UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
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Securities Exchange Act of 1934
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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
Item 7.01 Regulation FD Disclosure.
Sana Biotechnology, Inc. (the “Company”) intends to discuss an updated corporate presentation (the “Corporate Presentation”) at the 41st Annual J.P. Morgan Healthcare Conference on January 10, 2023. A copy of the Corporate Presentation is furnished as Exhibit 99.1 to this Current Report on Form 8-K (this “Current Report”) and is incorporated by reference herein.
By furnishing the information in this Item 7.01 of this Current Report, including Exhibit 99.1, the Company makes no admission as to the materiality of such information. The information contained herein is intended to be considered in the context of the Company’s filings with the U.S. Securities and Exchange Commission (the “SEC”) and other public announcements that the Company makes, by press release or otherwise, from time to time. The Company undertakes no duty or obligation to publicly update or revise the information contained in the Corporate Presentation, although it may do so from time to time as its management believes is appropriate. Any such updating may be made through the filing of other reports or documents with the SEC, through press releases or through other public disclosure.
In accordance with General Instruction B.2 of Form 8-K, the information furnished with this Current Report, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (“Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference into any other filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits
See the Exhibit Index below, which is incorporated by reference herein.
EXHIBIT INDEX
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99.1 |
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104 |
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Cover Page Interactive Data File (embedded within the Inline XBRL document) |
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
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Sana Biotechnology, Inc. |
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Date: January 10, 2023 |
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/s/ Bernard J. Cassidy |
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Bernard J. Cassidy |
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Executive Vice President and General Counsel |
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Corporate Presentation January 2023 Sana Biotechnology TM Exhibit 99.1
This presentation contains forward-looking statements about Sana Biotechnology, Inc. (the “Company,” “we,” “us,” or “our”) within the meaning of the federal securities laws. All statements other than statements of historical facts contained in this presentation, including, among others, statements regarding the Company’s strategy, expectations, cash runway and future financial condition, future operations, and prospects, are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “target,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. The Company has based these forward-looking statements largely on its current expectations, estimates, forecasts and projections about future events and financial trends that it believes may affect its financial condition, results of operations, business strategy and financial needs. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. These statements are subject to risks and uncertainties that could cause the actual results to vary materially, including, among others, the risks inherent in drug development such as those associated with the initiation, cost, timing, progress and results of the Company’s current and future research and development programs, preclinical studies, and clinical trials. For a detailed discussion of the risk factors that could affect the Company’s actual results, please refer to the risk factors identified in the Company’s SEC reports, including its Quarterly Report on Form 10-Q dated November 2, 2022. Except as required by law, the Company undertakes no obligation to update publicly any forward-looking statements for any reason. Cautionary Note Regarding Forward-Looking Statements Sana Biotechnology Tm © 2020-2023 Sana Biotechnology. All right Reserved 2
Sana’s ambition is to repair or replace any cell in the body. Technologies address fundamental barriers: Hypoimmune (HIP) technology: Overcoming immune rejection of allogeneic cells Fusogen technology: In vivo delivery of genomic modification reagents in a cell-specific manner Overcoming immune rejection of allogeneic cells has potential to change cell therapy: Allogeneic CAR T cells that perform clinically like autologous CAR T cells can transform treatment of hematological malignancies Key to unlocking the potential of stem cell-derived therapies such as pancreatic islet cells for the treatment of type 1 diabetes Two opportunities in 2023 for clear clinical proof of concept: SC291: Cell persistence and clinical efficacy HIP primary islets in patients with type 1 diabetes Results will provide insights in CAR T cell and stem-cell based platforms – ability to overcome allogeneic and autoimmune cell rejection Pipeline poised to deliver multiple clinical data readouts over next several years: Hypoimmune allogeneic CAR T cells: SC291 (CD19), SC262 (CD22), SC255 (BCMA), and beyond Regenerative medicine: SC451 (type 1 diabetes) and SC379 (CNS disorders) In vivo fusogen platform: SG295 Balance sheet allows potential for multiple data readouts Engineered Cells as Medicines Sana Biotechnology Sana Biotechnology TM ©2020-2023 Sana Biotechnology. All right Reserved 3
Pipeline poised to deliver multiple clinical data readouts over next several years Sana’s platforms, technology, and programs 1IST, investigator sponsored trial. Abbreviations: ALL, acute lymphoblastic leukemia; CLL, chronic lymphocytic leukemia; HD, Huntington’s disease; IND, investigational new drug; NHL, non-Hodgkin’s lymphoma; PMD, Pelizaeus-Merzbacher Disease; SCD, sickle cell disease; SPMS, Secondary Progressive Multiple Sclerosis. Sana Biotechnology TM ©2020-2023 Sana Biotechnology. All right Reserved 4
~75 years of organ and bone marrow transplants – immune rejection remains the largest problem Cell-based medicines face similar immune rejection challenges Significant immunosuppression is current standard Genome modification efforts to date have generally been incomplete Autologous therapies have limited scalability and are only available for a small number of cell types Red blood cell transfusions are the only example of successful, broadly available transplanted allogeneic cells Overcoming immune rejection of foreign cells has potential to unlock entire field of cellular medicine Overcoming allogeneic immune rejection has been key limitation in transplant and cellular medicine Biopsy of acute rejection of a pancreas transplant Drachenberg et Al. Am J. Transplant. 2008 Sana Biotechnology TM ©2020-2023 Sana Biotechnology. All right Reserved 5
Sana’s hypoimmune solution: Leverage insights from nature Abbreviations: MHC, major histocompatibility complex; RBC, red blood cell. Current clinical platform with multiple ongoing approaches in research phase. Leverage insights from nature to create hypoimmune cells Sana’s hypoimmune approach Red blood cells Overexpress CD47 Do not express MHC Class I and II Red blood cells Healthy donor cells Hypoimmune cells Disruption of MHC Class I & II expression Overexpression of CD47 1 2 3 Sana Biotechnology TM ©2020-2023 Sana Biotechnology. All right Reserved 6
Sana’s HIP modifications offer superior protection from innate cell killing Abbreviations: HLA, human leukocyte antigen; iPSC, induced pluripotent stem cells; KI, knock-in; KO, knock-out; NK, natural killer; PD-L1, Programmed death-ligand 1. No Killing Killing Killing Killing No Killing No Killing T cell Killing HLA-I/II KO — + HLA-CKI + HLA-EKI + HLA-GKI + PD-L1KI + CD47KI HLA-I/II KO — + HLA-CKI + HLA-EKI + HLA-GKI + PD-L1KI + CD47KI [BAR CHART] Sana Biotechnology TM ©2020-2023 Sana Biotechnology. All right Reserved 7
Sana’s HIP modifications offer superior protection from innate cell killing Abbreviations: HLA, human leukocyte antigen; iPSC, induced pluripotent stem cells; KI, knock-in; KO, knock-out; NK, natural killer; PD-L1, Programmed death-ligand 1. No Killing Killing Killing Killing No Killing No Killing T cell Killing Sana's HIP modifications offer superior protection from innate cell killing T-cell killing NK Cell Killing NK cell Allogeneic human iPSC without MHC (triggering innate immune response through "missing- self") Abbreviations: HLA, human leukocyte antigen; iPSC, induced pluripotent stem cells; KI, Knock-in; MHC, major histocompatibility complex; NK, natural Killer Sana Biotechnology TM @ 2020-2023 Sana Biotechnology. All right Reserved 9 Normalized Call Index HLA-I/II KO HLA-I/II KO
Hypoimmune cells survive in vivo when transplanted in NHP while unmodified iPSCs get rejected Abbreviations: NHP, non-human primate; Txp, transplant; WT, wild type. A B NHP unmodified iPSCs (WT) & NHP hypoimmune iPSCs (HIP) were transplanted into eight allogeneic NHP recipients [BAR CHART] Sana Biotechnology TM ©2020-2023 Sana Biotechnology. All right Reserved 9
Survival of allogeneic hypoimmune pancreatic islet cells for 10+ months without immunosuppression Study design: NHP primary islet cells isolated and HIP-edited Cells injected intramuscularly into a healthy, allogeneic NHP without immunosuppression WT HIP Survival HIP results (follow up shown: 10 months). Study ongoing.D0 12 wks 28 wks 40 wks WT results (no survival after 1 wk) D0 1wk n=1 HIP primary islet cells; n=1 WT primary islet cells.[BAR_CHART] Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All rights reserved. 10
Survival and immune evasion after transplant for different cell types in multiple NHP studies Abbreviations: RPE, retinal pigment epithelium. HIP primary islets HIP iPSC-derived RPE HIP iPSC-derived cardiomyocytes HIP iPSC Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All rights reserved. 11
Challenges Autologous CAR T cell scalability Many patients fail CAR T treatment Allogeneic CAR T cells immune rejection limits persistence and efficacy Opportunity Known targets Known efficacy and safety bar Sana’s HIP CAR T platform can address challenges and exploit opportunities Hematologic cancers continue to have a high unmet need 1Avezbakiyev et al. Blood. 2022 2Durie et al. The Oncologist. 2020 3Clarivate DRG NHL Market Forecast Nov 2021 4Scivida 2022 NHL Factbook Abbreviations: EU5, France, Germany, Italy, Spain, UK High mortality in lymphoma and myeloma in the US and EU5 ~250,000 annual incidence1,2 Over 100,000 deaths annually1,2 ~4,000 patients treated with CAR T in 20213 An estimated ~30 to 40% of patients treated with CAR T will experience durable complete responses4 Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All rights reserved. 12
Sana’s HIP platform can create a regenerative pipeline for allogeneic CAR T therapies 1Based on current scale, assuming 50% hold back for analytical and other testing, and variability in dose in Phase 1 study. Abbreviations: Cas12b, CRISPR associated protein 12b; GPRC5D, G protein–coupled receptor, class C, group 5, member D; PBMC, peripheral blood mononuclear cell. PBMCs from healthy donor 1 2 Select T cells T cell selection Expand and grow high-quality allogeneic CAR T cells at scale 5 Modify into HIP T cells 3 Insert CAR 4 Produces 100-600 doses per run1 Genes disrupted with Cas12b nuclease CD19 CD22 BCMA GPRC5D 1Based on current scale, assuming 50% hold back for analytical and other testing, and variability in dose in Phase 1 study Abbreviations: Cas12b, CRISPR associated protein 12b; GPRC5D, G protein–coupled receptor, class C, group 5, member D; PBMC, peripheral blood mononuclear cell. Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All rights reserved. 13
SC291 tumor control comparable at early timepoints to standard CD19 CAR T cells SC291 tumor control superior at later timepoints to standard CD19 CAR T cells SC291 controls tumors when animals are rechallenged with tumor HIP CD19 CAR T cells demonstrate persistence and continued efficacy in humanized mice model tumor cell re-injection Unmodified T Cells Unmodified CD19 CAR T Cells HIP CD19 CAR T Cells D0:Nalm6 D15 D27 D55 D75 D83 D87 tumor cell re-injection Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All rights reserved. 14
SC291: Sana’s CD19 HIP allogeneic CAR T IND filed First clinical data in 2023 Data show CAR T cell persistence correlates with long term complete response (CRs) rates1 Improved persistence can lead to best-in-class allogeneic CAR T platform Allogeneic HIP CAR T cell 1Porter et al. Science Translational Medicine. 2015 Allogeneic HIP CAR T cell MHC I MHC II TCRα + CD19 CAR CAR T Persistence Potential Efficacy Outcome ≤ 1 month 2 to 3 months 3 to 6 months ≥ 6 months Comparable to existing Allo CAR T Best-in-class Allo CAR T Comparable to Auto CAR T Better than Auto CAR T Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All rights reserved. 15
SC262: Targeting growing population of patients with inadequate response to CD19 therapy 1US, EU5, and Japan. 2Clarivate DRG NHL Market Forecast Nov 2021; 2027 Forecast is 2L+ LBCL patients. 3Di Blasi et al. Blood.2022; DESCAR-T registry. ~65% ~35% 100% ~12K ~12K Potential of ~7,800 CAR T failures annually by 20272; median survival of ~5 months post-CD19 CAR T therapy-failure3 Estimated ~12,000 B cell malignancy patients treated with CD19 CAR T by 20272 Estimated ~4,200 CAR T patients with durable complete responses4 Allogeneic HIP CAR T cell CD19 CAR T relapsed patients represent large and growing unmet need1 SC262 utilizes a clinically- validated CD22 CAR + CD47 MHC I MHC II TCRα + CD22 CAR Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All rights reserved. 16
2021 ASH Miklos/Stanford SC262 Goals: File IND this year; clinical data in 2024 N=24 SC262: Licensed CD22 CAR produced strong clinical data in CD19 failures when part of autologous CAR T N=16 N=21 >50% 6-month CR rate in CD19 CAR failure DLBCL patients High rate of CRs in CD19 failure ALL patients ~80% patients with prior CD19 therapy 2022 ASH Miklos/Stanford 2018 Nature Med Fry, et al. CR CR Expand our allo T platform to CD22 with Sana’s SC262 candidate Allogeneic HIP CAR T cell [BAR_CHART] [BAR_CHART] Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All right reserved. 17
SC255: Licensed BCMA CAR produced strong clinical data in myeloma when part of autologous CAR T 4 3 ORR: 98% 5 ORR: 95% ORR: 75% Total N=100 SC255 Goal: File IND as early as 2024 Prior CAR T n=12 No prior CAR T n=88 1 ~80% patients MRD negative at 12 months ~75% patients in CR/sCR with median follow-up ~1 year 2022 ASH Nanjing IASO Expand our allo T platform to BCMA with Sana’s SC255 candidate High response rate in multiple myeloma with 95% of patients MRD negative Allogeneic HIP CAR T cell Abbreviations: CR, complete response; ORR, objective response rate; PR, partial response; sCR, stringent complete response; VGPR, very good partial response. [BAR_CHART] Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All right reserved. 18
Goal is to build a best-in-class CAR T portfolio to treat patients with a range of cancers and beyond 1Avezbakiyev et al. Blood. 2022 2Durie et al. The Oncologist. 2020 Abbreviations: SLE, systemic lupus erythematosus. Unlocking the potential of our allogeneic CAR T franchise across multiple patient populations CD19 CD22 BCMA Autoimmune Unlocked by HIP validation Solid Tumors New Targets Hematologic Malignancies GPRC5D Known Future State Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All right reserved. Validated targets Validated CAR constructs >100,000 potential cancer patients worldwide1,2 HIP platform understood in preclinical models Potential for SLE and other autoimmune disorders Solid Tumors HIP platform understood in humans 19
Disease caused by autoimmune destruction of insulin-producing beta cells in the pancreas; results in inability to control blood glucose Type 1 diabetes is a large unmet need with 1.6M patients in the U.S. and 2.4M in Europe2 Long-term complications: end-organ damage, including heart attack, stroke, blindness, and kidney failure SC451 goal is euglycemia without exogenous insulin or immunosuppression Type 1 diabetes represents a large unmet need with a loss of ~15 years of life1 1Rawshani et al. Lancet. 2018 2Centers for Disease Control and Prevention, Diabetes Report, 2017-2018. Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All right reserved. 20
Sana’s solution: Hypoimmune islet cells for type 1 diabetes (SC451) PSCs can provide scale and product consistency HIP has potential to eliminate immunosuppression, protecting against both allogeneic and autoimmune rejection Islet cell transplantation has been shown to work in type 1 diabetes Islet cell transplants result in insulin independence in type 1 diabetics Phase 3 trial of primary islets showed 52% & 42% of patients become insulin independent at 1 & 2 years, respectively Utilization limited by need for lifelong immunosuppression Hering et al, Diabetes Care. 2016. N= 48 adults; demonstrated efficacy of islet transplant with 87.5%/71% achieving primary endpoint (HbA1c <7% and no serious hypoglycemia) at 1 and 2 years. [BAR_CHART] HIP islet cell Sana Biotechnology TM © 2020-2023 Sana Biotechnology. All right reserved. 21
D0 D3 D7 D5 D9 No glucose control Patient T cells eliminate islet cells due to autoimmunity Sana’s immunology, gene modification, & stem cell capabilities create proprietary type 1 diabetes model Abbreviations: T1DM, type 1 diabetes mellitus. Patient with T1DM PBMCs Autologous iPSC-derived islet cells Humanized T1DM mice Auto iPSCs HIP autologous iPSC-derived islet cells Unmodified patient stem cell-derived islet cells do not survive Type 1 diabetic PBMCs used to generate stem cell-derived islet cells and to humanize immune system in mice [BAR_CHART] [BAR_CHART] [BAR_CHART] Sana Biotechnology Tm© 2020-2023 Sana Biotechnology All rights reserved 22
HIP iPSC-derived pancreatic islet cells from T1DM patient evade autoimmune killing and control glucose Abbreviations: BLI, bioluminescence imaging. BLI: Islet Survival Glucose Control SC451 Goal: File IND and share clinical data in 2024 C-peptide [BAR_CHART] [BAR_CHART] [BAR_CHART] [BAR_CHART] Sana Biotechnology Tm© 2020-2023 Sana Biotechnology All rights reserved 23
Investigator sponsored trial Primary human HIP islet cells transplantation in type 1 diabetes patients Goal: Cell survival with no immunosuppression Goal: Data in 2023 Insight for SC451 Path to potential clinical validation of hypoimmune islet cells in T1DM patients in 2023 Cell survival & immune evasion C-peptide Glycemic control Key Measured Outcomes [BAR_CHART] [BAR_CHART] Sana Biotechnology Tm© 2020-2023 Sana Biotechnology All rights reserved 24
Fusosome technology: Development of cell-specific in vivo delivery platform Source: CDC website 3 2 1 Sana approach: Leverage insights from nature to deliver various payloads to specific cells Fusosome [BAR_CHART] [BAR_CHART] [BAR_CHART] Sana Biotechnology Tm© 2020-2023 Sana Biotechnology All rights reserved 25
Fusogen technology has potential to eliminate conditioning chemotherapy and ex vivo manufacturing Current ex vivo approaches have limitations Fusogen platform offers potential to overcome these limitations [BAR_CHART] [BAR_CHART] Sana Biotechnology Tm© 2020-2023 Sana Biotechnology All rights reserved 26
T cell fusosome delivers CAR construct directly to T cells in vivo CAR activity Expression/recognition Amplification Target cell killing Target cell killing Specificity CD4/CD8 T cell transduction Expression Transgene integration and CAR expression Function Targeted cell killing T cell Fusosome Cancer cell CAR T cell 2 Fusosome performance Transduction specificity Transduction efficiency 1 [BAR_CHART] Sana Biotechnology Tm© 2020-2023 Sana Biotechnology All rights reserved 27
IV administration of CD19 CAR delivered by fusosome can clear B cell tumors in humanized mice comparably to ex vivo CD19 CAR T CD19 CAR delivered by fusosome: in vivo CD19 CAR T: ex vivo Saline CD19 CAR T SG295 & Activated PBMC SG295 & Non-Activated PBMC Sana Biotechnology TM © 2020-2023 Sana Biotechnology All rights reserved 28
First clinical study incorporates SG295 made with V2.0 process Significant improvements in manufacturing process may lead to a better therapy Abbreviations: TU, transduction units. Potential for: Better efficacy Greater safety Higher yields and lower cost of goods SG295 made with V2.0 process results in >50X potency improvement SG295 made with V2.0 process transduces more T cells at the same dose SC295 Goals: File IND in 2023; clinical data in 2024 [BAR CHART] [BAR CHART] Sana Biotechnology TM © 2020-2023 Sana Biotechnology All rights reserved 29
Tumor control achieved with fusosomes targeting other cell types and alternate tumor antigens CD4 T cell targeting fusosomes delivering CD19 CAR achieve tumor control in mice CD8 T cell targeting fusosomes delivering CD22 CAR achieve tumor control in mice D-4 D3 D6 D10 D24 D28 D14 D17 D21 Control Low 1e6 IU High 5e6 IU CD8-CD22 CAR Fusogen Dose CD4-CD19 CAR Fusogen Dose D-3 D1 D10 D15 D17 D21 D7 High 1e7 IU Control Low 2.5e6 IU Sana Biotechnology TM © 2020-2023 Sana Biotechnology All rights reserved 30
Sana aspiration: Engineered cells as medicines 1Does not incorporate hypoimmune genomic modifications Allogeneic CAR T Franchise Oncology: SC291, SC262, SC255 Autoimmune Stem Cell-Derived Type 1 Diabetes: SC451 CNS: SC3791 In vivo CAR T: SG295 Cell-specific in vivo HSC editing: SG418 Cell-specific delivery of genomic modification material Engineered cells into new therapeutic areas Ex Vivo – Hypoimmune In Vivo – Fusogen Allogeneic HIP CAR T cell HIP islet cell 2023 +CD47 +CAR +Modified G protein F protein Sana Biotechnology TM © 2020-2023 Sana Biotechnology All rights reserved 31
Thank You Sana Biotechnology www.sana.com Sana Biotechnology TM