CIRM Hands Out $25 Million for Basic Research into Host of Diseases

Heart disease, HIV, cancer and more targeted with stem cells and gene therapy

Answering the “what if” question — Maria Millan, CIRM CEO — CIRM photo

The California stem cell agency awarded nearly $25 million today to search for treatments and techniques to deal with everything from ovarian cancer and heart disease to HIV and eye afflictions. 

The action came as directors ratified 17 awards approved earlier by its grant reviewers and added another two awards to the mix. The CIRM staff recommended that the two be approved despite scores below the 85 cutoff. Reviewers were nearly evenly split on the proposals. 

All the applications were for early-stage research in what CIRM calls its Quest program. It will likely take years before the proposals ever reach clinical-stage testing, if they survive that long, according to CIRM.  But even research that does not reach patients can be useful to scientists. 

“Every therapy that helps save lives or change lives begins with a researcher asking a simple question, “What if?” said Maria T. Millan, president and CEO of CIRM, said in a news release. “Our Quest awards reflect the need to keep supporting early-stage research, to gain a deeper understanding of how stem cells work and how we can best tap into that potential to advance the field.”

The awards that were not approved by reviewers, who make the de facto decisions on applications, were 13510, which involves HIV, and 13475, which involves Duchenne muscular dystrophy. Both scored 84 on a scale of 100.

Only 21 applications survived CIRM’s review process out of 75 applications in this round. Twenty-one (cq) were cut through an internal CIRM review process without being submitted to a full review.

Summaries of the reviews of all the applications that received a full review can be found here. The summaries do not carry the names of the researchers or their institutions. However, the names of the winners and their application numbers can be found below. CIRM does not identify applicants who were not funded. 

Here is the list of award winners:

DISC2-13400

Targeted Immunotherapy-Based Blood Stem Cell Transplantation

Judy Shizuru, Stanford University

$1,341,910

-0-

DISC2-13505

Combating Ovarian Cancer Using Stem Cell-Engineered Off-The-Shelf CAR-iNKT Cells

Lili Yang, UCLA

$1,404,000

-0-

DISC2-13515

A treatment for Rett syndrome using glial-restricted neural progenitor cells

Alysson Muotri, UC San Diego

$1,402,240

-0—

DISC2-13454

Targeting pancreatic cancer stem cells with DDR1 antibodies.

Michael Karin, UC San Diego

$1,425,600

-0-

DISC2-13483

Enabling non-genetic activity-driven maturation of iPSC-derived neurons

Alex Savtchenko, Nanotools Bioscience

$675,000

-0-

DISC2-13405

Hematopoietic Stem Cell Gene Therapy for Alpha

Thalassemia

Don Kohn, UCLA

$1,323,007

-0-

DISC2-13507

CAR T cells targeting abnormal N-glycans for the

treatment of refractory/metastatic solid cancers

Michael Demetriou, UC Irvine

$1,414,800

-0-

DISC2-13463

Drug Development of Inhibitors of Inflammation Using Human iPSC-Derived Microglia (hiMG)

Stuart Lipton, Scripps Research Inst.

$1,658,123

-0-

-0-

DISC2-13390

Cardiac Reprogramming Gene Therapy for Post-Myocardial Infarction Heart Failure

Timothy Hoey, Tenaya Therapeutics

$1,215,000

-0-

DISC2-13417

AAV-dCas9 Epigenetic Editing for CDKL5 Deficiency Disorder

Kyle Fink, UC Davis

$1,429,378

-0-

DISC2-13415

Defining the Optimal Gene Therapy Approach of

Human Hematopoietic Stem Cells for the Treatment of Dedicator of Cytokinesis 8 (DOCK8) Deficiency

Caroline Kuo, UCLA

$1,386,232

-0-

DISC2-13498

Bioengineering human stem cell-derived beta cell organoids to monitor cell health in real time and improve therapeutic outcomes in patients

Katy Digovich, Minutia, Inc.

$1,198,550

-0-

DISC2-13469

Novel antisense therapy to treat genetic forms of

neurodevelopmental disease.

Joseph Gleeson, UC San Diego

$1,180,654

-0-

DISC2-13428

Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS)

Michael Bollong, Scripps Research Inst.

$1,244,160

-0-

DISC2-13456

Novel methods to eliminate cancer stem cells

Dinesh Rao, UCLA

$1,384,347

-0-

DISC2-13441

A new precision medicine based iPSC-derived model to study personalized intestinal fibrosis treatments in pediatric patients with Crohn’s disease

Robert Barrett Cedars-Sinai

$776,340

-0-

DISC2-13512

Modified RNA-Based Gene Therapy for Cardiac

Regeneration Through Cardiomyocyte Proliferation

Deepak Srivastava, Gladstone Institutes

$1,565,784

-0-

DISC2-13510

An hematopoietic stem-cell-based approach to treat HIV employing CAR-T cells and anti-HIV broadly neutralizing antibodies

Brian Lawson, The Scintillon Institute

$1,143,600

-0-

DISC2-13475

Developing gene therapy for dominant optic atrophy using human pluripotent stem cell-derived retinal organoid disease model

Xian-Jie Yang, UCLA

$1,345,691

Labels: grantmaking, basic research, awards