Progress toward therapies: Projects in the pipeline

These active[1] disease-specific projects are in the later stages of moving toward clinical trials.

Find Out More:

Visit our Clinical Trials page for a complete list of all CIRM-funded clinical trials.
A complete list of all awards in a disease area, including those at an earlier stage of development at our Disease Fact Sheets.
The steps involved in developing a therapy, and the basic research that underlies all therapy development work on our Path to the Clinic page.

Descriptions of projects in development [pdf]

Active 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 Board that have not yet been issued an award agreement, the Approved Funds will temporarily reflect the amount approved by the governing Board.

Disease areasort descendingTitleApproved fundsPhase
Alzheimer's DiseaseIdentifying Drugs for Alzheimer's Disease with Human Neurons Made From Human IPS cells$1,774,420
Alzheimer's DiseaseStem cell based small molecule therapy for Alzheimer's disease $1,673,757
Age-related Muscle AtrophyLocal Delivery of Rejuvenated Old Muscle Stem Cells to Increase Strength in Aged Patients$1,825,920
Airway stenosisTissue Engineered Recellularized Laryngotracheal Implants$4,439,921
ALSStem Cells Secreting GDNF for the Treatment of ALS$63,487
ALSMolecules to Correct Aberrant RNA Signature in Human Diseased Neurons$1,532,323
ALSDevelopment of Novel Autophagy Inducers to Block the Progression of and Treat Amyotrophic Lateral Sclerosis (ALS) and Other Neurodegenerative Diseases$2,278,080
AML or Solid TumorClinical Investigation of a Humanized Anti-CD47 Antibody in Targeting Cancer Stem Cells in Hematologic Malignancies and Solid Tumors$7,672,364
Articular cartilage defectsTissue engineered cartilage from autologous, dermis-isolated, adult, stem (DIAS) cells$1,735,703
Autism Spectrum Disorder (ASD)A drug-screening platform for autism spectrum disorders using human astrocytes$1,656,456
Autoimmune Disease / Multiple SclerosisMultiple Sclerosis therapy: Human Pluripotent Stem Cell-Derived Neural Progenitor Cells$4,590,219
Autoimmune Disease / Multiple SclerosisTargeting Stem Cells to Enhance Remyelination in the Treatment of Multiple Sclerosis$2,623,242
b-thalassemiaA Treatment For Beta-thalassemia via High-Efficiency Targeted Genome Editing of Hematopoietic Stem Cells$2,760,540
Bone fracturesGene Targeting to Endogenous Stem Cells for Segmental Bone Fracture Healing$5,180,674
Cardiovascular DiseaseHeart Repair with Human Tissue Engineered Myocardium$4,397,241
Cardiovascular DiseaseHuman ES cell based therapy of heart failure without allogenic immune rejection$1,857,600
Cardiovascular DiseaseDirect Cardiac Reprogramming for Heart Regeneration$6,319,110
Cardiovascular DiseaseExtracellular Matrix Bioscaffold Augmented with Human Stem Cells for Cardiovascular Repair$4,633,149
Cardiovascular Disease - Danon diseaseIdentification of Novel Therapeutics for Danon Disease Using an iPS Model of the Disease$1,701,575
Cardiovascular, Vascular DiseaseDirect reprogramming towards vascular progenitors for the treatment of ischemia$2,340,000
Chronic Diabetic foot ulcersBone Marrow Mesenchymal Stem Cells to Heal Chronic Diabetic Wounds$4,635,576
CLLTherapeutic Eradication of Cancer Stem Cells$4,179,598
Conditioning regimen for allogeneic HSC transplantation for X-SCID A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants $19,068,382
Critical limb ischemia Phase I study of IM Injection of VEGF-Producing MSC for the Treatment of Critical Limb Ischemia$3,731,362
Diabetes: Type 1Preclinical and clinical testing of a stem cell-based combination product for insulin-dependent diabetes$10,075,070
Duchenne muscular dystrophy Combination therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy$1,823,545
End stage heart failure with LVAD Human Embryonic Stem Cell-Derived Cardiomyocytes for Patients with End Stage Heart Failure$73,030
End stage heart failure with LVAD Human Embryonic Stem Cell-Derived Cardiomyocytes for Patients with End Stage Heart Failure$19,942,877
GlioblastomaTargeting glioma cancer stem cells with receptor-engineered self-renewing memory T cells$5,217,004
Heart dysfunction after MI/Chronic heart failureAllogeneic Cardiac-Derived Stem Cells for Patients Following a Myocardial Infarction$19,782,136
Hemophilia BDevelopment of a cell and gene based therapy for hemophilia$2,298,634
HIV/AIDSA Phase I, Open-Label Study to Assess the Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells with Escalating Doses of Busulfan in HIV-1 (R5) Infected Subjects with Suboptim$5,583,438
HIV/AIDSStem Cell Programming With Chimeric Antigen Receptors to Eradicate HIV Infection$4,925,166
Huntington's DiseaseA hESc-based Development Candidate for Huntington's Disease$4,045,253
Huntington’s Disease MSC engineered to produce BDNF for the treatment of Huntington's disease$9,876,933
Limb Girdle Muscular Dystrophy Type 28Engineered iPSC for therapy of limb girdle muscular dystrophy type 2B$1,876,253
Liver Disease, ChronicGeneration of safe and therapeutically effective human induced hepatocyte-like cells$1,544,170
Liver Disease, CongenitalGeneration of hepatic cell from placental stem cell for congenital metabolic disorders$1,750,375
Lysosomal Storage DiseaseImmune-Matched Neural Stem Cell Transplantation for Pediatric Neurodegenerative Disease$4,272,774
MelanomaGenetic Re-programming of Stem Cells to Fight Cancer$19,875,776
Osteoporosis Treatment of osteoporosis with endogenous Mesenchymal stem cells$19,999,867
Prostate cancerClinical Development of an N-cadherin Antibody to Target Cancer Stem Cells$4,075,668
Retinitis PigmentosaRetinal progenitor cells for treatment of retinitis pigmentosa$17,144,825
Retinitis Pigmentosa, Age-related macular degeneration (dry form)Restoring vision by sheet transplants of retinal progenitors and retinal pigment epithelium (RPE) derived from human embryonic stem cells (hESCs)$3,998,948
SCID-AGene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID$3,931,662
Sickle Cell DiseaseClinical Trial of Stem Cell Gene Therapy for Sickle Cell Disease$13,145,465
Sickle Cell DiseaseBeta-Globin Gene Correction of Sickle Cell Disease in Hematopoietic Stem Cells $1,652,076
Solid TumorA Phase I dose escalation and expansion clinical trial in patients with advanced solid tumors$6,924,317
Spinal Cord InjuryA Phase I/IIa Dose Escalation Safety Study of [REDACTED] in Patients with Cervical Sensorimotor Complete Spinal Cord Injury $14,323,318
Spinal Cord InjuryHuman ES cell-derived MGE inhibitory interneuron transplantation for spinal cord injury$1,623,251
Spinal Cord InjuryFunctional Neural Relay Formation by Human Neural Stem Cell Grafting in Spinal Cord Injury$4,600,447
StrokeProgramming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke$885,000
Urea Cycle DisordersGene therapy-corrected autologous hepatocyte-like cells from induced pluripotent stem cells for the treatment of pediatric single enzyme disorders$1,801,629
Urinary IncontinenceAutologous iPSC Therapy for Urinary Incontinence$5,025,428
Ventricular arhythmias Improving Existing Drugs for Long QT Syndrome type 3 (LQT3) by hiPSC Disease-in-Dish Model$6,361,369


[1] The list does not include therapy-focused projects that have closed