Sana Biotechnology, Inc. · NASDAQ:SANA
Sana Biotechnology — Investor Presentation (September 2021)
32 slides from Sana Biotechnology's investor presentation, Sep 13, 2021
Slide 1: Corporate Presentation September 2021
Corporate Presentation September 2021 Exhibit 99.1
Slide 2: Cautionary Note Regarding Forward-Looking Statements
Cautionary Note Regarding Forward-Looking Statements 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 thi
Slide 3: Sana Biotechnology: Engineered cells as medicines
Sana Biotechnology Engineered cells as medicines We believe the ability to modify the genome and use engineered cells as medicines will be one of the most (if not the most) important advances in healthcare over the next several decades Three aspirations drive us in our pursuit to deliver on the prom
Slide 4: Repair and control genes or replace any cell in the body
Deliver any payload (DNA, RNA, protein, organelle, integrating vs non-integrating)… To any cell (unlimited volume of distribution) in a… Specific (e.g., just T cell),… And repeatable way (limit immunogenicity) Manufacture any cell at scale… That engrafts (the right cell in the right environment)… Fu
Slide 5: Assembling an experienced team across capabilities
Assembling an experienced team across capabilities Delivery of Genetic Material Modification of Genome Immunology Biology Manufacturing Cell Biology Stem Cell Biology Disease Biology Richard Mulligan, PhD Jagesh Shah, PhD Ed Rebar, PhD Christina Chaivorapol, PhD Stacey Ma, PhD Craig Lichtenstein Mik
Slide 6: Platform technologies, programs and potential IND submissions
PLATFORM TECHNOLOGY PROGRAMS (CELL TYPES) THERAPEUTIC AREA PRODUCT CANDIDATE POTENTIAL INDICATIONS POTENTIAL IND SUBMISSION in vivo cell engineering Fusogen T cells Oncology SG295 (CD8/CD19) NHL/ALL/CLL As early as 2022 SG239 (CD8/BCMA) Multiple myeloma As early as 2022 SG242 (CD4/CD19) NHL/ALL/CLL
Slide 7: Fusogen technology and fusosome structure
VHH G protein dictates binding to a target receptor Sana’s fusosome technology makes use of a viral fusogen to enable the targeting of specific cells and the delivery of different therapeutic payloads F G Fusosome F protein drives fusion Fusogen Technology
Slide 8: Development of cell-specific in vivo delivery platform
Development of cell-specific in vivo delivery platform Fusogen Technology Enveloped viruses incorporate viral and cellular proteins (fusogens) expressed on the infected cell membrane upon release from infected cells Fusogens on surface of the resulting virus particles (fusosomes) mediate virus entry
Slide 9: In vivo cell engineering across diverse cell types
in vivo cell engineering – creating targeted medicines across a diverse set of cell types T cells Hepatocytes Hematopoietic stem cells in vivo cell engineering strategy focused on developing therapies with transformative fusogen platform delivery based on cell specificity and payload diversity
Slide 10: Blood cancers remain high unmet need; CAR T limitations
Blood cancers remain high unmet need; despite success, current CAR T solutions have limitations Current ex vivo approaches have limitations T cell fusosome carrying CAR construct infused into patient; the patient is the bioreactor that creates CAR T Fusogen platform offers potential to overcome thes
Slide 11: Using a T cell-targeted fusosome to make CAR T cells in vivo
CAR Activity Expression/Recognition Amplification Target cell killing Target cell killing T cell amplification Using a T cell-targeted fusosome to make CAR T cells in vivo Specificity CD4/CD8 T cell transduction Expression Transgene integration and CAR expression Function Targeted cell killing T cel
Slide 12: Targeting of different T cell types by viral fusosomes
Targeting of different T cell types by viral fusosomes Sana has generated fusosomes that specifically target and transduce CD8, CD4 and CD3 T cells CD8-targeted fusosome in vitro primary T cells CD8-targeted CD4-targeted fusosome in vitro primary T cells CD4-targeted CD3-targeted CD3-targeted fusoso
Slide 13: IV CD19 CAR by fusogen clears B cell tumors in humanized mice
IV administration of CD19 CAR delivered by fusogen can clear B cell tumors in humanized mice comparably to ex vivo CD19 CAR T Saline SG295 & Activated PBMC SG295 & Non-Activated PBMC CD19 CAR delivered by fusogen: in vivo CD19 CAR: ex vivo Saline CD19 CAR T D3 D7 D15 D19 D27 D7 D10 D17 D24 D31 T Cel
Slide 14: CD8 fusogen delivering CD20 CAR causes B cell depletion in NHPs
B cell levels (CD20+ cells) CD8 fusogen delivering a CD20 CAR causes B cell depletion in NHPs Dosing well tolerated in all animals treated with CD20 CAR T delivered by CD8 fusogen, no infusion-related toxicity or evidence of CAR-associated toxicity Substantial B cell depletion observed in 4/6 treate
Slide 15: Potential first-in-class fusogen T cell programs
Potential first-in-class fusogen T cell programs – potential to target large markets with a single IV administration Next Steps SG295 and SG239 Future Development IND-enabling studies and scale GMP manufacturing Finalize development plan – expect initial indications in NHL for CD19 and multiple myel
Slide 16: Protecting cells from immune destruction
Protecting cells from immune destruction is key to unlocking potential of ex vivo cell engineering Fetomaternal tolerance during pregnancy “Allogeneic” fetus: Half of fetal proteins are from the father, not the mother. However, the fetus is not rejected by the mother. Sana approach: creating hypoimm
Slide 17: Broad ex vivo cell engineering strategy
Sana is pursuing a broad ex vivo cell engineering strategy T cells Pancreatic islets Glial progenitor cells Cardiomyocytes Differentiate pluripotent stem cells with hypoimmune edits Programs that benefit from, but do not require hypoimmune Transforming ex vivo cell engineering through development of
Slide 18: Hypoimmune cells evade adaptive and innate immune rejection in mouse
Hypoimmune cells evade rejection from the adaptive and innate immune system in a mouse T Cell Activation (ELISPOT) IgM Binding (FACS) No systemic T cell activation with HIP cell transplantation No binding of donor specific antibodies against HIP cells NK Cell Killing No NK cell killing with HIP Wild
Slide 19: Hypoimmune cells in NHP: no systemic adaptive immune activation
Unmodified iPSC Hypoimmune iPSC T Cell Activation (ELISPOT) IgM Production (ELISA) No systemic T cell activation by hypoimmune cells Implantation of hypoimmune cells does not cause activation of antibody production Transplantation of NHP iPSCs into allogeneic NHPs: immune evasion is achieved even af
Slide 20: Hypoimmune cells do not activate missing self response
Killing by macrophages Killing by NK cells Hypoimmune cells do not activate the “missing self” response from the macrophages Anti-CD47 safety switch Hypoimmune cells do not activate the “missing self” response from the NK cells Anti-CD47 safety switch Transplantation of NHP iPSCs into allogeneic NHP
Slide 21: Hypoimmune cells survive in sensitized NHPs without immunosuppression
HIP WT D0 (cross over) 4 wks (after crossover transplant) Hypoimmune iPSCs 8 wks (after crossover transplant) 3 wks (after crossover transplant) NHP 4 Summary Hypoimmune Unmodified cells were rejected within 3 weeks Hypoimmune cells survive in a crossover experiment in NHPs with pre-existing immunit
Slide 22: Hypoimmune cells survive in vivo in NHP while unmodified iPSCs are rejected
Hypoimmune cells survive in vivo in NHP while unmodified iPSCs get rejected D0 (cross over) Unmodified iPSCs 2wks (after crossover transplant) 16wks (after transplant) D0 (day of transplant) 2 wks (after transplant) Hypoimmune iPSCs 5 wks (after transplant) 10 wks (after transplant) moved NHP 5 Summ
Slide 23: Hypoimmune edits do not affect differentiation capacity
Hypoimmune edits (HLA-I knockout, HLA-II knockout, CD47tg) do not affect differentiation capacity nor intrinsic cell function. Unmodified iPSC Deuse T, Hu X, …, Schrepfer S. Nat Biotechnology. 2019; 37:252-258 Hypoimmune iPSC Cardiac cell differentiation Hypoimmune Human hypoimmune cells differentia
Slide 24: CD19 targeted allogeneic T cell
2 3 CD19 targeted allogeneic T cell Donor or iPSC T cells Cell engineering Hypo Allo T IMMUNE CHALLENGES CURRENT ALLO T SANA’S HYPOIMMUNE ALLO T GvHD HvGD: Adaptive Immune System HvGD: Innate Immune System We believe we are better positioned to overcome immune challenges versus existing allo T thera
Slide 25: HIP CAR T cells clear tumor in vivo
Day 0 Day 3 start of CAR dosing Day 7 Day 15 Untreated Day 23 Day 27 HIP CAR T cells Unmodified CAR T cells CD19 HIP CAR T cells clear tumor in vivo Hypo Allo T
Slide 26: HIP CAR T cells do not incite an antibody response
HIP CAR T cells do not incite an antibody response T cells are not activated by HIP CAR T cells HIP CAR T cells evade innate cell “missing self” response CD19 HIP CAR T cells do not activate adaptive or innate immune responses HIP CAR T Unmodified CAR T HIP CAR T Unmodified CAR T IgM Binding (FACS)
Slide 27: Allogeneic CAR T cells: potential best-in-class platform
Allogeneic CAR T cells: potential best-in-class CAR T platform for off-the-shelf therapies Next Steps for SC291 Future Development Develop GMP gene editing and manufacturing processes Finalize development plan – expect initial indication in NHL SC291 for other B cell malignancies SC255 for multiple
Slide 28: Type 1 diabetes represents a large unmet need
Type 1 diabetes represents a large unmet need with a loss of approximately 15 years of life Autoimmune disease where destruction of insulin-producing beta cells results in inability to control glucose 1.6 million patients with type 1 diabetes in the US and 2.4 million in Europe; 51k new patients/yea
Slide 29: SC451: combining HIP edits with leading beta cell protocol
SC451: combining HIP edits with leading beta cells protocol offers transformative potential for type 1 diabetes patients Superior insulin secretion and faster kinetics in vitro Robust rescue of type 1 diabetes mouse model Human Islet WashU (Sana) Tech Pagliuca Tech Beta Cell Ex Vivo
Slide 30: Beta cells: potential curative treatment for diabetes
Beta cells: potential to transform global diabetes pandemic with curative treatment Next Steps for SC451 GMP hypoimmune iPSC cell line Develop scalable GMP manufacturing process IND-enabling studies
Slide 31: Sana aspiration: engineered cells as medicines
Sana aspiration: engineered cells as medicines Almost all diseases result from damage to or dysfunction in a cell The challenge: Sana: engineered cells to treat a broad set of diseases Fusosome for CD19 CAR T in vivo Fusosome for BMCA CAR T in vivo Hypoimmune allo CD19 CAR T Hypoimmune allo BCMA CAR
Slide 32: Thank You
Thank You Biotechnology
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