Progress toward therapies

Progress toward therapies: Projects in the pipeline

Download PDF handouts describing our progress:

The awards shown here are currently active disease-specific projects that are in the later stages of moving toward clinical trials (the list does not include therapy-focused projects that have closed). For a complete list of all awards in a disease area, including those at an earlier stage of development, visit our disease fact sheets.

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

CIRM therapy development pipeline

Therapy development projects

The Approved Funds represents the current award amount under agreement with the grantee which can be less the amount approved by the governing board. For projects approved by the governing that have not yet been issued an award agreement, the Approved Funds will temporarily reflect the amount approved by the governing board.
Disease area Approved funds Project goal
Amyotrophic Lateral Sclerosis Molecules to Correct Aberrant RNA Signature in Human Diseased Neurons $1,532,323 2- Development candidate
Amyotrophic Lateral Sclerosis Progenitor Cells Secreting GDNF for the Treatment of ALS $16,961,287 3- FDA filing
Amyotrophic Lateral Sclerosis Development of Novel Autophagy Inducers to Block the Progression of and Treat Amyotrophic Lateral Sclerosis (ALS) and Other Neurodegenerative Diseases $2,278,080 2- Development candidate
Amyotrophic Lateral Sclerosis Stem Cell-Derived Astrocyte Precursor Transplants in Amyotrophic Lateral Sclerosis $4,139,754 2- Development candidate
Alzheimer's disease Restoration of memory in Alzheimer’s disease: a new paradigm using neural stem cell therapy $8,901,641 3- FDA filing
Arthritis Cartilage Regeneration by the Chondrogenic Small Molecule PRO1 during Osteoarthritis $6,180,114 2- Development candidate
Blood Disorders Stem Cell Gene Therapy for Sickle Cell Disease $8,833,695 3- FDA filing
Bone or Cartilage Disease Enhancing healing via Wnt-protein mediated activation of endogenous stem cells $6,464,126 2- Development candidate
Bone or Cartilage Disease Harnessing native fat-residing stem cells for bone regeneration $5,359,076 2- Development candidate
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 Preclinical development of a pan Bcl2 inhibitor for cancer stem cell directed therapy $3,103,041 2- Development candidate
Blood cancer Development of Therapeutic Antibodies Targeting Human Acute Myeloid Leukemia Stem Cells $18,759,276 3- FDA filing
Blood cancer Therapeutic Eradication of Cancer Stem Cells $4,179,598 4- Early phase clinical trials
Blood cancer Dual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradication $2,756,536 2- Development candidate
Blood cancer Clinical Investigation of a Humanized Anti-CD47 Antibody in Targeting Cancer Stem Cells in Hematologic Malignancies and Solid Tumors $7,672,364 4- Early phase clinical trials
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
Brain cancer Stem Cell-mediated Therapy for High-grade Glioma: Toward Phase I-II Clinical Trials $18,015,429 3- FDA filing
Melanoma Genetic Re-programming of Stem Cells to Fight Cancer $19,875,776 3- FDA filing
Prostate cancer Clinical Development of an N-cadherin Antibody to Target Cancer Stem Cells $4,075,668 2- Development candidate
Solid Tumor A Phase I dose escalation and expansion clinical trial in patients with advanced solid tumors $6,924,317 4- Early phase clinical trials
Diabetes Cell Therapy for Diabetes $22,999,933 3- FDA filing
HIV/AIDS Development of RNA-based approaches to stem cell gene therapy for HIV $3,097,160 2- Development candidate
HIV/AIDS Zinc Finger Nuclease-Based Stem Cell Therapy for AIDS $14,573,835 3- FDA filing
Liver Disease Liver Cell Transplantation $4,215,748 2- Development candidate
Muscular Dystrophy Stem Cell Therapy for Duchenne Muscular Dystrophy $2,267,261 1- Proof of concept
Autism Developing a drug-screening system for Autism Spectrum Disorders using human neurons $1,376,198 1- Proof of concept
Huntington's Disease A hESc-based Development Candidate for Huntington's Disease $4,045,253 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 $4,998,871 2- Development candidate
Parkinson's disease Developmental Candidates for Cell-Based Therapies for Parkinson's Disease (PD) $5,190,752 2- Development candidate
Spinal Cord Injury Repair of Conus Medullaris/Cauda Equina Injury using Human ES Cell-Derived Motor Neurons $1,614,231 1- Proof of concept
Spinal Cord Injury Human ES cell-derived MGE inhibitory interneuron transplantation for spinal cord injury $1,623,251 2- Development candidate
Spinal Cord Injury Functional Neural Relay Formation by Human Neural Stem Cell Grafting in Spinal Cord Injury $4,600,447 2- Development candidate
Spinal Cord Injury A Phase I/IIa Dose Escalation Safety Study of [REDACTED] in Patients with Cervical Sensorimotor Complete Spinal Cord Injury $14,323,318 4- Early phase clinical trials
Stroke Embryonic-Derived Neural Stem Cells for Treatment of Motor Sequelae following Sub-cortical Stroke $18,026,648 3- FDA filing
Stroke Programming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke $2,124,000 2- Development candidate
Skin Disease iPS Cell-Based Treatment of Dominant Dystrophic Epidermolysis Bullosa $11,039,208 3- FDA filing
Skin Disease Bone Marrow Mesenchymal Stem Cells to Heal Chronic Diabetic Wounds $4,635,576 2- Development candidate
Trauma Neural restricted, FAC-sorted, human neural stem cells to treat traumatic brain injury $1,517,767 1- Proof of concept
Vision Loss Retinal progenitor cells for treatment of retinitis pigmentosa $17,144,825 3- FDA filing
Vision Loss Regeneration of Functional Human Corneal Epithelial Progenitor Cells $1,524,947 1- Proof of concept
Vision Loss Stem cell based treatment strategy for Age-related Macular Degeneration (AMD) $18,904,916 3- FDA filing
Epilepsy Inhibitory Nerve Cell Precursors: Dosing, Safety and Efficacy $1,564,016 1- Proof of concept
Canavan disease Developing a therapeutic candidate for Canavan disease using induced pluripotent stem cell $1,835,983 1- Proof of concept
Icon for xls export

© 2013 California Institute for Regenerative Medicine