Sana Biotechnology, Inc. · NASDAQ:SANA
Sana Biotechnology — Investor Presentation (January 2025)
35 slides from Sana Biotechnology's investor presentation, Jan 13, 2025
Slide 1: Corporate Presentation January 2025
Exhibit 99.1 Corporate Presentation January 2025
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: Changing the Possible for Patients
Sana Biotechnology Changing the Possible for Patients Recent data confirm Sana's hypoimmune platform (HIP) overcomes allogeneic rejection in people Ÿ Transplanted HIP-modified pancreatic islets overcome allogeneic and autoimmune rejection in type 1 diabetes Ÿ We believe T1D result generalizable acro
Slide 4: Overcoming allogeneic immune rejection has been key limitation in transplant and cellular medicine
Overcoming allogeneic immune rejection has been key limitation in transplant and cellular medicine Allogeneic cell rejection Sana’s hypoimmune approach Ÿ ~75 years of transplants – immune rejection remains the largest problem Blocks innate Blocks adaptive immune system immune system Ÿ Lifelong immun
Slide 5: Type 1 diabetes (T1D) remains a significant unmet need
Type 1 diabetes (T1D) remains a significant unmet need Improvement in glycemic control reduces microvascular complications Ÿ T1D is an autoimmune destruction of insulin-producing pancreatic beta 1 but increases the risk of hypoglycemia cells and leads to lifelong insulin therapy requirement Ÿ 8.4M p
Slide 6: Advancing toward a cure for broad T1D population
Advancing toward a cure for broad T1D population T1D is a disease of missing pancreatic beta cells 1 2 Primary islet transplants provide Stem-cell derived islets provide The Goal: long-term glucose control, but: a scalable supply, but: A single treatment with • Supply is an issue long-term normal bl
Slide 7: Potential clinical validation of hypoimmune islet cells in T1DM patients
Potential clinical validation of hypoimmune islet cells in T1DM patients IST Design Ÿ Trial at Uppsala University Hospital 1 2 3 Ÿ Primary human HIP-modified islet cells transplanted in type 1 diabetes patients Ÿ Intramuscular administration in forearm Ÿ No immunosuppression Transplant into HIP gene
Slide 8: All primary and secondary endpoints met
All primary and secondary endpoints met Primary and Secondary Endpoints: Data Summary Endpoints D7 D14 D21 D28 Safety (no AE/SAE related to drug) Cell survival/function (C-peptide) Not performed Graft visibility (MRI) (as per protocol) Adaptive immune evasion Innate immune evasion © 2020-2025 Sana B
Slide 9: Stable C-peptide demonstrates survival and function of cells after HIP islet cell transplantation
Stable C-peptide demonstrates survival and function of cells after HIP islet cell transplantation Basal C-peptide in peripheral blood 15 11.4 11.3 12 10.2 9.7 9 6 3 0 Baseline D7 D14 D21 D28 Summary: No detectable C-peptide before transplantation; present and stable C-peptide observed after transpla
Slide 10: Increased C-peptide levels with a mixed meal tolerance test highlight survival and function
Increased C-peptide levels with a mixed meal tolerance test (MMTT) highlight survival and function Before transplantation Day 28 20 17.2 17.0 16.3 15 13.4 12.1 10 10.5 5 0 0 30 60 90 120 Time (min) Summary: Before transplantation, C-peptide is below detection limit during MMTT. 28 days after UP421 t
Slide 11: Day 28 MRI: Further evidence of graft survival
Day 28 MRI: Further evidence of graft survival MR T2-STIR-weighted trans images showing signal in musculus brachioradialis after injection of UP421 Arrows indicate the location of some examples of injected cells D28 MRI showing several punctual signals at the site of graft injection, no inflammation
Slide 12: The drug product's mixed cell population allows detailed immune analysis
The drug product’s mixed cell population allows detailed immune analysis Donor islet cells contain wild type & double knockout cells as well as HIP cells Donor Immune analysis using patient’s (recipient) immune cells after transplantation + CD47 HLA I HIP islet cells Ÿ T cells HLA II Ÿ Donor-specifi
Slide 13: Unmodified islet cells: Do not evade T cell or B cell immune response
Unmodified islet cells: Do not evade T cell or B cell immune response WT islet cells WT islet cells WT islet cells Patient’s T cells are activated and kill WT Patient’s B cells produce donor-specific CD47 HLA I islet cells with peak at 7 days after transplant antibodies (switch from IgM to IgG at D1
Slide 14: dKO islet cells: Evade B and T cell responses but are killed by NK cells
dKO islet cells: Evade B and T cell responses but are killed by NK cells dKO islet cells dKO islet cells dKO islet cells dKO islet cells No killing of HLA deficient cells No binding of donor-specific Patient’s NK cells are killing CD47 HLA I by patient’s T cells antibodies to HLA deficient cells HLA
Slide 15: HIP islet cells: Evade T cell, B cell, and NK cell immune responses
HIP islet cells: Evade T cell, B cell, and NK cell immune responses HIP islet cells HIP islet cells HIP islet cells HIP islet cells No killing of HIP islet cells No binding of donor-specific No killing of HIP islet cells + CD47 HLA I by patient’s T cells antibodies to HIP islet cells by patient’s NK
Slide 16: HIP islet cells overcome patient's allogeneic and autoimmune barrier
HIP islet cells overcome patient's allogeneic and autoimmune barrier still image before movie D7 sample: PBMC (containing all immune cell populations) Donor islet cells with NK cells plus serum (containing antibodies and complement) killing assay editing profile in column title Target: WT islet cell
Slide 17: still image after movie
still image after movie © 2020-2025 Sana Biotechnology. All rights reserved. 17
Slide 18: SC451: A drug for the broad T1D population
SC451: A drug for the broad T1D population Make hypoimmune islet Manufacture Deliver as a 1 2 3 cells from stem cells at scale single therapy Graft site + CD47 MHC I MHC II SC451 program – HIP stem cell-derived islet cell therapy delivered with no immunosuppression © 2020-2025 Sana Biotechnology. Al
Slide 19: Four major challenges to realizing the vision of SC451
Four major challenges to realizing the vision of SC451 1 2 3 4 Overcoming immune Differentiating PSCs into Generating a gene- Manufacturing enough rejection without islet cells at a purity, modified MCB from a GMP- product to treat the immunosuppression potency, and yield to compliant PSC line that
Slide 20: HIP-modified PSC differentiated islet cells persist & control blood glucose in mice for >15 months
HIP-modified PSC differentiated islet cells transplanted into muscle persist & control blood glucose in mice for >15 months PSCs differentiated into HIP-modified PSC HIP-modified PSC islets persist islets at high purity islets produce human and control blood glucose (non-fasted blood glucose*) c-pep
Slide 21: B-cells drive autoimmune disease in millions of patients
B-cells drive autoimmune disease in millions of patients >75 different types of autoimmune disorders with underlying B cell pathology and high unmet need • SLE • NMDAR encephalitis • Vasculitis (granulomatosis • Thrombocytopenic purpura with polyangiitis & microscopic • Amyloidosis >5M polyangiitis)
Slide 22: Autologous CAR Ts have shown curative potential; allogeneic cells have inherent advantages
Autologous CAR Ts have shown curative potential; allogeneic cells have inherent advantages Autologous CAR T CD19 Autologous CAR T treatment has Challenges 1 delivered long term, drug-free remissions Ÿ Difficult to scale SLEDAI-2K Urinary Protein Excretion Ÿ Prescription-to-infusion time over 4 weeks
Slide 23: Sana's T cell manufacturing process provides high yields of successfully edited cells
Sana’s T cell manufacturing process provides high yields of successfully edited cells SC291: Highly efficient editing of cells Product ready when the patient needs it 1 100s of autoimmune patient batches/manufacturing run + CD47 + CAR MHC I ~85% full knock-out of MHC class I and II MHC II >99.5% TCR
Slide 24: SC291 safely administered with deep, dose-dependent B-cell depletion in oncology
SC291 can be safely administered and results in deep, dose-dependent B-cell depletion in oncology Deep B-cell depletion seen in NHL patients ARDENT Safety Data (N=16) 5000 Dose Level 3 Dose Level 4 300-006 (M5) 300-012 1000 300-007 (M3) Ÿ No cases of Grade 2 or higher CRS 103-002 (D28) 500 300-008 (
Slide 25: SC291: GLEAM Phase 1 trial goals
SC291: GLEAM Phase 1 trial goals are to understand safety, dose, and early efficacy Allogeneic HIP CAR T cell + CD47 Ÿ Key features of Phase 1 trial (GLEAM) Ÿ Patients with refractory systemic lupus erythematosus and ANCA-associated MHC I vasculitis + CD19 CAR Ÿ Dose escalation study MHC II Ÿ Potent
Slide 26: Sana is pursuing in vivo engineering of CAR T cells using a fusosome vector system
Sana is pursuing in vivo engineering of CAR T cells using a fusosome vector system Delivery Expression Function Transduction of CD8 T cells Transgene integration and CAR expression Targeted cell killing B cell T cell Target cell killing CAR CAR Transgene T cell amplification for CAR expression CAR T
Slide 27: Fusosome technology: Cell-specific in vivo delivery
Fusosome technology: Cell-specific in vivo delivery Sana approach: Leverage insights from nature to deliver various payloads to specific cells without lymphodepletion G F Fusosome © 2020-2025 Sana Biotechnology. All rights reserved. 27
Slide 28: Potent and cell-specific in vivo delivery demonstrated with SG299 in GLP tox study
Potent and cell-specific in vivo delivery demonstrated with SG299 in GLP tox study 4 NHPs at each dose received single SG299 injection Potent transduction of No off-target transduction detected in + circulating CD8 T cells hepatocytes or gonadal cells Low dose High dose 0 -1 10 10 ON-TARGET Low dose
Slide 29: Surrogate SG299 leads to T cell transduction, CAR expansion, and B-cell depletion
Surrogate SG299 with additional component leads to T cell transduction, CAR expansion, and B-cell depletion Surrogate SG299 transduces T cells & expresses a CAR that recognizes NHP B cells + + CD8 CAR cells expand in circulation Deep B-cell depletion achieved in peripheral blood Control NHP 1 NHP 2
Slide 30: Study shows B cell clearance in lymph nodes without lymphodepletion
Study shows B cell clearance in lymph nodes without lymphodepletion Anti CD20 staining of day 28 lymph node biopsy Control NHP 1 NHP 2 Brown indicates CD20 IHC staining; black reflects tattoo ink. © 2020-2025 Sana Biotechnology. All rights reserved. 30
Slide 31: SC262: Targets growing population of patients with inadequate response to CD19 therapy
SC262: Targets growing population of patients with inadequate response to CD19 therapy CD19 CAR T relapsed patients represent CD22 CAR T is a promising approach to 1 5 large and growing unmet need treat CD19 therapy failure Ÿ Autologous CD22 CAR T results in >50% CR rate in Estimated ~12,000 CD19 CA
Slide 32: SC262: VIVID Phase 1 trial goals
SC262: VIVID Phase 1 trial goals are to understand safety, dose, and early efficacy Allogeneic HIP CAR T cell + CD47 Ÿ Key features of Phase 1 trial (VIVID) Ÿ CD19 CAR T exposed patients with relapsed and/or refractory NHL MHC I + CD22 CAR Ÿ Starting dose of 90 million CAR T cells MHC II Ÿ Expect to
Slide 33: We anticipate meaningful clinical data in multiple diseases in 2025 and beyond
We anticipate meaningful clinical data in multiple diseases in 2025 and beyond Ÿ HIP platform shows ability to overcome allogeneic and autoimmune rejection in people across multiple cell types Ÿ Type 1 diabetes – all components for a curative therapy are now in place: Ÿ Patients have remained off in
Slide 34: Sana pipeline positioned to deliver meaningful clinical data
Sana pipeline positioned to deliver meaningful clinical data PRODUCT PRECLINICAL SANA’S CANDIDATE MECHANISM INDICATIONS IND-ENABLING PHASE 1 PHASE 2/3 RIGHTS Type 1 Diabetes 1 UP421 HIP primary islet cells T1D WW SC451 Stem-cell derived pancreatic islet cells T1D WW B-cell Mediated Autoimmune Diseas
Slide 35: Thank You
Thank You Biotechnoloay*
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