CIRM Showcase Features Promising Stem Cell Therapies for Patients

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CIRM Showcase Features Promising Stem Cell Therapies for Patients

By Dr. Karen Ring, Big3Bio Events Correspondent


Neil Littman of CIRM

What has the California Institute for Regenerative Medicine (CIRM) accomplished since it was started ten years ago? The answer is nine clinical stage programs, two stem cell screening platforms, and a whole lot of promise.

CIRM hosted its first showcase on “Accelerating Stem Cell Treatments to Patients” in partnership with Johnson & Johnson Innovation, J-Labs, at UCSF on Wednesday. The showcase featured CIRM-funded academics and biotech companies that have developed promising stem cell therapies that are entering clinical trials for disease indications including diabetes, cardiovascular disease, neurodegeneration, and cancer.

Niel Littman, business development officer at CIRM, kicked off the event by emphasizing CIRM’s main goal of getting stem cell treatments to patients with unmet medical needs. He explained that CIRM will pursue this goal by fostering collaborations between industry, investors, and CIRM grantees.

Then, Randy Mills, president of CIRM, next unveiled CIRM 2.0, which is CIRM’s new plan to speed up the development of effective stem cell treatments. The new plan will involve a more efficient grant-funding process involving open enrollment RFAs (requests for applications) and reduced cycle time for funding grants.

He explained that the main goal of CIRM 2.0 is to allow projects that are ready for clinical trials to move forward more quickly and to “attract, award, and administer higher quality grant applications that better serve CIRM’s mission of addressing unmet medical needs.” CIRM 2.0 will be enacted on January 1, 2015 and will be open to both corporate and academics applications.

The rest of the showcase featured progress reports by CIRM-funded scientists whose exciting stem cell therapeutics are entering or in early-stage clinical trails.

Here’s a breakdown of what was discussed:

– Neurodegeneration: Scientists from the Gladstone Institutes and Cedars-Sinai Medical center discussed new stem cell based approaches for the treatment of ALS and other neurodegenerative diseases

Retinal diseases: jCyte and Regenerative Patch Technologies discussed stem cell derived retinal tissue transplants to treat retinitis pigmentosa and age-related macular degeneration


Paul Laikind, President and CEO of ViaCyte

ViaCyte’s VC-01 islet implantation device for treatment of Type 1 diabetes is currently in Phase 1/2 clinical trials. Their first patient was successfully transplanted two weeks ago

Sangamo BioSciences is “engineering genetic cures” and mentioned recent therapeutic programs for Beta-thalassemia and HIV using genome targeting zinc finger proteins

Capricor is advancing its cardiosphere derived cell (CDC) therapy for cardiovascular disease and Duchenne’s muscular dystrophy. (Check out their ALLSTAR and CADUCEUS clinical trials)

Asterias Biotherapeutics is picking up from where Geron left off and pursuing stem cell therapies for spinal cord injury and cancer immunotherapy. (Check out their AST-OPC-1 first-in-man trial for spinal cord injury)

Overall, the event was an exciting preview of what’s to come in the field of stem cell therapeutics. It was well attended and brought together some of the major thought-leaders in the field of regenerative medicine.

“The CIRM showcase provided a great opportunity for current grantees to present a snapshot of the status of their funded research,” said Nick Mordwinkin, former postdoc at Stanford and now technical sales consultant at Miltenyi Biotec. “The attendees included a diverse mix of academic researchers, biotech executives, as well as life science investors. In order to maintain its success and keep California at the forefront of stem cell research in the United States, CIRM will have to continue to fund high impact academic translational research, as well as make a greater effort to reach biotechnology companies conducting cutting-edge research.”


Linda Marban, President and CEO of Capricor Therapeutics

November 6th, 2014|