Progress toward therapies

Progress toward therapies: Projects in the pipeline

Download PDF handouts describing our progress:

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.

Therapy development projects

Disease area Funds committed Project goal
Amyotrophic Lateral Sclerosis Progenitor Cells Secreting GDNF for the Treatment of ALS $17,842,617 4- Early phase clinical trials
Amyotrophic Lateral Sclerosis Molecules to Correct Aberrant RNA Signature in Human Diseased Neurons $1,654,830 1- Proof of concept
Amyotrophic Lateral Sclerosis Stem Cell-Derived Astrocyte Precursor Transplants in Amyotrophic Lateral Sclerosis $10,857,762 3- FDA filing
Heart Disease Human ES cell based therapy of heart failure without allogenic immune rejection $1,857,600 1- Proof of concept
Heart Disease Allogeneic Cardiac-Derived Stem Cells for Patients Following a Myocardial Infarction $19,782,136 4- Early phase clinical trials
Heart Disease Human Embryonic Stem Cell-Derived Cardiomyocytes for Patients with End Stage Heart Failure $19,999,899 3- FDA filing
Heart Disease Direct reprogramming towards vascular progenitors for the treatment of ischemia $2,340,000 1- Proof of concept
Heart Disease Heart Repair with Human Tissue Engineered Myocardium $4,766,231 2- Development candidate
Heart Disease Extracellular Matrix Bioscaffold Augmented with Human Stem Cells for Cardiovascular Repair $4,939,140 2- Development candidate
Heart Disease Autologous cardiac-derived cells for advanced ischemic cardiomyopathy $5,560,232 3- FDA filing
Heart Disease Identification of Novel Therapeutics for Danon Disease Using an iPS Model of the Disease $1,701,575 1- Proof of concept
Heart Disease Direct Cardiac Reprogramming for Heart Regeneration $6,319,110 2- Development candidate
Alzheimer's disease Restoration of memory in Alzheimer’s disease: a new paradigm using neural stem cell therapy $20,000,000 3- FDA filing
Alzheimer's disease Identifying Drugs for Alzheimer's Disease with Human Neurons Made From Human IPS cells $1,857,600 1- Proof of concept
Alzheimer's disease Stem cell based small molecule therapy for Alzheimer's disease $1,673,757 1- Proof of concept
Alzheimer's disease Neural Stem Cells as a Developmental Candidate to Treat Alzheimer Disease $3,599,997 2- Development candidate
Arthritis Stem Cell-Based Therapy for Cartilage Regeneration and Osteoarthritis $3,118,431 2- Development candidate
Arthritis Cartilage Regeneration by the Chondrogenic Small Molecule PRO1 during Osteoarthritis $6,792,660 2- Development candidate
Blood Disorders Stem Cell Gene Therapy for Sickle Cell Disease $9,212,365 3- FDA filing
Blood Disorders A Phase 1/2, Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects with β-Thalassemia by Transplantation of Autologous Hematopoietic Stem Cells [REDACTED] $9,363,335 4- Early phase clinical trials
Bone or Cartilage Disease Enhancing healing via Wnt-protein mediated activation of endogenous stem cells $5,767,050 2- Development candidate
Bone or Cartilage Disease Tissue engineered cartilage from autologous, dermis-isolated, adult, stem (DIAS) cells $1,735,703 1- Proof of concept
Bone or Cartilage Disease Harnessing native fat-residing stem cells for bone regeneration $5,391,560 2- Development candidate
Bone or Cartilage Disease Treatment of osteoporosis with endogenous Mesenchymal stem cells $19,999,867 4- Early phase clinical trials
Bone or Cartilage Disease Systemic Adult Stem Cell Therapy for Osteoporosis-Related Vertebral Compression Fractures $1,927,698 1- Proof of concept
Blood cancer Development of Highly Active Anti-Leukemia Stem Cell Therapy (HALT) $19,999,826 3- FDA filing
Blood cancer Development of Therapeutic Antibodies Targeting Human Acute Myeloid Leukemia Stem Cells $19,999,996 3- FDA filing
Blood cancer Preclinical development of a pan Bcl2 inhibitor for cancer stem cell directed therapy $3,341,758 2- Development candidate
Blood cancer Dual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradication $3,607,305 2- Development candidate
Brain cancer Stem Cell-mediated Therapy for High-grade Glioma: Toward Phase I-II Clinical Trials $18,015,429 3- FDA filing
Brain cancer Targeting glioma cancer stem cells with receptor-engineered self-renewing memory T cells $5,217,004 2- Development candidate
Brain cancer Stem cell-based carriers for RCR vector delivery to glioblastoma $3,370,607 2- Development candidate
Melanoma Genetic Re-programming of Stem Cells to Fight Cancer $19,999,563 4- Early phase clinical trials
Solid Tumor THERAPEUTIC OPPORTUNITIES TO TARGET TUMOR INITIATING CELLS IN SOLID TUMORS $19,979,660 3- FDA filing
Diabetes Cell Therapy for Diabetes $22,999,937 3- FDA filing
Diabetes Preclinical and clinical testing of a stem cell-based combination product for insulin-dependent diabetes $10,075,070 4- Early phase clinical trials
HIV/AIDS ZIinc Finger Nuclease-Based Stem Cell Therapy for AIDS $14,583,187 3- FDA filing
HIV/AIDS Development of RNA-based approaches to stem cell gene therapy for HIV $3,124,130 2- Development candidate
HIV/AIDS HPSC based therapy for HIV disease using RNAi to CCR5. $9,905,604 3- FDA filing
Immune Disease Curing Hematological Diseases $6,649,347 2- Development candidate
Immune Disease Gene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID $3,931,662 2- Development candidate
Immune Disease A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants $20,000,000 4- Early phase clinical trials
Liver Disease Liver Cell Transplantation $5,199,767 2- Development candidate
Liver Disease Generation of hepatic cell from placental stem cell for congenital metabolic disorders $1,750,375 1- Proof of concept
Liver Disease Generation of safe and therapeutically effective human induced hepatocyte-like cells $1,544,170 1- Proof of concept
Muscular Dystrophy Stem Cell Therapy for Duchenne Muscular Dystrophy $2,325,933 1- Proof of concept
Muscular Dystrophy Combination Therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy $6,000,000 2- Development candidate
Autism Developing a drug-screening system for Autism Spectrum Disorders using human neurons $1,491,471 1- Proof of concept
Huntington's Disease MSC engineered to produce BDNF for the treatment of Huntington's disease $18,950,061 4- Early phase clinical trials
Huntington's Disease Sustained siRNA production from human MSC to treat Huntingtons Disease and other neurodegenerative disorders $2,753,559 2- Development candidate
Huntington's Disease A hESc-based Development Candidate for Huntington's Disease $3,799,817 2- Development candidate
Multiple Sclerosis Multiple Sclerosis therapy: Human Pluripotent Stem Cell-Derived Neural Progenitor Cells $4,799,814 2- Development candidate
Multiple Sclerosis Targeting Stem Cells to Enhance Remyelination in the Treatment of Multiple Sclerosis $4,327,175 2- Development candidate
Parkinson's disease Crosstalk: Inflammation in Parkinson’s disease (PD) in a humanized in vitro model $2,472,839 1- Proof of concept
Parkinson's disease Banking transplant ready dopaminergic neurons using a scalable process $6,016,624 2- Development candidate
Parkinson's disease Developmental Candidates for Cell-Based Therapies for Parkinson's Disease (PD) $5,416,003 2- Development candidate
Spinal Muscular Atrophy New Drug Discovery for SMA using Patient-derived Induced Pluripotent Stem Cells $5,665,887 2- Development candidate
Spinal Cord Injury Functional Neural Relay Formation by Human Neural Stem Cell Grafting in Spinal Cord Injury $4,699,569 2- Development candidate
Spinal Cord Injury Repair of Conus Medullaris/Cauda Equina Injury using Human ES Cell-Derived Motor Neurons $1,614,441 1- Proof of concept
Spinal Cord Injury Neural stem cell transplantation for chronic cervical spinal cord injury $20,000,000 3- FDA filing
Spinal Cord Injury Human ES cell-derived MGE inhibitory interneuron transplantation for spinal cord injury $1,623,251 1- Proof of concept
Stroke Embryonic-Derived Neural Stem Cells for Treatment of Motor Sequelae following Sub-cortical Stroke $20,000,000 3- FDA filing
Pediatrics Immune-Matched Neural Stem Cell Transplantation for Pediatric Neurodegenerative Disease $5,509,978 2- Development candidate
Skeletal Muscle Local Delivery of Rejuvenated Old Muscle Stem Cells to Increase Strength in Aged Patients $1,825,920 1- Proof of concept
Skin Disease iPS Cell-Based Treatment of Dominant Dystrophic Epidermolysis Bullosa $11,709,574 3- FDA filing
Skin Disease Bone Marrow Mesenchymal Stem Cells to Heal Chronic Diabetic Wounds $4,526,900 2- Development candidate
Trauma Neural restricted, FAC-sorted, human neural stem cells to treat traumatic brain injury $1,708,549 1- Proof of concept
Vision Loss Retinal progenitor cells for treatment of retinitis pigmentosa $17,306,668 4- Early phase clinical trials
Vision Loss Autologous Retinal Pigmented Epithelial Cells Derived from Induced Pluripotent Stem Cells for the Treatment of Atrophic Age Related Macular Degeneration $5,945,738 2- Development candidate
Vision Loss Regeneration of Functional Human Corneal Epithelial Progenitor Cells $1,654,058 1- Proof of concept
Vision Loss Development of a Stem Cell-based Transplantation Strategy for Treating Age-related Macular Degeneration $5,503,069 2- Development candidate
Vision Loss Stem cell based treatment strategy for Age-related Macular Degeneration (AMD) $18,904,916 3- FDA filing
Vision Loss Human retinal progenitor cells as candidate therapy for retinitis pigmentosa $4,738,251 2- Development candidate
Epilepsy Inhibitory Nerve Cell Precursors: Dosing, Safety and Efficacy $1,752,058 1- Proof of concept
Canavan disease Developing a therapeutic candidate for Canavan disease using induced pluripotent stem cell $1,731,750 1- Proof of concept
Vascular disease Phase I study of IM Injection of VEGF-Producing MSC for the Treatment of Critical Limb Ischemia $14,184,595 4- Early phase clinical trials
Incontinence Autologous iPSC Therapy for Urinary Incontinence $5,279,607 2- Development candidate
Total:
$615,593,627.00
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© 2013 California Institute for Regenerative Medicine