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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 histor

Key Takeaway: Sana Biotechnology, Inc. presented forward-looking statements regarding its technology and business strategy, highlighting two significant opportunities for clinical proof of concept in 2023. The company aims to overcome immune rejection of allogeneic cells through its hypoimmune technology, potentially transforming cell therapy treatments. Sana's pipeline includes developments for hematological malignancies and regenerative medicine, with various clinical data readouts expected in the coming years. However, the company cautions that its projections are subject to significant risks and uncertainties inherent in drug development.

Market Sentiment Analysis

POSITIVE FACTORS

  • Sana Biotechnology's technologies aim to overcome significant barriers in cell therapy.
  • The company has clear clinical proof of concept opportunities in 2023.
  • Their innovative platforms for hypoimmune allogeneic CAR T therapies may transform treatment for hematological malignancies.
  • Sana's pipeline presents multiple upcoming clinical data readouts, enhancing their growth potential.

Full Press Release Details

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
Autologous CAR T cell scalability
Many patients fail CAR T treatment
Allogeneic CAR T cells immune rejection limits persistence and efficacy
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
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.
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: Sana's CD19 HIP allogeneic CAR T
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
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=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
Investigator sponsored trial
Primary human HIP islet cells transplantation in type 1 diabetes patients
Goal: Cell survival with no immunosuppression
Path to potential clinical validation of hypoimmune islet cells in T1DM patients in 2023 Cell survival & immune evasion
T cell fusosome delivers CAR construct directly to T cells in vivo CAR activity
Expression/recognition
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
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:
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
5e6 IU CD8-CD22 CAR Fusogen Dose CD4-CD19 CAR
Fusogen Dose D-3 D1 D10 D15 D17 D21 D7 High
Sana aspiration: Engineered cells as medicines 1Does not incorporate hypoimmune genomic modifications Allogeneic CAR T Franchise
Oncology: SC291, SC262, SC255
Type 1 Diabetes: SC451
CNS: SC3791 In vivo CAR T: SG295
Cell-specific in vivo HSC editing: SG418
Thank You Sana Biotechnology www.sana.com Sana Biotechnology TM

Frequently Asked Questions

What is the focus of Sana Biotechnology's forward-looking statements?

Sana Biotechnology's forward-looking statements concern its strategy, expectations, and future operations.

What technologies does Sana Biotechnology's hypoimmune platform use?

Sana's hypoimmune platform employs HIP technology to overcome immune rejection and fusogen technology for targeted gene delivery.

What are Sana's key clinical proof of concept opportunities for 2023?

Key opportunities include SC291's cell persistence and efficacy, and HIP primary islets for type 1 diabetes patients.

How does Sana address immune rejection in cell therapy?

Sana's approach modifies cells to mitigate immune rejection, allowing for broader application in cellular medicine.

What ongoing programs does Sana have in its pipeline?

Sana's pipeline includes hypoimmune CAR T cells and initiatives in regenerative medicine targeting various diseases.

Last updated: Jan 10, 2023