Sangamo Therapeutics, Inc. · NASDAQ:SGMO
Sangamo Therapeutics — Q2 2025 Earnings Presentation
51 slides from Sangamo Therapeutics's earnings presentation, Aug 7, 2025
Slide 1: Delivering the Future of Genomic Medicines
Delivering the Future of Genomic Medicines August 2025
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 is a differentiated genomic medicine company focused on neurological diseases
Sangamo is a differentiated genomic medicine company focused on treating debilitating neurological diseases Potent zinc finger Industry-leading AAV Powerful research platform Strong roster of current partners epigenetic regulation capsid discovery platform continually innovates in new and a clear re
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: Epigenetic regulation and capsid delivery capabilities for neurology genomic medicines
Sangamo pairs the epigenetic regulation and capsid delivery capabilities needed to create neurology genomic medicines Genome-Targeting Cargo Capsid Delivery Engine Epigenetic regulation platform AAV capsid delivery platform via intravenous delivery ZFR Prion RNA SGMO Intravenous Negative Control Cap
Slide 6: Company pipeline and business development opportunities
Company pipeline and business development opportunities NEUROLOGY PIPELINE – WHOLLY OWNED Indication Preclinical Phase 1/2 Pivotal Partner Commentary Idiopathic Small Fiber Neuropathy First clinical site initiated in Phase 1/2 STAND study. - (ST-503) Dosing expected fall 2025. Prion Disease - CTA su
Slide 7: Gateway neurology indication: ST-503 for chronic neuropathic pain
Gateway neurology KEY ANTICIPATED indication: MILESTONES ST-503 for chronic neuropathic pain Fall 2025: Initiate patient dosing Q4 2026: Preliminary proof of efficacy data Starting in idiopathic small fiber neuropathy (iSFN), a debilitating chronic neuropathic pain impacting 43,000 in the U.S. Nav1.
Slide 8: Gateway neurology indication: Prion disease
Gateway neurology KEY ANTICIPATED indication: MILESTONES Prion disease As early as mid-2026: Prion CTA submission Late-2026: Clinical trial enrollment and dosing Mid-2027: Preliminary clinical data Progressive condition leading to rapid neurodegeneration and death, with no disease modifying therapy
Slide 9: Widespread CNS delivery is challenging with conventional AAVs
Widespread CNS STAC-BBB Negative control (Nuclear-localized GFP) (no AAV treatment) delivery is challenging with conventional AAVs Our capsid engineering platform has demonstrated the ability to expand delivery, with industry- leading results 2e13 vg/kg STAC-BBB, 19 days post administration Enabled
Slide 10: STAC-BBB partnerships
Biopharma agreements have demonstrated industry interest in STAC-BBB and could provide significant economics for Sangamo Numerous Benefits of Partnerships: STAC-BBB Partner buy-in validates the partnerships science Potential for additional STAC-BBB license agreements Provides potential non-dilutive
Slide 11: Company Highlights
Company Advancing epigenetic regulation for important gateway neurology diseases like Highlights chronic neuropathic pain and prion disease, with preliminary clinical data anticipated in Q4 2026 for iSFN Proprietary AAV blood-brain barrier penetrant capsid (STAC-BBB) with industry leading CNS tropis
Slide 12: 2Q25 Business Updates
2Q25 Business Updates 12
Slide 13: 2Q25 Key Takeaways
2Q25 Key Takeaways Announced positive topline results from registrational STAAR study in Fabry disease, including positive mean annualized eGFR slope at 52-weeks across all dosed patients, which FDA has agreed will serve as primary basis of approval. Financial Highlights Neurology Pipeline • Initiat
Slide 14: Q2 Pipeline Progress & Anticipated Milestones
CORPORATE UPDATES ✓ Raised approximately $21 million in net proceeds from an underwritten registered equity offering. ▪ Continue to engage in potential business development discussions across the Sangamo pipeline and platforms. NEUROLOGY ✓ Initiated first clinical site in Phase 1/2 STAND study of ST
Slide 15: Financial metrics
Financial metrics Historical Forward Looking $910m Up to $6.1b Cash received from In potential future milestones and exercise fees, assuming exercise of all partners to date options and targets $33.0m* ~$38.3m $125m – $145m** (2025) 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: Tools 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: Flexible 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 for neurology
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: Neurology portfolio: wholly owned and partnering 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 Phelan- Myotonic Haploin- C U R R E N T LY Dravet Charcot Marie Charcot Marie McDermi
Slide 21: Epigenetic regulation to address chronic neuropathic pain
Epigenetic regulation to address chronic neuropathic pain 21
Slide 22: The urgent need for novel chronic neuropathic pain therapeutics
The urgent need for novel chronic Past Present Future neuropathic Anti-inflammatories Pain signal Epigenetic pain therapeutics Analgesics inhibitors regulation Opioids ST-503 is an investigational epigenetic regulator for the treatment of intractable, chronic neuropathic pain Peripheral neuropathies
Slide 23: 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 ST-503 targets a gene validated by human genetics and leverages an AAV delivery capsid already in the clinic • A significant body of evidence implicates sodium channels in mediating the pathophysiology of neur
Slide 24: Zinc finger repressors potently reduced Nav1.7 in human neurons
Zinc finger repressors potently reduced Nav1.7 in human neurons with high level of specificity Potent and dose-dependent Selective repression Specific repression of Nav1.7 repression of SCN9A gene, of SNC9A as shown without impacting other sodium which encodes Nav1.7 by global transcriptome analysis
Slide 25: Nav1.7 repressor reversed neuropathic pain in mouse models
Nav1.7 repressor reversed neuropathic pain in preclinical mouse models Potent Scn9a mRNA repression in mouse Full restoration of normal Lumbar DRG nociceptors sensitivity to mechanical pain Intrathecal Data Collected on Day 28 lumbar injection 2.5 Mechnical Threshold 2.0 **** **** 1.5 **** (g) 1.0 0
Slide 26: Potent and selective repression of SCN9A observed in NHPs
Potent and selective repression of SCN9A observed in NHPs, with no clinical signs of toxicity or adverse clinical pathology Potent and dose-dependent Selective repression Specific repression of Nav1.7 repression of SCN9A gene, of SNC9A as shown without impacting which encodes Nav1.7 by single cell a
Slide 27: First clinical site initiated; proof of efficacy data anticipated Q4 2026
First clinical site has been initiated, with preliminary proof of efficacy data anticipated in Q4 2026 Cohort 3 High dose Cohort 2 n=9 Cohort 1 Mid dose Investigational product n=3 Low dose Sham n=3 FDA clearance of IND received November 2024 to assess ST-503 in iSFN patients Preparing for double-bl
Slide 28: Epigenetic regulation to address prion disease, leveraging STAC-BBB
Epigenetic regulation to address prion disease, leveraging STAC-BBB 28
Slide 29: Prion disease is rapidly progressive and always fatal
Prion disease is rapidly progressive and always fatal Path to potential clinical validation in a devastating disease with no current approved treatment options • Progressive condition, with no disease modifying therapy • Caused by the misfolding of the prion protein (PrP) into toxic species, leading
Slide 30: Zinc finger repressors extended survival in mouse 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 widespread expression in nonhuman primate brain
STAC-BBB demonstrated widespread and robust expression throughout the nonhuman primate brain STAC-BBB Negative control (Nuclear-localized GFP) (no AAV treatment) – No signal Nissl staining (light blue): Grey matter All cell nuclei (cell bodies) Antibody labeling for green florescent protein (GFP) ex
Slide 32: 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 Multiplexed RNAscope ISH / IHC assay for NeuN, GFP, PRNP mRNA, and ZFR mRNA
Slide 33: 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 Cortex Prion disease model % Normalized expression 462 >494 100 125 125 125 125 % Survival 100 100 100 100 75 75 75 75 75 50 50 50 50 50 25 25 25 25 25 0 0 0 0 0 e 3
Slide 34: Phase 1/2 CTA-enabling activities and clinical study preparations ongoing
Phase 1/2 CTA-enabling activities and clinical study preparations are ongoing CTA submission anticipated as early as mid-2026 Clinical study expected to be a Bayesian Optimal Interval (BOIN) design to assess safety and efficacy, while potentially enabling rapid escalation to maximum tolerated dose S
Slide 35: Achieving Widespread CNS Delivery for Optimal Therapeutic Benefit
Achieving Widespread Central Nervous System Delivery for Optimal Therapeutic Benefit 35
Slide 36: SIFTER platform for selecting neurotropic AAV capsids
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 36
Slide 37: 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 38: In vivo library evaluation identified STAC-BBB as top performing capsid
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 39: 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 40: STAC-BBB widespread neuronal transduction across cortical regions
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 40
Slide 41: 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 41
Slide 42: Neurons widely transduced in regions integral to disease pathology
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 43: 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 44: Advancing Next-Generation Genome Engineering
Advancing Next-Generation Genome Engineering 44
Slide 45: 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 46: Targeted integration improves existing therapies, and enables new 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 47: Optimizing Value of Clinical Programs
Optimizing Value of Clinical Programs 47
Slide 48: Fabry Disease: Isaralgagene civaparvovec (ST-920)
Fabry Disease: Isaralgagene civaparvovec (ST-920) Abbreviated clinical pathway supports efforts to secure a commercialization partner Largest known gene therapy program in Fabry disease • All 32 patients in the Phase 1/2 STAAR study have now rolled into the long-term follow-up study Positive topline
Slide 49: Fabry Disease: summary of positive topline readout, June 2025
Fabry Disease: isaralgagene civaparvovec (ST-920) Summary of positive topline readout, June 2025 • Following a single dose of ST-920, a positive mean annualized eGFR slope of 1.965 mL/min/1.73m 2/year (95% confidence interval (CI): -0.153, 4.083) at 52-weeks was observed across all 32 dosed patients
Slide 50: 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 51: Sangamo is a differentiated genomic medicine company focused on neurological diseases
Sangamo is a differentiated genomic medicine company focused on treating debilitating neurological diseases Potent zinc finger Industry-leading AAV Powerful research platform Strong roster of current partners epigenetic regulation capsid discovery platform continually innovates in new and a clear re