A $10.1 million grant the California Institute for Regenerative Medicine (CIRM) awarded to ViaCyte last week will be used to move the San Diego diabetes company’s stem cell and cell encapsulation technologies into human clinical trials, according to ViaCyte CEO Paul Laikind. CIRM administers $3 billion in funding for stem cell science that California voters approved in 2004 with the passage of Proposition 71.
ViaCyte has developed a pair of related technologies that have eliminated symptoms in diabetic rats and mice. The company was founded in 1999, merged with CyThera and BresaGen in 2004, and was renamed ViaCyte in 2010.
Using genetic tools, ViaCyte says it can direct embryonic stem cells to produce pancreatic progenitor cells with the ability to produce large quantities of safe and functional islet cells, which can be used to treat patients with insulin-dependent Type 1 and Type 2 diabetes. A related proprietary technology enables ViaCyte to encapsulate its engineered cells in packets that can be implanted beneath the skin in a way that allows the encapsulated cells to release insulin into the bloodstream. The company describes its technology as an “artificial pancreas” that does not require drugs to suppress the body’s immune response to the implanted cells.
Laikind, who was named as ViaCyte CEO in June, told me yesterday at San Diego’s annual Stem Cell Meeting on the Mesa that CIRM has supported ViaCyte through previous rounds of funding. In announcing the grant, CIRM noted that the independent Grants Working Group characterized ViaCyte’s proposed therapy as the “holy grail” of diabetes treatments.
“The focus now is on getting through the pre-IND (Investigational New Drug) studies,” Laikind said. “We just had a pre-IND meeting with the FDA and laid out the path to the clinic and the preliminary clinical trial protocol.” He added that the company would likely file its IND application with the FDA toward the end of 2013.
The California stem cell agency said it also awarded $9.3 million to Bluebird Bio, which has offices in Cambridge, MA, and San Francisco, to use a stem cell and gene therapy approach to young patients with Beta-thalassemia, a genetic blood disorder. B-thalassemia patients have an inadequate amount of functional hemoglobin, the protein that distributes oxygen through the circulatory system. In severe cases, B-thalassemia can cause organ damage and death.
Both grants were awarded at the October 25th meeting of the stem cell agency’s governing board, the Independent Citizens Oversight Committee (ICOC). CIRM says they are the first awards under the agency’s Strategic Partnership Awards initiative, which is designed to engage more effectively with industry and to increase outside investment in CIRM-funded stem cell research.
The statement from CIRM quotes ICOC chairman Jonathan Thomas as saying: “These awards are designed to help companies complete early stage clinical trials within four years. We feel if we can help companies demonstrate that their therapies are safe, even in small groups of patients, that they will then be able to attract funding from a large biotech or pharmaceutical company to help them complete larger-scale clinical trials and get FDA approval.”