This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform
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- Julie Davidson
- 5 years ago
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2 This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements include, but are not limited to, the design of clinical trials and expected timing for enrollment and presentation of data; the anticipated clinical development milestones and other potential value drivers in the future; the expected benefits of our collaborations, the expanded capability of Sangamo s technologies; the research and development of novel gene-based therapies and the application of Sangamo s ZFP technology platform to specific human diseases; successful manufacturing of our product candidates; the potential of Sangamo s genome editing technology to safely treat genetic diseases; the potential for ZFNs to be effectively designed to treat diseases through genome editing; plans to conduct controlled withdrawal of weekly ERT infusions in MPS II subjects in the CHAMPIONS Study; anticipated next steps for the CHAMPIONS and Alta Studies, and Sangamo s expectation that it will present longer-term safety and efficacy results from the CHAMPIONS Study in February at the 2019 WORLDSymposium meeting. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties. Factors that could cause actual results to differ include, but are not limited to, the dependence on the success of clinical trials of lead programs, the lengthy and uncertain regulatory approval process, uncertainties related to the timing of initiation, enrollment and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of Sangamo s therapeutics, risks and uncertainties related to preliminary data, whether the preliminary data from the CHAMPIONS and Alta Studies and will be representative of final results, whether the final results from the CHAMPIONS and Alta Studies will validate and support the safety and efficacy of SB-913 and SB-525 respectively, and the reliance on partners and other third-parties to meet their obligations. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable gene-based therapeutics. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo s operations. These risks and uncertainties are described more fully in Sangamo s Quarterly Reports on Form 10-Q for the quarter ended September 30, 2018 as filed with the Securities and Exchange Commission. Forward-looking statements contained in this presentation are made as of the date hereof, and Sangamo undertakes no obligation to update such information except as required under applicable law. 2
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7 Therapeutic Area Research Preclinical Phase I/II Clinical Inherited Metabolic Diseases Phase III Registrational Collaborator MPS I (SB-318) MPS II (SB-913) Hematology Hemophilia A (SB-525) Hemophilia B (SB-FIX) Hemoglobinopathies (ST-400, BIVV-003)
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13 Phase I/II Open Label Study 9 Adults (18+ yrs), 2+ adolescents (12-17 yrs) and 2+ children (5-11 yrs) with MPS II Dose Escalation Complete Cohort 1 Cohort 2 Cohort 3 October SMC Review / Recommendations Adult Cohort Expansion (5e13 vg/kg) Adult Cohort Expansion and Adolescent Screening Underway Next steps Interim data update from cohorts 1-3 at WORLDSymposium 2019 Initiate Adolescent Dosing (12-17 years) Complete ERT withdrawal Complete adult expansion cohort enrollment 5e12 vg/kg 1e13 vg/kg 5e13 vg/kg Initiate ERT withdrawal 5e13 vg/kg Adolescent cohort enrollment Orphan Drug Fast Track Rare Pediatric Disease Orphan Medicinal Product IND open CTA granted Goals Patient safety Reduction in GAGs IDS enzyme production ERT withdrawal 13
14 Phase I/II Open Label Study Up to 9 adults (18+ yrs) with MPS I Dose Escalation Complete October SMC Review / Recommendations Next steps Cohort 1 Cohort 2 Cohort 3 Advance Enrollment to the 5e13 vg/kg Dose Cohort Preliminary data from first 3 patients at WORLDSymposium 2019 SMC review and recommendations 5e12 vg/kg 1e13 vg/kg 5e13 vg/kg Orphan Drug Fast Track Rare Pediatric Disease Orphan Medicinal Product IND open CTA granted Goals Patient safety Reduction in GAGs IDS enzyme production ERT withdrawal 14
15 First update: SB-913 and SB-318 interim data presentations February 7 th - WORLDSymposium SB-913 (MPS II): Update on safety and biochemical changes at up to 24 weeks from 6 adult patients (low, mid and high-dose cohorts) - SB-318 (MPS I): Preliminary safety and biochemical changes at up to 4 weeks from first 3 adult patients (mid and high-dose cohorts) Potential additional updates throughout 2019 as programs progress and data accumulate Cohort expansion data Liver biopsy analyses ERT withdrawal Adolescent cohort updates Outcomes measurements 15
16 Phase I/II Open Label Study Up to 20 adult (18+ yrs) males with severe hemophilia A Dose Escalation Ongoing October SMC Review and Recommendations Next steps Cohort 1 Cohort 2 Cohort 3 Cohort 4 Continue Escalation to an Complete cohort 4 enrollment Additional Dose SMC review and recommendation Orphan Drug Orphan Medicinal Product Fast Track IND open Goals Patient safety FVIII activity Reduction of bleeding events Reduction of factor replacement use 16
17 Phase I/II Open Label Study Up to 12 adult (18+ yrs) males and 3 adolescents (12-17 yrs) with severe hemophilia B Dose Escalation Ongoing October SMC Review and Recommendations Next steps Cohort 1 Cohort 2 Cohort 3 Begin Adult Enrollment at the 5e13 vg/kg Dose Cohort Complete 5e13 vg/kg cohort enrollment SMC review and recommendation 5e12 vg/kg 1e13 vg/kg 5e13 vg/kg Orphan Drug Orphan Medicinal Product Fast Track IND open CTA granted Goals Patient safety FIX activity Reduction of bleeding events Reduction of factor replacement use 17
18 Study Update - SMC cohort expansion recommendation Study Update - Patient enrollment and SMC recommendations Data Update - Following completion of dose escalation - Safety and factor levels, factor replacement utilization, bleeding events Data Update - Following SMC review of first 2 patients in 5e13 vg/kg dose cohort - Safety and factor levels 18
19 Phase I/II Open Label Study 6 adults (18+) with transfusion-dependent beta thalassemia Patient Enrollment Ongoing Potential Advantages Leverages naturally-occurring, protective mechanism to increase fetal-hemoglobin to reduce or potentially eliminate blood transfusions Highly efficient, precise gene editing; low risk of insertional mutagenesis Next steps Complete enrollment of 6 patients Non-viral delivery of ZFNs IND open Goals Patient safety Successful engraftment Fetal hemoglobin (HbF) production Reduction / elimination of transfusions 19
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21 Therapeutic Area Research Preclinical Phase I/II Clinical Inherited Metabolic Diseases Collaborator Fabry Disease (ST-920) Immunology Solid Organ Transplant (Tx200) CNS Diseases Tauopathies ALS/FTLD - C9ORF72 Oncology Huntington s Disease Autologous and Allogeneic CAR/TCR/NKR
22 Nmol/hr/mg protein %Gb3 Remaining α Tissue α-gal A Activity (2 months) 100 Gb3 in Tissues *216x 17x 21x 3x Wild Type Untreated ST Plasma Liver Heart Kidney 25% Remaining 1 Liver Spleen Heart Kidney *ST-920 compared to Wild Type 0 Untreated ST-920 WT GLAKO 10% Remaining 22
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25 CAR TCR IL2 IL2R CCR Inhibitory cytokines Treg FoxP3 FoxP3 FoxP3 IL6R HLA 25
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29 COOH NH 2 5 T A C C C A 3 29
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31 Efficiency Precision Specificity New linkers for configuring DNA-binding modules 300-fold increase in design options for targeting any given sequence Efficiency Precision Specificity New dimer architectures yield higher modification activity Increase DNA editing efficiency to as high as 99.5% Precision Efficiency Specificity Phosphate contact tuning via replacement of key residues Off-target cleavage undetectable (>1000 fold reduction) 31
32 ± G A A G C T a G T C T A G t G C A A G C X T G C T T T T A T C A C A G G C T C C T A G T C t A G T G C A A G C X T G C T T T T A T C A C A G G C T C G A A G C T a G T C T A G t G C A A G C X G C T T T T A T C a C A G G C T C T A G T C T A G T G C a A G C T A A X C T T T T A T C A C A G G C T C T A G T C T A G T G C a A G C T A A X T T T T A T C A C A G G C T C G T C T A G T G C a A G C T A A X T T T T A T C A C A G G C T C G T C T A G t G C A A G C T A A X T T T T A T C A C A G G C T C C T A G T G C A A G C T A A C X T T T A T C A C A G G C T C C C T A G T C t A G T G C A a G C T A A C X T T T A T C A C A G G C T C C C T A G T G C A A G C T A A C X T T T T A T C A C A G G C T C G T C T A G t G C A A G C T A A X T T T A T C A C A G G C T C C T G C A A G C T A A C A X T T T A T C A C A G G C T C C C T A G T C t A G T G C A a G C T A A C X T A T C A C A G G C T C C A G C T A G T G C A A G C T A A C X T A T C A C A G G C T C C A G G T G C A A G C T A A C A G T X T A T C A C A G G C T C C A G G T C T A G t G C A A G C T A A C A G X T A T C A C A G G C T C C A G G T C T A G t G C A A G C T A A C A G X A T C A C A G G C T C C A G G G T G C A A G C T A A C A G T X T A T C A C A G G C T C C A G T A G T G C a A G C T A A C A G X T A T C A C A G G C T C C A G G T C T A G t G C A A G C T A A C A G X T A T C A C A G G C T C C A G G T G C A A G C T A A C A G T X A T C A C A G G C T C C A G G T A G T G C a A G C T A A C A G X T C A C A G g C T C C A G G A A G G G T A G T G C a A G C T A A C A G T T G X T C A C A G g C T C C A G G A A G G G G T G C A A G C T A A C A G T X T C A C A G g C T C C A G G A A G G G T G C A A G C T A A C A G T T X A T C A C A G G C T C C A G G G T G C A A G C T a A C A G T T X T C A C A G g C T C C A G G A A G G G G T G C A A G C T A A C A G T X C A C A G G C T C C A G G A A G G G T G C A A G C T A A C A G T T X T C A C A G g C T C C A G G A A G G G T A G T G C a A G C T A A C A G T T G X T C A C A G G C T C C A G G A G T G C A A G C T A A C A G T X T C A C A G G C T C C A G G A T G C A A G C T A A C A G T T X C A C A G G C T C C A G G A A G G G G T G C A A G C T a A C A G T T X T C A C A G G C T C C A G G A T G C A A G C T A A C A G T T X T C A C A G G C T C C A G G A G T G C A A G C T A A C A G T X C A C A G G C T C C A G G A A T G C A A G C T A A C A G T T X C A C A G G C T C C A G G A A G T G C A A G C T A A C A G T T G C X T C A C A G g C T C C A G G A A G G G G T G C A A G C T A A C A G T T G C X T C A C A G G C T C C A G G A T G C A A G C T A A C A G T T X A C A G G C T C C A G G A A G G G T T G C A A G C T A A C A G T T X A C A G G C T C C A G G A A G T A G T G C a A G C T A A C A G T T G X C A C A G G C T C C A G G A A G G G T A G T G C a A G C T A A C A G T T G X C A C A G G C T C C A G G A A G T G C A A G C T A A C A G T T G C X C A G G C T C C A G G A A G G G T T G T G C A A G C T A A C A G T T G C X C A C A G G C T C C A G G A A G G G G T G C A A G C T A A C A G T T G C X A C A G G C T C C A G G A A G G G T G T G C A A G C T A A C a G T T G C T X A C A G G C T C C A G G A A G G G T G T G C A A G C T A A C A G T T G C X A C A G G C T C C A G G A A G G T G C A A G C T A A C a G T T G C T X A C A G G C T C C A G G A A G G T G C A A G C T A A C A G T T G C X C A G G C T C C A G G A A G G G T G C A A G C T A A C a G T T G C T X A C A G G C T C C A G G A A G G G T G T G C A A G C T A A C a G T T G C T X A C A G G C T C C A G G A A G G T G C A A G C T A A C A G T T G C X C A C A G G C T C C A G G A A T A G T G C a A G C T A A C A G T T G X C A G G C T C C A G G A A G G G T T G T G C A A G C T A A C a G T T G C T X C A C A G G C T C C A G G A A G G G T A G T G C a A G C T A A C A G T T G X A C A G G C T C C A G G A A G G G T T A G T G C a A G C T A A C A G T T G X A C A G G C T C C A G G A A G T A G T G C a A G C T A A C A G T T G X C A G G C T C C A G G A A G G G T G C A A G C T A A C a G T T G C T X C A C A G G C T C C A G G A A G T G C A A G C T A A C a G T T G C T X C A G G C T C C A G G A A G G G T T G T G C A A G C T A A C a G T T G C T X C A G G C T C C A G G A A G G G T T G T G C A A G C T A A C a G T T G C T X C A G G C T C C A G G A A G G G T G C A A G C T A A C a G T T G C T X C A G G C T C C A G G A A G G G T G C A A G C T A A C A G T T G C X A G G C T C C A G G A A G G G G C T A A C A G T T G C T T T X C A G G C T C C A G G A A G G G T T A G C T A A C A G T T G C T T X C A G G C T C C A G G A A G G G T T G T G C A A G C T A A C a G T T G C T X G G C T C C A G G A A G G G T G T G C A A G C T A A C a G T T G C T X A G G C T C C A G G A A G G G G C T A A C A G T T G C T T T X A G G C T C C A G G A A G G G G C T A A C A G T T G C T T T X C A G G C T C C A G G A A G G A G C T A A C A G T T G C T T X C A G G C T C C A G G A A G G G C T A A C A G T T G C T T T X G G C T C C A G G A A G G G T G C T A A C A G T T G C T T T X C T C C A G G A A G G G t T T G G C C G C T A A C A G T T G C T T T X G C T C C A G G A A G G G T T A G C T A A C A G T T G C T T T T A X G C T C C A G G A A G G G T T G C T A A C A G T T G C T T T T A T X G C T C C A G G A A G G G T T A G C T A A C A G T T G C T T T T A X C T C C A G G A A G G G t T T G G C C G C T A A C A G T T G C T T T T A T X C T C C A G G A A G G G t T T G G C C G C T A A C a G T T G C T T T T A T C X T C C A G G A A G G G T T T G G C C G C T A A C A G T T G C T T T T A T X T C C A G G A A G G G T T T G G C C G C T A A C A G T T G C T T T T A T X C C A G G A A G G G T T T G G G C T A A C a G T T G C T T T T A T C X C T C C A G G A A G G G t T T G G C C A G C T A A C A G T T G C T T T T A X T C C A G G A A G G G T T T G G C C A G C T A A C A G T T G C T T T T A X C C A G G A A G G G T T T G G G C T A A C a G T T G C T T T T A T C X C C A G G A A G G G T T T G G G C T A A C A G T T G C T T T T A T X C A G G A A G G G t T T G G C C G C T A A C a G T T G C T T T T A T C X A G G A A G G G T T T G G C C A A C A G T T G C T T T T A T C A C X C A G G A A G G G t T T G G C C G C T A A C a G T T G C T T T T A T C X C A G G A A G G G t T T G G C C A A C A G T T G C T T T T A T C A C X C C A G G A A G G G T T T G G C A G T T G C T T T T A T C A X C C A G G A A G G G T T T G G C A G T T G C T T T T A T C A X A G G A A G G G T T T G G C C A A C A G T T G C T T T T A T C A C X A G G A A G G G T T T G G C C A A C A G T T G C T T T t A T C A C A X C A G G A A G G G t T T G G C C A A C A G T T G C T T T T A T C A C X G A A G G G t T T G G C C T C T G A T A A C A G T T G C T T T t A T C A C A X A G G A A G G G T T T G G C C A A C A G T T G C T T T t A T C A C A X G A A G G G t T T G G C C T C T G A T A A C A G T T G C T T T T A T C A C X G G A A G G g t T T G G C C T C T G A T A A C A G T T G C T T T t A T C A C A X G G A A G G g t T T G G C C T C T G A T A A C A G T T G C T T T t A T C A C A X A A G G G T T T G G C C T C T G A T A G T T G C T T T T A T C A C A G G X G G A A G G g t T T G G C C T C T G A T A G T T G C T T T T A T C A C A G G X G A A G G G t T T G G C C T C T G A T A G T T G C T T T T A T C A C A G G X A A G G G T T T G G C C T C T G A T T G C T T T t A T C A C A G G C X A A G G G T T T G G C C T C T G A T T G C T T T t A T C A C A G G C X G A A G G G t T T G G C C T C T G A T G T T G C T T T T A T C a C A G G C T X A A G G G T T T G G C C T C T G A T TTGAAGCTAGTCTAGTGCAAGCTAACAGTTGCTTTTATCACAGGCTCCAGGAAGGGTTTGGCCTCTGATTAGGG AACTTCGATCAGATCACGTTCGATTGTCAACGAAAATAGTGTCCGAGGTCCTTCCCAAACCGGAGACTAATCCC 32 G A A G C T a G T C T A G t G C A A G C X T G C T T T T A T C A C A G G C T C C T A G T C t A G T G C A A G C X T G C T T T T A T C A C A G G C T C G A A G C T a G T C T A G t G C A A G C X G C T T T T A T C a C A G G C T C T A G T C T A G T G C a A G C T A A X C T T T T A T C A C A G G C T C T A G T C T A G T G C a A G C T A A X T T T T A T C A C A G G C T C G T C T A G T G C a A G C T A A X T T T T A T C A C A G G C T C G T C T A G t G C A A G C T A A X T T T T A T C A C A G G C T C C T A G T G C A A G C T A A C X T T T A T C A C A G G C T C C C T A G T C t A G T G C A a G C T A A C X T T T A T C A C A G G C T C C C T A G T G C A A G C T A A C X T T T T A T C A C A G G C T C G T C T A G t G C A A G C T A A X T T T A T C A C A G G C T C C T G C A A G C T A A C A X T T T A T C A C A G G C T C C C T A G T C t A G T G C A a G C T A A C X T A T C A C A G G C T C C A G C T A G T G C A A G C T A A C X T A T C A C A G G C T C C A G G T G C A A G C T A A C A G T X T A T C A C A G G C T C C A G G T C T A G t G C A A G C T A A C A G X T A T C A C A G G C T C C A G G T C T A G t G C A A G C T A A C A G X A T C A C A G G C T C C A G G G T G C A A G C T A A C A G T X T A T C A C A G G C T C C A G T A G T G C a A G C T A A C A G X T A T C A C A G G C T C C A G G T C T A G t G C A A G C T A A C A G X T A T C A C A G G C T C C A G G T G C A A G C T A A C A G T X A T C A C A G G C T C C A G G T A G T G C a A G C T A A C A G X T C A C A G g C T C C A G G A A G G G T A G T G C a A G C T A A C A G T T G X T C A C A G g C T C C A G G A A G G G G T G C A A G C T A A C A G T X T C A C A G g C T C C A G G A A G G G T G C A A G C T A A C A G T T X A T C A C A G G C T C C A G G G T G C A A G C T a A C A G T T X T C A C A G g C T C C A G G A A G G G G T G C A A G C T A A C A G T X C A C A G G C T C C A G G A A G G G T G C A A G C T A A C A G T T X T C A C A G g C T C C A G G A A G G G T A G T G C a A G C T A A C A G T T G X T C A C A G G C T C C A G G A G T G C A A G C T A A C A G T X T C A C A G G C T C C A G G A T G C A A G C T A A C A G T T X C A C A G G C T C C A G G A A G G G G T G C A A G C T a A C A G T T X T C A C A G G C T C C A G G A T G C A A G C T A A C A G T T X T C A C A G G C T C C A G G A G T G C A A G C T A A C A G T X C A C A G G C T C C A G G A A T G C A A G C T A A C A G T T X C A C A G G C T C C A G G A A G T G C A A G C T A A C A G T T G C X T C A C A G g C T C C A G G A A G G G G T G C A A G C T A A C A G T T G C X T C A C A G G C T C C A G G A T G C A A G C T A A C A G T T X A C A G G C T C C A G G A A G G G T T G C A A G C T A A C A G T T X A C A G G 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