President and Chief Executive Officer C. Randal Mills, Ph.D. Clinical Portfolio Review
President and Chief Executive OfficerC. Randal Mills, Ph.D.
Clinical Portfolio Review
CIRM’s Seamless Pathway
2
Single ProductCandidate
NewIdea
Program Offerings Per Year
Pre-‐IND Meeting or Equivalent
ApprovedTherapy
2/Year 3/Year 12/Year
Availability12/Year
3
The Clinical Program Has Doubled
Pre-‐IND Meeting or Equivalent
ApprovedTherapy
All Meritorious Applications
CIRM Therapeutics Portfolio32 individual projects addressing serious conditions
422 Clinical Trials • 10 Pre-IND projects
Clinical TrialsNeurologic and Ophthalmic
Indication Investigator / OrganizationApproach
PhaseStatus
Spinal Cord Injury Lebkowski / AsteriashESC-derived oligodendrocyte progenitor cells
Phase 1/2Enrolling
Retinitis Pigmentosa Klassen / UC IrvineNeural progenitor cells for injection into eye
Phase 1/2Enrolling
Age-Related Macular Degeneration
Humayun / USChESC-derived retinal cells on a synthetic matrix
Phase 1Enrolling
ALS (Lou Gehrig's Disease)
Svendsen / Cedars-SinaiNeural progenitor cells expressing a protective factor
Phase 1/2Initiating
Huntington’s Disease Wheelock / UC DavisMonitor natural progression of Huntington’s disease Observational
Spinal Cord Injury Lebkowski / GeronhESC-derived oligodendrocyte progenitor cells
Phase 1 Closed
Clinical TrialsOrgan Systems
Indication Investigator / OrganizationApproach
PhaseStatus
Vascular Access Lawson / HumacyteEngineered blood vessel
Phase 3Enrolling
Myocardial Infarction Smith / CapricorHeart-derived progenitor cells
Phase 2Follow-up
Duchenne Muscular Dystrophy (Cardiac)
Ascheim / CapricorHeart-derived progenitor cells
Phase 2Follow-up
Type 1 Diabetes Foyt / ViacyteEncapsulated hESC-derived insulin-producing cells
Phase 1/2Enrolling
Osteonecrosis Lane / UC DavisSmall molecule recruiting endogenous bone stem cells
Phase 1/2 Enrolling
Clinical TrialsOncology
Indication Investigator / OrganizationApproach
PhaseStatus
Glioblastoma Gringeri / ImmunocellularAutologous cellular vaccine
Phase 3Enrolling
Solid Tumor Slamon / UCLASmall molecule inhibiting cancer stem cells
Phase 1Enrolling
Chronic Lymphocytic Leukemia
Kipps / UCSDAntibody inhibiting cancer stem cells
Phase 1Enrolling
Acute MyelogenousLeukemia
Weissman / StanfordAntibody inhibiting cancer stem cells
Phase 1Enrolling
Melanoma Dillman / CaladriusAutologous cellular vaccine
Phase 3 Closed
Clinical TrialsHematology
Indication Investigator / OrganizationApproach
PhaseStatus
Severe Combined Immunodeficiency
Shizuru / StanfordChemotherapy-free bone marrow transplant
Phase 1/2Enrolling
HIV/AIDS Symonds / CalimmuneStem cell gene therapy to create HIV resistance
Phase 1/2Follow-up
Chronic Granulomatous Disease
Kohn / UCLAStem cell gene therapy to fix enzyme in WBC
Phase 1/2Enrolling
Sickle Cell Disease Kohn / UCLAStem cell gene therapy to correct gene defect
Phase 1Enrolling
HIV/AIDS Abedi / UC DavisStem cell gene therapy for AIDS Lymphoma
Phase 1Enrolling
HIV/AIDSZaia / City of Hope
Stem cell gene editing to create HIV resistance Phase 1 Enrolling
Featured ProgramRetinitis Pigmentosa
INVESTIGATORHenry Klassen, MD, PhD
INSTITUTIONU.C. Irvine
AWARD$17 M to conduct a Ph 1/2 trial
APPROACHDirect injection of neural progenitor cells into the damaged retina to save and regrow rods and cones
Rosie NAME• Mother of twin girls and son• Diagnosed at age 26• Blind in both eyes at treatment• Left eye injected September of 2015• Visual acuity has improved to enable
reading
Featured ProgramSpinal Cord Injury
INVESTIGATORJane Lebkowski, PhD
INSTITUTIONAsterias Biotherapeutics
AWARD$14.3 M to conduct a Ph 1/2 trial
APPROACHDirect injection of hESC-derived oligodendrocyte progenitor cells into patients with neurologically complete cervical (C5-7) SCI.
Kris Boesen• Injured in a car crash March 6, 2016• Completely paralyzed from the neck
down• Treated with 10M cells in April, 2016• Regained two levels of motor and
sensory function
Featured ProgramChronic Granulomatous Disease
INVESTIGATORDonald B. Kohn, MD
INSTITUTIONUCLA
AWARD$7 M to conduct a Ph 1/2 trial
APPROACHAutologous bone marrow stem cell gene therapy to replace the defective enzyme in blood cells in patients with CGD.
Brenden Whittaker• Diagnosed at age one • Defective gene leads to weak immune
system unable to fight infections• Lost portions of lung and liver• December 2015 got transplant of his
own gene-modified blood stem cells
Accelerate stem cell treatments to patients with unmet medical needs.
MISS
ION CIRM’s Mission