Wednesday, March 16, 2016

California Okays $7.3 Million for Stem Cell Research Related to Duchenne's and Immunodeficiencies

Directors of the California stem cell this morning awarded $4.3 million to researchers at UC San Francisco and $3 million to Capricor, Inc., of Beverly Hills for clinical trial-related research for therapies connected respectively to an extremely rare immunodeficiency affliction and Duchenne's disease.

The larger award was ratified on a 10-3 vote after questions arose about whether sufficient patients could be recruited for a clinical trial, additionally a concern of the agency's grant reviewers who earlier approved the application, also on a split vote.

The agency has already provided $3.9 million for the UCSF research. The lead scientists on that effort were Morton Cowan and Jennifer Puck. The treatment is aimed at the "bubble boy" immunodeficiency disease. The agency's summary of the application review said that the research "could lead to a lasting cure" for that version of the affliction.

The Capricor award was approved on a 13-0 vote after Jeff Sheehy, a member of the governing board, said it was "pretty much a pure CIRM product," referring to the initials of the stem cell agency, formally known as the California Institute for Regenerative Medicine.

Capricor, a publicly traded firm, has already received $19.8 million from the agency to develop stem cell heart treatments, The $19.8 million came on top of earlier, related funding for research at Cedars-Sinai that hit $7 million.

The treatment is aimed at Duchenne muscular dystrophy cardiomyopathy using a Capricor product called CAP-1002.

California's expected action on the two awards was first reported by the California Stem Cell Report on March 8.

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