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