out Evelo Therapeutics, a company focused on microbiome therapies for the treatment of cancer, as well as Codiak Biosciences , a company focused on developing exosome-based therapies and products. Codiak launched under the leadership of Doug Williams, a former vice president of Biogen, Inc. .

“Each company is introducing new therapeutic modalities and bringing them to the forefront with major clinical implications. This is an exciting time for the field,” Krasner said.

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