Immuno-Oncology Program
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1 Immuno-Oncology Program 2018
2 Forward Looking Statements 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, financial guidance for full year 2017 as it relates to revenues, operating expenses, and year end cash balances; the design of clinical trials and expected timing for release of data; the anticipated clinical development milestones and other potential value drivers in the future; the expected benefits of the collaboration with Pfizer, the expanded capability of Sangamo s technologies; the benefits of rebranding and reorganization, the research and development of novel gene-based therapies and the application Sangamo s ZFP technology platform to specific human diseases; corporate partnerships; and the potential of Sangamo s genome editing technology to treat genetic diseases. 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 and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of Sangamo s therapeutics, the ability to establish strategic partnerships and our ability to control expenses and achieve our milestones that generate revenues under our agreements. 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 and business environments. These risks and uncertainties are described more fully in Sangamo s Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q 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 duty to update such information except as required under applicable law. 2
3 We are committed to translating ground-breaking science into genomic therapies that transform patients lives 3
4 Sangamo is investing across four technological platforms for genomic medicines Gene Therapy Genome Editing Cell Therapy Gene Regulation 4
5 Clearly defined scope of the Kite-Gilead deal Strategic Collaboration in Oncology T cells and NK cells Ex Vivo genome editing (ZFNs and AAV) Cell Therapy Oncology CARs, TCRs, and NKRs directed to tumor antigens Autologous and allogeneic products Solid and liquid tumor types 5
6 Kite-Gilead deal economics $150M $3.01B Upfront payment $300M in potential per product milestone payments, up to 10 times Cell Therapy Oncology Tiered royalties on net sales 6
7 Product portfolio diversified across therapeutic area and technology Therapeutic Area Research Preclinical Phase 1/2 Phase 3 Collaborator Inherited Metabolic Diseases MPS I (SB-318) MPS II (SB-913) Fabry Disease (ST-920) Hematology Hemophilia A (SB-525) Hemophilia B (SB-FIX) Beta-thalassemia (ST-400) Sickle Cell Disease (BIVV-003) CNS Diseases Tauopathies ALS/FTLD - C9ORF72 Huntington s Disease Oncology Autologous and Allogeneic CAR/TCR/NKR Immunology Undisclosed Autoimmune Disease Targets Investigator Sponsored Clinical Research HIV (T cell and Stem Cell) Gene Therapy Genome Editing Cell Therapy Gene Regulation 7
8 ZFNs: The platform of choice for therapeutic genome editing T A C C C A A C G C G A A T T A T G A T G G G T T G C G C T T A A T A C ZFN T G C G C T T A A C G C A T G G G T A C G C G A A T T G C G T A C C C A ZFPs Precision Target any desired nucleotide in the genome ZFPs ZFN ZFNs Efficiency Level of modification at the desired target nucleotide Specificity Edit the targeted nucleotide without editing elsewhere in the genome 8
9 T-cell Receptor Expression We can reliably hit >90% gene editing efficiency in T-cells 93% Double KO 91% Targeted Insertion (TI) HLA Expression Transgene Expression Precise KO at TRAC and B2M loci with no detectable off-target activity Precise TI at TRAC locus with no detectable off-target activity 9
10 Off-the-shelf T cell therapy: eliminating TCR and/or HLA on CAR + T cells Healthy donor T cells Patient 10
11 Manufacturing allogeneic T-cell therapies with ZFNs cgmp Manufacturing Facility HLA knock-out TCR knock-out Targeted insertion of CAR gene Apheresis (Healthy Donor) Isolate T-cells from a healthy donor s blood T-cell Manufacturing Transient, non-viral delivery of ZFN mrna to manufacture universal CAR-T cells via singlestep, multiplexed gene editing Harvest & Storage Universal CAR-T cells are harvested, cryo-preserved and stored in a secure cell bank Infusion (Patients) Universal, off-the-shelf CAR-T cells are infused into new patients, on-demand 11
12 Allogeneic cell therapy production: high efficiency is critical for single-step multiplexed genome editing 1 KO + 1 KO + 1 TI Compounded Efficiency Very High 99% x 99% x 99% = 97% High 90% x 90% x 90% = 73% Medium 70% x 70% x 70% = 34% Low 50% x 50% x 50% = 13% 12
13 Cell therapy platform: simultaneous multiplex editing efficiencies with 3x ZFN KO + 1x targeted integration % EDITING POTENTIAL APPLICATION: Universal T cells with checkpoint gene knock-out % 9 6 % 9 3 % 9 1 % SINGLE STEP EDITING ZFN Knock-out TCR (TRAC) HLA-class I (β2m) CISH (checkpoint gene) Targeted Insertion GFP (into TRAC) T C R - 2 M - C I S H - G F P + 76% of cells have all 4 edits KO: TCR, β2m and CISH; TI: GFP (TRAC) 13
14 Harnessing the precision, efficiency and specificity of ZFNs for autologous and allogeneic cell therapy products for oncology Autologous / Allogeneic Cell Therapy Strategy Targeted integration of CAR/TCR gene High editing efficiency (>90%) significantly reduces manufacturing cost >90% endogenous TCR knock-out (KO) eliminates GvHD >90% HLA KO eliminates host immune response to modified cells Precision of ZFNs better safety profile expected vs comparative therapies in development Strategic Advantages 90% editing efficiency (~2x other gene editing technologies) Consistent transduction efficiency and low risk of insertional mutagenesis vs retrovirus-based cell modification Highly efficient, targeted integration at TRAC locus for better regulation of CAR-T construct under endogenous promoter Precise HLA/TCR gene KO for allogeneic cell therapy development 14
15 Thank you.
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