2008-0471S1-4

Human Embryonic Stem Cells: Considerations for Therapeutic Development Jane Lebkowski Ph.D. Geron Corporation Cell, Tissue and Gene Therapy Advisory Committee Meeting April 10, 2008

Human Embryonic Stem Cells Large Characterized cGMP Banks Therapeutic Cells Blastocyst Human Embryonic Stem Cells Neural Cells Spinal Cord Injury Parkinson’s Disease Cardiomyocytes Heart Failure Islets Diabetes Osteoblasts Osteoporosis And Bone Fractures Chondrocytes Arthritis Hepatocytes Drug Discovery Liver Failure Dendritic Cells Tolerance Induction Cancer Immunotherapy

hESCs: New Cell Type for Potential Broad Therapeutic Application ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Necessary Technological Developments

Spinal Cord Injury: GRNOPC1 Transplantation

Differentiation Process ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

GRNOPC1 Contains Oligodendroglial Progenitors Phenotype Testing of Over 75 Lots ,[object Object],[object Object],[object Object],[object Object],[object Object],Challenges Identity Purity Strength Potency Lineage Marker Neural Progenitors Nestin Oligodendroglial Progenitors Olig 1 Oligodendroglial Progenitors NG2 Oligodendroglial Progenitors PDGFRa Early Ectoderm Pax 6 Early Ectoderm Sox 10 Neurons  TubIII Astrocytes GFAP Early Endoderm HNF3  Endoderm AFP Early Mesoderm GATA4 Mesoderm MSA Undifferentiated hESCs OCT4 Undifferentiated hESCs Tra-1-60

Considerations for Nonclinical Studies for hESC-Based Therapeutics ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Pharmacology Studies ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],What Is the Activity of the Cells? 9 mos GRNOPC1 hNuc EC 1mm 100  m 9 mos vehicle hNuc EC 1mm 100  m

Where Do The Cells Go? Safety and Efficacy Implications ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Toxicology Studies Toxicity of Delivery ,[object Object],[object Object],[object Object],[object Object],[object Object],GRNOPC1 ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Considerations

Tumorigenicity Studies ,[object Object],[object Object],[object Object],[object Object],Challenges ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Tumorigenicity Studies: Lessons Learned GRNOPC1 Deliberately Spiked with hESCs Important Factors In Teratoma Formation ,[object Object],[object Object],[object Object],2 x 10 6 Cells Intraspinal Cord Injection Assessment 12 mos.

Allogenicity Studies ,[object Object],[object Object],Challenges ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Approaches

Cell Delivery in the Clinic Considerations ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Design of Clinical Trials Key Consideration: Patient Safety ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],Interactions with:

Phase 1 Multi-Center Trial ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

Conclusions ,[object Object],[object Object],[object Object],[object Object],[object Object],[object Object]

2008-0471S1-4

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2008-0471S1-4