Stem cell therapies in discovery

Progress toward therapies: Projects in the pipeline

Therapies in the Pipeline | Path to the Clinic | What we Fund | How we Accelerate Therapies

CIRM's therapy development portfolio [pdf]

Our projects focusing on specific disease therapies have the end goal of generating proof of concept for their therapeutic approach, finding a candidate drug or cell type to be developed into a therapy or filing an investigation new drug (IND) application with the Food and Drug Administration to begin clinical trials. In future years, the agency will fund increasing amounts of new therapy development in different diseases.

Learn more about these steps, and the basic research that underlies all therapy development work, by reading our Path to the Clinic background.

In addition to the grants listed below, you can learn more about CIRM funding in specific disease areas by visiting our disease fact sheets.

Projects moving toward disease therapies

PC=Proof of Concept DC=Development Candidate IND=Investigational New Drug Application

Disease area Grant title Total Funds Project Goal
Blood disease Stem Cell Gene Therapy for Sickle Cell Disease $9,212,365 3-IND
Blood disease Curing Hematological Diseases $6,649,347 2-DC
Bone disease Systemic Adult Stem Cell Therapy for Osteoporosis-Related Vertebral Compression Fractures $1,927,698 1-PC
Bone disease Harnessing native fat-residing stem cells for bone regeneration $5,391,560 2-DC
Cancer: brain Stem Cell-mediated Therapy for High-grade Glioma: Toward Phase I-II Clinical Trials $18,015,429 3-IND
Cancer: brain Stem cell-based carriers for RCR vector delivery to glioblastoma $3,370,607 2-DC
Cancer: leukemia Development of Highly Active Anti-Leukemia Stem Cell Therapy (HALT) $19,999,826 3-IND
Cancer: leukemia Development of Therapeutic Antibodies Targeting Human Acute Myeloid Leukemia Stem Cells $19,999,996 3-IND
Cancer: leukemia Preclinical development of a pan Bcl2 inhibitor for cancer stem cell directed therapy $3,341,758 2-DC
Cancer: leukemia Dual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradication $3,607,305 2-DC
Cancer: solid tumor Therapeutic Opportunities To Target Tumor initiating Cells In Solid Tumors $19,979,660 3-IND
Cartilage disease Stem Cell-Based Therapy for Cartilage Regeneration and Osteoarthritis $3,118,431 2-DC
Cartilage disease Cartilage Regeneration by the Chondrogenic Small Molecule PRO1 during Osteoarthritis $6,792,660 2-DC
Diabetes Cell Therapy for Diabetes $19,999,937 3-IND
Eye disease Stem cell based treatment strategy for Age-related Macular Degeneration (AMD) $15,904,916 3-IND
Eye disease Autologous Retinal Pigmented Epithelial Cells Derived from Induced Pluripotent Stem Cells for the Treatment of Atrophic Age Related Macular Degeneration $5,945,738 2-DC
Eye disease Development of a Stem Cell-based Transplantation Strategy for Treating Age-related Macular Degeneration $5,503,069 2-DC
Eye disease Regeneration of Functional Human Corneal Epithelial Progenitor Cells $1,654,058 1-PC
Eye disease Human retinal progenitor cells as candidate therapy for retinitis pigmentosa $3,855,277 2-DC
Heart disease Autologous cardiac-derived cells for advanced ischemic cardiomyopathy $5,560,232 3-IND
HIV/AIDS HPSC based therapy for HIV disease using RNAi to CCR5. $19,999,580 3-IND
HIV/AIDS ZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS $14,583,187 3-IND
HIV/AIDS Development of RNA-based approaches to stem cell gene therapy for HIV $3,124,130 2-DC
Liver disease Liver Cell Transplantation $5,199,767 2-DC
Multiple diseases Enhancing healing via Wnt-protein mediated activation of endogenous stem cells $5,767,050 2-DC
Muscle disease Stem Cell Therapy for Duchenne Muscular Dystrophy $2,325,933 1-PC
Neurological disorders: ALS Stem Cell-Derived Astrocyte Precursor Transplants in Amyotrophic Lateral Sclerosis $10,857,762 3-IND
Neurological disorders: Alzheimer's Neural Stem Cells as a Developmental Candidate to Treat Alzheimer Disease $3,599,997 2-DC
Neurological disorders: Autism Developing a drug-screening system for Autism Spectrum Disorders using human neurons $1,491,471 1-PC
Neurological disorders: Canavan disease Developing a therapeutic candidate for Canavan disease using induced pluripotent stem cell $1,731,750 1-PC
Neurological disorders: Epilepsy Inhibitory Nerve Cell Precursors: Dosing, Safety and Efficacy $1,752,058 1-PC
Neurological disorders: Huntington's Sustained siRNA production from human MSC to treat Huntingtons Disease and other neurodegenerative disorders $2,753,559 2-DC
Neurological disorders: Huntington's A hESc-based Development Candidate for Huntington's Disease $3,799,817 2-DC
Neurological disorders: Parkinson's 'Developmental Candidates' for Cell-Based Therapies for Parkinson's Disease (PD) $3,562,824 2-DC
Neurological disorders: Parkinson's Banking transplant ready dopaminergic neurons using a scalable process $6,016,624 2-DC
Neurological disorders: Parkinson's Crosstalk: Inflammation in Parkinson's disease (PD) in a humanized in vitro model $2,336,404 1-PC
Neurological disorders: Spinal cord injury Repair of Conus Medullaris/Cauda Equina Injury using Human ES Cell-Derived Motor Neurons $1,614,441 1-PC
Neurological disorders: Spinal muscular atrophy New Drug Discovery for SMA using Patient-derived Induced Pluripotent Stem Cells $5,665,887 2-DC
Neurological disorders: Stroke Embryonic-Derived Neural Stem Cells for Treatment of Motor Sequelae following Sub-cortical Stroke $20,000,000 3-IND
Neurological disorders: Trauma Neural restricted, FAC-sorted, human neural stem cells to treat traumatic brain injury $1,708,549 1-PC
Skin disease iPS Cell-Based Treatment of Dystrophic Epidermolysis Bullosa $11,709,574 3-IND
Skin disease Bone Marrow Mesenchymal Stem Cells to Heal Chronic Diabetic Wounds $4,526,900 2-DC