ViaCyte, Bluebird score $19.4M in CA stem cell funds for game-changing therapies

California has steered nearly $20 million in stem cell awards to a pair of biotech companies working on potentially revolutionary treatments for diabetes and the blood disorder beta-thalassemia. And the pair of grants comes from the state's California Institute for Regenerative Medicine (CIRM) under a program that aims to boost investment and the tricky business of commercializing stem cell research.

San Diego-based ViaCyte scored $10.1 million from CIRM to gear up for clinical development of a stem cell-based therapy for diabetes that could reduce or eliminate the need for some patients with the disease to take injections of insulin, if successful. And Bluebird Bio, based in Cambridge, MA, has a potential breakthrough gene therapy for beta-thalassemia that could free patients with the blood disorder from requiring transfusions. The California stem cell agency awarded the biotech $9.3 million.

Bluebird and ViaCyte are the first two companies to gain funds via CIRM's new program to partner with the industry, which has been slow to translate stem cell discoveries from academia into therapies for patients. Eight years after hopeful California taxpayers approved the $3 billion stem cell program, stem cell science hasn't lived up to its promise to cure a wide variety of illnesses. And the FDA says it's approved just one cord stem cell therapy for certain blood disorders.

The California stem cell agency, which has devoted much of its funding to academic research, is now boosting its bets in biotech. At ViaCyte, which has received previous awards from CIRM, the company has what CEO Paul Laikind called a therapy that is "replacing the pancreas," where insulin-producing cells are destroyed in patients with Type 1 diabetes, The North County Times reported. His company is using the funds to prepare for clinical trials of its artificial pancreas.

Bluebird (a 2012 Fierce 15 company) has raised more than $100 million from investors for its experimental gene therapies. With the CIRM funds, the company plans to begin a Phase I/II study next year of a gene therapy that corrects patients' own hematopoietic stem cells with a healthy beta-globin gene to allow patients with beta-thalassemia to produce red blood cells.

"Moving a promising stem cell therapy into clinical trials can be very demanding technically and also very expensive," Alan Trounson, the stem cell agency's president, said in a statement. "That's why we created this initiative, to make it easier for companies to partner with CIRM in clinical trials and to more effectively enable critical expertise and industry resources to support and help manage product development to achieve widespread availability for patients."

- here's CIRM's release
- see Bluebird's release
- and the article from The North County Times

Special Report: Bluebird Bio – 2012 Fierce 15