Sangamo Therapeutics, Inc. · NASDAQ:SGMO
Sangamo Therapeutics — Q4 2025 Earnings Presentation
49 slides from Sangamo Therapeutics's earnings presentation, Mar 30, 2026
Slide 1: Delivering the Future of Genomic Medicines
Delivering the Future of Genomic Medicines March 30, 2026
Slide 2: Forward-Looking Statements and Legal Disclaimers
Forward-Looking Statements and Legal Disclaimers This presentation, and accompanying oral commentary, contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to: the therapeutic and commercial potential
Slide 3: Sangamo Value Proposition
Sangamo Value • Differentiated genomic medicine platform combines core capabilities Proposition required to unlock treatments for devastating neurological diseases • Genome-targeting epigenetic regulation cargo • Capsid delivery engine for central nervous system administration • Focused neurology pi
Slide 4: Why neurology genomic medicines?
Why neurology genomic medicines? • Widespread, debilitating diseases, largely unserved by current approaches • Many neurology indications are single-gene or gene- associated • Genomic medicines are well suited to neurology: • Targeting diseases at the DNA level reduces therapeutic complexity • Gene
Slide 5: Sangamo pairs epigenetic regulation and capsid delivery capabilities
Sangamo pairs the epigenetic regulation and capsid delivery capabilities needed to create neurology genomic medicines Genome-Targeting Cargo Capsid Delivery Engine No Treatment: SGMO Prion ZF No Treatment SGMO STAC-BBB Prion RNA Repressor (ZFR) Intravenous Capsid NeuN NeuN Prion Prion Nonhuman prima
Slide 6: Differentiated neurology pipeline with franchise potential and industry validation
Differentiated neurology pipeline with franchise potential and industry validation NEUROLOGY PIPELINE – WHOLLY OWNED Indication Preclinical Phase 1/2 Pivotal Partner Cargo STAC-BBB Small Fiber Neuropathy (ST-503) - ✓ - Prion Disease (ST-506) - ✓ ✓ Undisclosed neurology target(s) - ✓ ✓ NEUROLOGY PIPE
Slide 7: ST-503 for chronic neuropathic pain
ST-503 for chronic neuropathic pain Clinical-stage chronic pain • Starting in small fiber neuropathy (SFN), a debilitating chronic neuropathic pain Indication program with compelling impacting 650,000 people across the US, Europe and Japan preclinical data, short Construct • Nav1.7 targeted zinc fin
Slide 8: ST-506 for prion disease
ST-506 for prion disease Compelling preclinical • A progressive condition leading to rapid neurodegeneration and death, Indication data with short timeframe to resulting in 1,500 deaths per year across the US, Europe and Japan expected clinical readout Construct • Prion targeted ZFR, delivered intra
Slide 9: Intravenous delivery via STAC-BBB
Intravenous STAC-BBB No AAV Treatment delivery via STAC-BBB NHP, 2e13 vg/kg STAC-BBB, 19 days post administration Mediates brain-wide • Widespread central nervous system (CNS) delivery is challenging with conventional Problem statement delivery in nonhuman AAVs primates and mice, • Enabled strong ex
Slide 10: Demonstrated industry interest in STAC-BBB
Demonstrated industry interest in STAC-BBB could provide significant economics for Sangamo STAC-BBB partnerships $88m Up to $4.6b Additional potential cash received from in potential future milestones and exercise fees product royalties partners to date assuming exercise of all options and targets 1
Slide 11: Next-generation, large-scale genome editing – MINT platform
Next-generation, large-scale genome editing – Modular Integrase (MINT) platform A versatile, protein-guided • Effectively and precisely integrating large synthetic DNA constructs into desired Problem statement genome editing method chromosomal sites is challenging designed to integrate large • Succe
Slide 12: Company Highlights
Company Highlights Developing epigenetic regulation for important gateway neurology diseases like chronic neuropathic pain and prion disease Proprietary AAV blood-brain barrier penetrant capsid (STAC-BBB) with industry leading CNS tropism in NHPs. Already the subject of license agreements with Genen
Slide 13: 4Q25 Business Updates
4Q25 Business Updates 13
Slide 14: 4Q25 Key Takeaways and Financial Highlights
4Q25 Key Takeaways Rolling submission of BLA to FDA seeking ST-920 approval is in progress under Accelerated Approval pathway Financial Highlights Fabry Disease As of December 31, 2025, we had cash and • The preclinical and clinical modules have been submitted to the FDA for review. In addition, the
Slide 15: Financial metrics
Financial metrics Historical Forward Looking $911m Up to $4.8b Cash received from In potential future milestones and exercise fees, assuming exercise of all partners to date options and targets $36.0m* ~$20.9m $110m – $130m** (2026) Cash and cash equivalents balance Non-GAAP OpEx guidance excludes c
Slide 16: Engineering Versatile Zinc Finger Payloads for Neurology
Engineering Versatile Zinc Finger Payloads for Neurology 16
Slide 17: Sangamo has the tools needed to advance a next-generation neurology genomic medicine company
Sangamo has the tools needed to advance a next-generation neurology genomic medicine company Potent Zinc Finger Cargo Small Size. Easily Packaged. Level of potency is precisely customizable to the Zinc fingers can be easily packaged into indication being targeted viral vectors Versatility and Exquis
Slide 18: Sangamo's differentiated genomic engineering platform
Sangamo’s differentiated genomic engineering platform is flexible, creating specific tools for the needs of each target Epigenetic Regulation 18
Slide 19: Zinc finger epigenetic regulators are the ideal cargo
Zinc finger epigenetic regulators are the ideal cargo for neurology-focused genomic medicines ZFR/ZFA ASO CRISPR Single administration Other n=244 Human derived Target any sequence Cell-type specificity ~ Compact / multiplexing ~ Nervous system Supplement with cDNA n=538 All RNA / protein ~ forms T
Slide 20: Sangamo's neurology portfolio provides opportunities
Sangamo’s neurology portfolio provides opportunities for wholly owned program advancement and potential partnering opportunities Chronic Prion W H O L LY Neuropathic Disease OWNED Pain PRIORITY Nav1.7 PRNP PROGRAMS ALS Huntington’s Tauopathies Undisclosed Undisclosed Undisclosed Disease neurology ne
Slide 21: Epigenetic regulation to address chronic neuropathic pain
Epigenetic regulation to address chronic neuropathic pain 21
Slide 22: Targeting the Nav1.7 pathway at the DNA level
Targeting the Nav1.7 pathway at the DNA level, we seek to succeed where others have failed Clinical-stage chronic pain program with compelling preclinical data, short timeframe to expected clinical readout, and pain franchise potential • Nav1.7 is a voltage gated sodium channel expressed in the Dors
Slide 23: Zinc finger repressors reduced Nav1.7 in human neurons
Zinc finger repressors potently reduced Nav1.7 in human neurons with a high level of specificity Potent and dose-dependent Selective repression Specific repression of Nav1.7 repression of SCN9A gene, of SNC9A, out of >20,000 genes without impacting other sodium which encodes Nav1.7 tested channels i
Slide 24: Nav1.7 repressor reversed neuropathic pain in mouse models
Nav1.7 repressor reversed neuropathic pain in mouse models Full restoration of normal ~40% repression of SCN9A Efficacy is observed as early Potent Scn9a mRNA sensitivity to mechanical is sufficient to induce more repression in mouse as Day 3 post-treatment pain than 50% pain recovery 2.0 Mechanical
Slide 25: Potent and selective repression of SCN9A observed in NHPs
Potent and selective repression of SCN9A observed in NHPs, with no signs of toxicity or adverse clinical pathology Specific repression of Nav1.7 Potent and dose-dependent Selective repression without impacting repression of SCN9A gene of SNC9A other sodium channels ✱✱✱ ✱✱✱ hZFR Scn9a 2.0 Normalized
Slide 26: First six clinical sites activated in Phase 1/2 STAND study
First six clinical sites have been activated in Phase 1/2 STAND study Cohort 3 High dose Cohort 2 n=6 Cohort 1 Mid dose Investigational product n=3 Low dose Sham n=3 FDA clearance of IND received to assess ST-503 in SFN patients (which impacts 650,000 people across the US, Europe and Japan) Preparin
Slide 27: Epigenetic regulation to address prion disease, leveraging STAC-BBB
Epigenetic regulation to address prion disease, leveraging STAC-BBB 27
Slide 28: Prion disease is rapidly progressive and always fatal
Prion disease is rapidly progressive and always fatal Compelling preclinical data with short timeframe to expected clinical readout in a deadly disease with no currently approved treatment options • Progressive condition, with no disease modifying therapy • Caused by the misfolding of the prion prot
Slide 29: Persistent ZFR activity up to 17-months
Persistent ZFR activity up to 17-months suggests the potential for lifelong prion suppression in neurons Sustained brainwide Prnp mRNA repression Sustained brain wide PrP knockdown at 17 months 125 % Normalized Prnp Vehicle 100 expression 75 5.5 months 17 months 50 25 O ere tem b f C tex in ca us y
Slide 30: Zinc finger repressors extended survival in prion model
Zinc finger repressors extended survival in a mouse model of aggressive prion disease, even when administered post-symptomatically 119 dpi 100 Uninoculated 75 High dose % Survival MST >494 dpi 50 Vehicle Mid dose MST 462 dpi MST 169.5 dpi 25 Low dose Days post inoculation (dpi) MST 340 dpi 0 0 50 10
Slide 31: STAC-BBB mediated ZFR expression and Prion repression in NHP brain
STAC-BBB mediated ZFR expression and Prion repression in the NHP brain ZFR+ cells (GFP) Neurons (NeuN) Vehicle Control Motor cortex STAC-BBB Prion mRNA STAC-BBB transgene encodes a nuclear-localized GFP and PRNP-targeted ZFR Data presented at ASGCT 2025 2e13 vg/kg dose, 19 days post administration 3
Slide 32: ST-506 mediated prion repression in NHPs
ST-506 mediated prion repression in NHPs that matched or exceeded levels associated with profound survival extension in mice Brainstem Prion disease model % Normalized expression 462 >494 100 125 125 % Survival 100 100 75 75 75 50 50 50 25 25 25 0 0 0 e 3 4 0 100 200 300 400 500 c l +1 +1 3E le 1E 1
Slide 33: Phase 1/2 CTA-enabling activities are ongoing
Phase 1/2 CTA-enabling activities and clinical study preparations are ongoing CTA-enabling activities in progress with GLP Toxicology study complete and analysis ongoing Clinical study expected to be a Bayesian Optimal Interval (BOIN) design to assess safety and efficacy Study expected to use the MR
Slide 34: Achieving Widespread Central Nervous System Delivery
Achieving Widespread Central Nervous System Delivery for Optimal Therapeutic Benefit 34
Slide 35: Widespread CNS delivery is challenging with conventional AAVs
Widespread CNS delivery is challenging with conventional AAVs. Our SIFTER platform is designed to enable the selection of neurotropic AAV capsids to potentially advance our innovative preclinical programs to the clinic. SIFTER: Selecting In vivo For Transduction and Expression of RNA 35
Slide 36: Sangamo STAC-BBB findings exceeded expectations
Sangamo STAC-BBB STAC-BBB achieved robust penetration of the blood-brain barrier and findings exceeded widespread distribution throughout the brain in NHPs expectations for a successful blood- Industry-leading performance: 700-fold better enrichment than the brain barrier benchmark AAV9 penetrant ca
Slide 37: In vivo library evaluation identified STAC-BBB
In vivo library evaluation in cynomolgus macaques identified STAC-BBB as the top performing BBB-penetrant capsid, with additional enhancements in progress Capsid-mediated expression of cargo in neurons 644-fold enrichment in brain Unique Molecular Log2 Fold Change Identifier count (Color): (Y-axis):
Slide 38: Neuronal mRNA Expression
A nt er i or A Log2 Fold Change in C ng Neuronal mRNA Expression in u gu la E la G r 0 2 4 6 8 -6 -4 -2 10 Fr n on tor te Gyru to hi C y s In Inf F -or nal un rus fe er u b c eu ri io si ita or s or r fo l te t fr rm gy x La em ont g ru te p a yr s ra oral gy us l l r M Mid orb gy us id d L it In ru
Slide 39: STAC-BBB showed widespread neuronal transduction
STAC-BBB showed widespread neuronal transduction across all cortical regions Precentral Gyrus (Motor Cortex) Postcentral Gyrus Superior Temporal Gyrus Middle Temporal Gyrus Inferior Temporal Gyrus 2e13 vg/kg STAC-BBB, 19 days post administration 39
Slide 40: STAC-BBB mediated widespread brain transduction
STAC-BBB mediated widespread brain transduction Putamen Caudate Substantia nigra Globus pallidus Pons Dentate nucleus Cuneate nucleus Thalamus Hippocampus (CA2) Lateral geniculate nucleus Temporal cortex Motor cortex 2e13 vg/kg STAC-BBB, 19 days post administration 40
Slide 41: Neurons were widely transduced in disease regions
Neurons were widely transduced in regions integral to disease pathology Putamen Caudate Substantia nigra Globus pallidus Huntington’s Disease Huntington’s Disease Huntington’s Disease Parkinson’s Disease Parkinson’s Disease Parkinson’s Disease Parkinson’s Disease Pons Dentate nucleus Cuneate nucleus
Slide 42: We believe STAC-BBB is manufacturable at scale
We believe STAC-BBB is manufacturable at scale • Capsid manufacturability is critical to create a successful potential commercial drug product for patients • We believe STAC-BBB is: • Manufacturable at commercial scale using standard cell culture and purification processes • Soluble using known exci
Slide 43: Advancing Next-Generation Genome Engineering
Advancing Next-Generation Genome Engineering 43
Slide 44: What is an integrase and why is it important?
What is an integrase and why is it important? Targeted integration of a therapeutic paragraph of DNA (a gene) ..... Targeted integration enables large-scale genome editing ...vs. single base editing. Capable of delivering large payloads - 10 kb+ Bxb1 Integration Mechanism No copying required - low e
Slide 45: Targeted integration improves existing therapies
Targeted integration improves existing therapies, and enables new therapies Targeted vs. Random Integration One medicine vs. multiple variants for each mutation Multiple mutations requiring multiple medicines Cell 1 All cells, Patient 2 Stop one gene location Cell 2 Exon 1 Exon 2 Patient 1 Patient 3
Slide 46: Optimizing Value of Clinical Programs
Optimizing Value of Clinical Programs 46
Slide 47: Fabry Disease: Isaralgagene Civaparvovec
Fabry Disease: Isaralgagene Civaparvovec Rolling submission of BLA to FDA in progress Best-in-class Fabry gene therapy program • All 32 Phase 1/2 STAAR study patients rolled into long-term follow-up Compelling clinical data • Potential to be a one-time, durable treatment to provide meaningful, multi
Slide 48: Hemophilia A: Giroctocogene fitelparvovec
Hemophilia A: Giroctocogene fitelparvovec Compelling readout for Phase 3 AFFINE trial Pfizer reported positive topline results from the Phase 3 AFFINE trial in July 2024, which met primary and key secondary endpoints Phase 3 data presented at ASH Annual Meeting and Exposition in December 2024 via pl
Slide 49: Sangamo Value Proposition
Sangamo Value • Differentiated genomic medicine platform combines core capabilities Proposition required to unlock treatments for devastating neurological diseases • Genome-targeting epigenetic regulation cargo • Capsid delivery engine for central nervous system administration • Focused neurology pi