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.”
