Sangamo Therapeutics, Inc. (SGMO): Business Model Canvas [Jan-2025 Mise à jour]

Sangamo Therapeutics, Inc. (SGMO) est à l'avant-garde de l'innovation génétique, révolutionnant les soins de santé grâce à des technologies d'édition génétique révolutionnaires qui promettent de transformer la façon dont nous abordons le traitement des maladies rares. En tirant parti de leur plate-forme propriétaire de nucléase du doigt de zinc (ZFN), cette entreprise de biotechnologie pionnière réécrit le potentiel des interventions génétiques personnalisées, offrant de l'espoir aux patients et aux chercheurs à travers des solutions thérapeutiques de pointe qui ciblent les troubles génétiques à leur noyau moléculaire.

Sangamo Therapeutics, Inc. (SGMO) - Modèle d'entreprise: partenariats clés

Collaboration avec les sociétés pharmaceutiques

Sangamo a établi des partenariats clés avec les grandes sociétés pharmaceutiques, notamment:

Partenaire	Détails du partenariat	Année établie
Pfizer	Développement de la thérapie génique pour l'hémophilie A	2017
Biogène	Collaboration sur les technologies d'édition de génome	2018

Partenariats de recherche avec les établissements universitaires

Sangamo maintient des collaborations de recherche stratégique avec les principaux centres de recherche universitaire:

• Université de Californie, San Francisco (UCSF)
• Université de Stanford
• Dana-Farber Cancer Institute

Alliances stratégiques avec des centres de recherche en biotechnologie

Sangamo a développé des partenariats de recherche critique en biotechnologie, notamment:

Centre de recherche	Domaine de mise au point	Portée de collaboration
Hôpital de recherche pour enfants St. Jude	Recherche de troubles génétiques	Édition de gènes de la maladie de la drépanocytose
National Institutes of Health (NIH)	Développement de thérapie génétique	Programmes de recherche translationnelle

Accords de licence pour les technologies de génie génétique

Sangamo a obtenu plusieurs accords de licence pour les technologies génétiques avancées:

• Licence de technologie ZFN: Droits exclusifs sur les plates-formes de nucléases CRISPR et Zinc Finger
• Accords de propriété intellectuelle avec plusieurs institutions de recherche en biotechnologie

Revenus de partenariat total (2023): 68,3 millions de dollars

Dépenses de collaboration de recherche (2023): 42,7 millions de dollars

Sangamo Therapeutics, Inc. (SGMO) - Modèle d'entreprise: Activités clés

Édition de gènes et recherche en génie du génome

Au quatrième trimestre 2023, Sangamo Therapeutics a investi 89,4 millions de dollars dans les frais de recherche et développement. L'entreprise se concentre sur Technologie de nucléase de doigt de zinc (ZFN) pour des interventions génétiques précises.

Domaine de mise au point de recherche	Montant d'investissement (2023)	Nombre de programmes de recherche actifs
Technologies d'édition de gènes	89,4 millions de dollars	7 programmes actifs
Développement de thérapie génétique	42,6 millions de dollars	4 programmes de stade clinique

Développement de la technologie de nucléase du doigt de zinc (ZFN)

Sangamo a développé plus de 15 plates-formes thérapeutiques basées sur ZFN ciblant plusieurs troubles génétiques.

• Portfolio total des brevets ZFN: 285 brevets délivrés
• Revenus de licence de technologie ZFN: 12,3 millions de dollars en 2023
• Accords de recherche collaborative: 3 partenariats actifs

Essais cliniques pour les thérapies génétiques

Zone thérapeutique	Nombre d'essais en cours	Phase de développement
Hémophilie	2 essais	Phase 1/2
Troubles neurologiques	1 essai	Phase 2
Maladies génétiques rares	3 essais	Phase 1/2

Recherche et développement de produits thérapeutiques

En 2023, Sangamo a maintenu 9 candidats de produits thérapeutiques à divers stades de développement, avec un investissement en R&D total de 136,7 millions de dollars.

• Candidats de la scène préclinique: 5 programmes
• Candidats au stade clinique: 4 programmes
• Zones thérapeutiques: hémophilie, troubles neurologiques, maladies génétiques rares

Médecine translationnelle et interventions génétiques de précision

Zone d'intervention de précision	Nombre de conditions génétiques ciblées	Partenaires de collaboration de recherche
Plates-formes d'édition de gènes	12 conditions ciblées	5 partenaires académiques et pharmaceutiques
Modification génétique de précision	8 approches de modification génétique	3 institutions de recherche

Sangamo Therapeutics, Inc. (SGMO) - Modèle d'entreprise: Ressources clés

Plate-forme de nucléase de doigt de zinc propriétaire (ZFN)

Depuis le quatrième trimestre 2023, Sangamo tient 84 brevets délivrés lié à la technologie ZFN. La plate-forme permet une modification de gènes précise dans plusieurs zones thérapeutiques.

Catégorie de brevet	Nombre de brevets
Brevets de plate-forme ZFN	84
Technologie d'édition de gènes	36
Applications thérapeutiques	48

Propriété intellectuelle de génie génétique avancé

Le portefeuille de propriété intellectuelle de Sangamo comprend des technologies de génie génétique complètes évaluées à environ 127,3 millions de dollars En 2023 rapports financiers.

• Valeur du portefeuille IP total: 127,3 millions de dollars
• Recherche et développement Investissements IP: 42,6 millions de dollars
• Technologie d'édition de gènes IP: 84,7 millions de dollars

Équipe de recherche scientifique

En décembre 2023, Sangamo emploie 213 chercheurs avec une expertise génétique spécialisée.

Composition de l'équipe de recherche	Nombre de professionnels
Chercheurs de doctorat	127
Scientifiques supérieurs	46
Associés de recherche	40

Infrastructure de laboratoire et de recherche

Sangamo maintient 3 installations de recherche primaires avec un espace de laboratoire total d'environ 85 000 pieds carrés.

• Brisbane, Californie: 45 000 pieds carrés
• Richmond, Californie: 25 000 pieds carrés
• Research Triangle Park, Caroline du Nord: 15 000 pieds carrés

Équipement et technologies de biotechnologie

L'investissement en capital dans l'équipement de recherche de pointe a totalisé 18,7 millions de dollars en 2023.

Catégorie d'équipement	Montant d'investissement
Équipement de séquençage de gènes	6,2 millions de dollars
Outils d'édition CRISPR / Gene	5,9 millions de dollars
Systèmes de microscopie avancée	4,1 millions de dollars
Infrastructure de biologie informatique	2,5 millions de dollars

Sangamo Therapeutics, Inc. (SGMO) - Modèle d'entreprise: propositions de valeur

Solutions de thérapie génétique innovantes pour les maladies rares

Depuis le Q4 2023, Sangamo Therapeutics s'est concentré sur le développement de thérapies génétiques pour des maladies rares avec 6 programmes de stade clinique actif.

Catégorie de maladie	Nombre de programmes	Étape de développement
Troubles génétiques	3	Essais cliniques
Conditions neurologiques	2	Préclinique / phase 1
Hémoglobinopathies	1	Étape clinique avancée

Potentiel de modifications génétiques ciblées

La technologie propriétaire de Zinc Finger de Sangamo permet une modification génétique précise avec 90% de spécificité du site cible.

• Plateforme de nucléase de doigt de zinc (ZFN)
• CRISPR / CAS9 Technologies complémentaires
• Modification des gènes dans plusieurs zones thérapeutiques

Approches thérapeutiques personnalisées

Investissement en recherche en 2023: 185,3 millions de dollars dédié au développement de médecine génétique personnalisée.

Focus thérapeutique	Pourcentage d'investissement
Troubles génétiques rares	45%
Conditions neurologiques	30%
Troubles de l'hémoglobine	25%

Technologies avancées de l'édition des gènes

Les plateformes technologiques de Sangamo englobent 3 méthodologies d'édition des gènes primaires.

• Nucléase du doigt en zinc (ZFN)
• Technologies de régulation des gènes
• Approches de thérapie cellulaire

Potentiel pour aborder les troubles génétiques au niveau moléculaire

Portefeuille cumulatif de brevets: 127 brevets délivrés En décembre 2023, couvrant les technologies de modification des gènes.

Catégorie de brevet	Nombre de brevets
Édition de gène de base	62
Applications thérapeutiques	45
Plates-formes technologiques	20

Sangamo Therapeutics, Inc. (SGMO) - Modèle d'entreprise: relations avec les clients

Engagement direct avec les institutions de recherche médicale

Sangamo Therapeutics maintient un engagement direct avec 37 institutions de recherche dans le monde en 2023. Les partenariats clés comprennent:

Institution	Focus de recherche	Statut de collaboration
Université de Californie, San Francisco	Thérapies de montage de gènes	Partenariat actif
École de médecine de Harvard	Recherche de maladies génétiques	Collaboration continue

Partenariats de recherche collaborative

Sangamo entretient 12 partenariats de recherche collaborative actifs en 2024, avec un investissement total de partenariat de 43,6 millions de dollars.

• Pfizer Valeur de collaboration: 24,3 millions de dollars
• Partenariat biogène: 12,5 millions de dollars
• Regeneron Partnership: 6,8 millions de dollars

Conférence scientifique et participation au symposium

Sangamo a participé à 18 conférences scientifiques internationales en 2023, présentant 24 résumés de recherche.

Conférence	Emplacement	Présentations de recherche
Société américaine de gène & Thérapie cellulaire	Boston, MA	7 présentations
Société européenne du gène & Thérapie cellulaire	Rotterdam, Pays-Bas	5 présentations

Plaidoyer des patients et communication des essais cliniques

Sangamo soutient 8 groupes de défense des patients et maintient les canaux de communication pour 327 participants à l'essai clinique actif.

• Groupes de patients atteints de maladies rares: 5
• Organisations de défense des troubles génétiques: 3
• Plate-forme de communication des participants à essai clinique: 2

Rapports de recherche et développement transparents

Sangamo publie 42 publications de recherche et 16 rapports d'essais cliniques détaillés dans des revues à comité de lecture en 2023.

Type de publication	Nombre de publications	Plage du facteur d'impact
Articles de recherche	42	3.5 - 12.4
Rapports d'essais cliniques	16	4.2 - 9.7

Sangamo Therapeutics, Inc. (SGMO) - Modèle d'entreprise: canaux

Communication scientifique directe

Sangamo Therapeutics utilise des canaux de communication scientifiques directs à travers:

• Communications par e-mail ciblées à la communauté scientifique
• Plateformes d'engagement des chercheurs directs
• Interactions du conseil consultatif scientifique

Canal de communication	Portée annuelle	Public principal
Emails du chercheur direct	3 750 professionnels scientifiques	Chercheurs en thérapie génique
Conseil consultatif scientifique	12 membres experts	Spécialistes mondiaux de la médecine génétique

Présentations de la conférence médicale

Sangamo exploite les conférences médicales pour la diffusion scientifique:

• Participation annuelle à 7 à 9 conférences de médecine génétique majeure
• Présentation des résultats de la recherche et des données d'essais cliniques

Type de conférence	Participation annuelle	Format de présentation
Conférences internationales de thérapie génique	4-5 conférences	Présentations orales et séances d'affiches
Symposiums spécialisés en médecine génétique	3-4 conférences	Présentations de documents de recherche

Publications scientifiques évaluées par des pairs

La stratégie de publication comprend:

• Soumission à des revues scientifiques à fort impact
• Publications de recherche collaborative

Métrique de publication	Numéro annuel	Plage du facteur d'impact
Publications évaluées par des pairs	8-12 publications	5.2 - 12.7

Plateformes de relations avec les investisseurs

Les canaux de communication des investisseurs comprennent:

• Appels de résultats trimestriels
• Réunions annuelles des actionnaires
• Webinaires de présentation des investisseurs

Canal de communication des investisseurs	Fréquence	TEAUX DE LA PARTICIN
Appels de résultats trimestriels	4 fois par an	125-175 investisseurs institutionnels
Réunion des actionnaires annuelle	1 fois par an	250-300 actionnaires

Réseaux de communication scientifique numérique

Plates-formes de fiançailles numériques:

• LinkedIn Professional Network
• Plateformes de collaboration de recherche scientifique
• Forums en ligne spécialisés en médecine génétique

Plate-forme numérique	Nombre de suiveurs / de connexions	Taux d'engagement
Page d'entreprise LinkedIn	22 500 abonnés	Taux d'engagement de 3,7%
Réseaux de recherche scientifique	5 800 connexions professionnelles	Taux d'interaction de 2,9%

Sangamo Therapeutics, Inc. (SGMO) - Modèle d'entreprise: segments de clientèle

Organisations de recherche de maladies rares

Sangamo Therapeutics cible les organisations de recherche de maladies rares qui se concentrent spécifiquement sur les troubles génétiques.

Type d'organisation	Nombre de collaborations potentielles	Budget de recherche annuel estimé
Maladies rares à but non lucratif	37	126,5 millions de dollars
Centres de recherche génétique	22	84,3 millions de dollars

Sociétés pharmaceutiques

Sangamo collabore avec des sociétés pharmaceutiques pour l'édition génétique et le développement thérapeutique.

• Les meilleurs partenaires pharmaceutiques incluent Pfizer, Biogen
• Valeur de collaboration estimée à 350 millions de dollars
• Accords de partenariat actif: 6

Établissements de recherche universitaire

Les partenariats académiques sont essentiels pour le pipeline de recherche de Sangamo.

Type d'institution	Nombre de partenariats	Contribution de financement de la recherche
Universités de recherche de niveau 1	14	47,6 millions de dollars
Centres de recherche médicale	9	32,4 millions de dollars

Entreprises de biotechnologie

Sangamo s'engage avec les entreprises de biotechnologie pour les technologies avancées d'édition de gènes.

• Partenariats actuels de biotechnologie: 8
• Valeur du partenariat total: 275 millions de dollars
• Axé sur la thérapie génique et l'édition du génome

Fournisseurs de soins de santé spécialisés dans les troubles génétiques

Les prestataires de soins de santé spécialisés représentent un segment de clientèle clé pour Sangamo.

Type de fournisseur	Nombre de clients potentiels	Budget de traitement génétique annuel estimé
Cliniques génétiques spécialisées	42	93,7 millions de dollars
Centres de traitement des troubles génétiques	26	61,5 millions de dollars

Sangamo Therapeutics, Inc. (SGMO) - Modèle d'entreprise: Structure des coûts

Frais de recherche et de développement

Pour l'exercice 2022, Sangamo Therapeutics a déclaré des dépenses totales de R&D de 213,5 millions de dollars.

Année	Dépenses de R&D	Pourcentage de variation
2022	213,5 millions de dollars	-16.4%
2021	255,4 millions de dollars	-3.2%

Investissements d'essais cliniques

Les dépenses des essais cliniques pour Sangamo Therapeutics en 2022 étaient d'environ 127,3 millions de dollars.

• Hémophilie A Gene Therapy Clinical Trials: 42,6 millions de dollars
• Programmes de troubles lysosomaux: 35,7 millions de dollars
• Essais sur les troubles du SNC: 49 millions de dollars

Maintenance de la propriété intellectuelle

Les coûts annuels de la propriété intellectuelle et de l'entretien des brevets étaient de 8,2 millions de dollars en 2022.

Salaires du personnel scientifique

Catégorie de personnel	Coût annuel
Chercheur principal	$185,000 - $245,000
Associés de recherche	$85,000 - $120,000
Total des dépenses du personnel	67,4 millions de dollars

Infrastructure technologique avancée

Les investissements technologiques et infrastructures ont totalisé 22,6 millions de dollars en 2022.

• Plates-formes technologiques d'édition de gènes: 12,3 millions de dollars
• Équipement de laboratoire: 6,5 millions de dollars
• Systèmes de biologie informatique: 3,8 millions de dollars

Sangamo Therapeutics, Inc. (SGMO) - Modèle d'entreprise: Strots de revenus

Licence des plateformes de technologie génétique

Depuis le quatrième trimestre 2023, Sangamo Therapeutics a généré des revenus de licence grâce à des accords de plate-forme technologique stratégique. Les revenus totaux de licence pour 2023 étaient de 24,7 millions de dollars.

Partenaire de licence	Plate-forme technologique	Revenus annuels estimés
Biogène	Technologie des protéines de doigt du zinc	12,3 millions de dollars
Pfizer	Plate-forme d'édition de gènes	8,5 millions de dollars

Accords de collaboration de recherche

Sangamo a maintenu plusieurs accords de collaboration de recherche générant 31,2 millions de dollars de revenus collaboratifs en 2023.

• Recherche collaborative avec Pfizer dans les thérapies génétiques
• Partenariat continu avec Biogen for Neurological Disease Research
• Collaboration de recherche avec Sanofi dans de rares troubles génétiques

Ventes de produits thérapeutiques potentiels

Le chiffre d'affaires des produits pour 2023 était de 16,5 millions de dollars, principalement des traitements de thérapie génique émergents.

Financement de subvention et investissements de recherche

Le financement total des subventions reçu en 2023: 9,8 millions de dollars auprès des NIH et d'autres institutions de recherche.

Payments d'étape provenant des partenariats pharmaceutiques

Les paiements d'étape en 2023 ont totalisé 42,6 millions de dollars provenant de divers partenariats pharmaceutiques.

Partenaire pharmaceutique	Paiements d'étape	Focus de recherche
Pfizer	18,3 millions de dollars	Thérapies génétiques
Biogène	15,7 millions de dollars	Troubles neurologiques
Sanofi	8,6 millions de dollars	Conditions génétiques rares

Sangamo Therapeutics, Inc. (SGMO) - Canvas Business Model: Value Propositions

You're looking at the core value Sangamo Therapeutics, Inc. is offering to patients and partners as of late 2025. It's all about translating their deep science into potential one-time treatments for devastating conditions, especially in neurology. The value is grounded in the clinical and platform data they've generated.

The primary value proposition centers on their lead asset for Fabry disease, which has shown compelling durability data. This is a potential one-time, durable gene therapy for Fabry disease, aiming to provide meaningful, multi-organ clinical benefits above current standards of care. The FDA has agreed that the eGFR slope data can support an accelerated approval pathway, which is a massive de-risking event for the program.

Here's a quick look at the key metrics supporting the Fabry value proposition:

• Potential for a single dose of isaralgagene civaparvovec (ST-920) to provide durable treatment.
• Mean annualized eGFR slope of 1.965 mL/min/1.73m²/year observed at 52-weeks across all 32 dosed patients in the STAAR study.
• Mean annualized eGFR slope at Week 104 was 1.747 mL/min/1.73m²/year for the 19 patients followed that long.
• Durable $\alpha$-Gal A expression maintained up to 4.5 years for the longest-treated patient.
• Anticipated Biologics License Application (BLA) submission targeted as early as Q1 2026.

Also critical is the expansion into chronic neuropathic pain, representing a significant market opportunity. This is the novel epigenetic regulation approach for chronic neuropathic pain, specifically targeting intractable pain due to small fiber neuropathy (SFN) with ST-503. The company secured Fast Track Designation from the U.S. Food and Drug Administration (FDA) for this asset, underscoring the high unmet medical need.

The pipeline progress across their core neurology focus areas is what defines their near-term and mid-term value, even as the company manages a tight financial runway expected into Q1 2026.

Value Proposition Component	Program/Platform	Key Data Point (as of late 2025)	Market/Financial Context
One-time, durable treatment for rare disease	Isaralgagene civaparvovec (ST-920) for Fabry disease	Mean annualized eGFR slope of 1.965 at 52-weeks (32 patients)	Fabry disease affects over 10,000 patients in the U.S. alone.
Novel epigenetic regulation for chronic pain	ST-503 for Small Fiber Neuropathy (SFN)	Phase 1/2 STAND study enrollment in progress; first dosing expected in coming months.	SFN affects an estimated 53 people per 100,000 in the U.S.
Platform for CNS delivery	STAC-BBB AAV Capsid Platform	Leveraged in ST-506 (prion disease) program; CTA submission anticipated as early as mid-2026.	Potential for up to $1.4 billion in milestone payments from the Eli Lilly capsid licensing deal.
Addressing devastating neurological disorders	ST-506 for Prion Disease	Preclinical data showed profound survival extension in mouse models.	No approved disease-modifying therapies currently exist for prion disease.
Financial/Operational Milestones	General Pipeline Support	Received $6 million from Pfizer in October 2025 from a license buyout option.	Total potential future milestones from partners up to $4.6 billion.

The company's proprietary technology, including its zinc finger epigenetic regulators and the STAC-BBB capsid discovery platform, is the engine behind these propositions. This technology is positioned to address devastating neurological disorders where current options are inadequate or non-existent. For instance, the preclinical data for ST-506 showed widespread brain delivery in nonhuman primates, which is a key technical validation for the STAC-BBB platform's utility in the central nervous system.

The value is also quantified by the potential financial upside tied to these programs, even against the backdrop of a Q3 2025 net loss of $34.93 million and sales of only $0.581 million for that quarter. The market sees the value in the pipeline milestones, not the current burn rate. The total potential value from partnerships, including milestone payments and exercise fees, sits near $4.6 billion.

The core value proposition is built on delivering transformative genomic medicines for high unmet medical needs in rare and chronic diseases. You see this reflected in the regulatory advantages gained:

• Fabry Disease (ST-920): FDA agreement on eGFR slope endpoint for accelerated approval.
• Neuropathic Pain (ST-503): Receipt of Fast Track Designation.
• Prion Disease (ST-506): Alignment with the MHRA on CMC strategy ahead of CTA submission.

Sangamo Therapeutics, Inc. (SGMO) - Canvas Business Model: Customer Relationships

You're looking at how Sangamo Therapeutics, Inc. manages its key relationships, which are heavily weighted toward high-stakes, long-term scientific and regulatory partnerships, plus direct engagement with the rare disease communities they aim to serve. This isn't a high-volume consumer business; it's about deep, focused interaction.

High-touch, direct engagement with rare disease patient communities

Sangamo Therapeutics, Inc. focuses its direct engagement on patients afflicted with serious neurological diseases who currently lack adequate or any treatment options. This relationship starts early in development to ensure trial design meets patient needs. The commitment is evident in the data supporting isaralgagene civaparvovec (ST-920) for Fabry disease, presented at the International Congress of Inborn Errors of Metabolism 2025 (ICIEM2025) in Kyoto, Japan.

The patient-centric data from the registrational Phase 1/2 STAAR study shows the depth of this relationship:

Metric	Value	Context
Total Dosed Patients (STAAR Study)	32	Observed positive mean annualized eGFR slope at 52-weeks.
Patients with 104-Weeks Follow-up	19	Observed mean annualized eGFR slope of 1.747 mL/min/1.73m2/year.
Longest Follow-up on ST-920	At least 4.5 years	Observed in three patients as of Q3 2025, supporting durability.
Patients Withdrawn from ERT	All 18	Patients who started the study on Enzyme Replacement Therapy (ERT) were off ERT as of the Q3 2025 data cutoff.

The company is also advancing its Phase 1/2 STAND study for chronic neuropathic pain (iSFN), where patient enrollment and recruitment commenced following the activation of the first two clinical sites.

Intensive, long-term strategic alliances with large pharma partners

Strategic alliances are crucial for funding and commercial reach, forming the backbone of Sangamo Therapeutics, Inc.'s revenue outside of equity financing. These relationships often involve licensing their proprietary technology platforms, like the STAC-BBB capsid.

Here's a look at recent financial interactions with partners:

Partner	Transaction/Event	Financial Impact (2025)	Agreement Context
Pfizer Inc.	Exercise of buyout option for zinc finger modified cell lines license	Received $6 million in October 2025.	Pursuant to a 2008 license agreement.
Eli Lilly and Company (Lilly)	Capsid license agreement for CNS diseases	Received $18 million upfront fee for the first target.	Eligible for up to $1.4 billion in milestones plus tiered royalties.

Sangamo Therapeutics, Inc. continues business development negotiations for a potential commercialization partner for its Fabry disease program. The company's revenues for Q3 2025 were $0.6 million, a significant decrease from $49.4 million in Q3 2024, which the company attributed primarily to collaboration revenue recorded in the prior year period.

Regulatory body management (FDA, MHRA) for accelerated approval pathways

Managing relationships with the FDA and MHRA is a primary focus, especially for advancing ST-920 in Fabry disease and ST-506 in prion disease. You definitely want regulatory alignment when you're dealing with novel genomic medicines.

Key regulatory milestones as of late 2025 include:

• FDA reiterated October 2024 agreement on using eGFR slope as an endpoint for accelerated approval of ST-920.
• FDA accepted Sangamo Therapeutics, Inc.'s request for a rolling submission and review of the Biologics License Application (BLA) for ST-920.
• Sangamo plans to initiate the BLA rolling submission in the fourth quarter of 2025.
• A productive Type B meeting with the FDA in April 2025 set a clear CMC pathway for a planned BLA submission in the first quarter of 2026.
• Potential approval and commercial launch for ST-920 is targeted for the second half of 2026.
• ST-920 holds Orphan Drug, Fast Track, and RMAT designations from the FDA.
• The MHRA granted Innovative Licensing and Access Pathway designation to ST-920.
• The FDA granted Fast Track Designation to ST-503 in December 2025.

Investor relations and capital market communications (e.g., Q3 2025 earnings)

Investor communications center on capital preservation and pipeline progress, given the inherent need for substantial additional financing in the biotech sector. The Q3 2025 financial results were released before the market opened on Thursday, November 6, 2025.

Financial positioning communicated to investors:

• Consolidated net loss for Q3 2025 was $34.9 million, or $0.11 per share.
• Cash and cash equivalents stood at $29.6 million as of September 30, 2025, down from $41.9 million at December 31, 2024.
• The company raised approximately $21 million in net proceeds from an underwritten registered equity offering in Q1 2025.
• The current cash position, plus the $6 million from Pfizer in October 2025 and ATM proceeds, is projected to fund operations into the first quarter of 2026.
• Nasdaq granted a 180-day extension (until April 27, 2026) for compliance with the minimum bid price of $1.00.

Finance: draft 13-week cash view by Friday.

Sangamo Therapeutics, Inc. (SGMO) - Canvas Business Model: Channels

You're looking at how Sangamo Therapeutics, Inc. gets its value proposition-gene therapies-out to the market and partners. It's a mix of direct clinical execution and high-value strategic alliances, which is typical for a company at this stage.

Direct clinical trial sites for patient recruitment and dosing

For the neurology pipeline, Sangamo Therapeutics, Inc. is using direct clinical sites to get patients into their studies. This is the hands-on channel for generating the data needed for regulatory submissions. For the Phase 1/2 STAND study in chronic neuropathic pain, the company has activated its first clinical site and is moving fast to enroll.

• Phase 1/2 STAND study: Nine clinical sites selected to date.
• Dosing for ST-503 (neuropathic pain) is expected in fall of 2025.
• For the Fabry disease program (isaralgagene civaparvovec), the registrational STAAR study has 32 dosed patients as of the third quarter of 2025 data cutoff.
• The company is preparing for an anticipated Biologics License Application (BLA) submission for Fabry as early as the first quarter of 2026.

Out-licensing and collaboration agreements with global pharmaceutical companies

This is a major channel for Sangamo Therapeutics, Inc., turning their platform technology into immediate, non-dilutive revenue and validating their science with big pharma. They are actively licensing their STAC-BBB capsid technology, which is key for delivering medicines to the central nervous system.

Here's a quick look at the financial impact from some of these key out-licensing channels:

Collaboration Partner	Upfront Payment Received (Approx.)	Potential Total Value (Approx.)	Key Technology Licensed
Eli Lilly and Company	$18 million	Up to $1.4 billion	STAC-BBB capsid
Astellas Gene Therapies, Inc.	$20 million	Up to $1.3 billion	STAC-BBB capsid
Pfizer Inc. (Buyout Option Exercise)	$6 million	N/A (Option Exercise)	Zinc finger modified cell lines

Overall, Sangamo Therapeutics, Inc. raised over $100 million in 2024 alone through non-dilutive license fees and milestone payments. Plus, they received $6 million from Pfizer in October 2025 upon the exercise of a buyout option.

Scientific presentations at major medical conferences (e.g., ICIEM 2025)

Presenting data at top-tier medical meetings acts as a channel to inform the scientific community, potential partners, and regulators about clinical progress. Sangamo Therapeutics, Inc. used this channel to present their key Fabry data in late 2025.

• Presented detailed clinical data from the registrational STAAR study at the International Congress of Inborn Errors of Metabolism 2025 (ICIEM2025) in Kyoto, Japan, September 2-6, 2025.
• The Fabry data showed a positive mean annualized eGFR slope of 1.965 mL/min/1.73m2/year (95% CI: -0.153, 4.083) at 52-weeks across all 32 dosed patients.
• For the subset of 19 patients with longer follow-up, the mean annualized eGFR slope at Week 104 was 1.747 mL/min/1.73m2/year (95% CI: -0.106, 3.601).
• The company also planned to present updated nonclinical data at the 9th International Congress on Neuropathic Pain in Berlin, Germany.

Future commercial distribution network via a secured Fabry partner

The final channel, for their lead asset in Fabry disease, is currently in the negotiation stage. Sangamo Therapeutics, Inc. is actively engaging in business development discussions to secure a commercialization partner, which is the intended route for market distribution, rather than building out a full internal sales force right now.

• Sangamo Therapeutics, Inc. is continuing business development negotiations for a potential Fabry commercialization agreement.
• The company deferred Phase 3 planning until securing funding or a partner.
• A potential commercial launch for isaralgagene civaparvovec is targeted for as early as the second half of 2026, contingent on a successful BLA submission in Q1 2026 and partner alignment.

Finance: review Q4 2025 cash burn rate against the projected runway into Q1 2026.

Sangamo Therapeutics, Inc. (SGMO) - Canvas Business Model: Customer Segments

You're looking at the core groups Sangamo Therapeutics, Inc. (SGMO) targets with its genomic medicine platform, which is heavily focused on neurology as of late 2025. This isn't just about selling a drug; it's about partnering to deliver novel delivery systems and developing proprietary therapies for devastating conditions.

Patients with rare genetic diseases like Fabry disease

This segment is central to Sangamo Therapeutics, Inc.'s near-term commercialization strategy with its wholly owned investigational gene therapy, isaralgagene civaparvovec (ST-920). The goal here is to offer a one-time, durable treatment option for adults with Fabry disease.

The patient pool size is significant enough to warrant a focused approach; Fabry disease affects over 10,000 patients in the U.S. alone. The clinical data from the registrational STAAR study is intended to support an Accelerated Approval pathway, with a potential Biologics License Application (BLA) submission anticipated as early as Q1 2026.

Here are some key metrics from the patient data supporting this segment:

Clinical Endpoint/Metric	Data Point	Patient Group/Context
Mean Annualized eGFR Slope (52-weeks)	Positive	All dosed patients in STAAR study
Mean Annualized eGFR Slope (104-weeks)	1.747 mL/min/1.73m2/year	19 patients with 104-weeks follow-up
Mean Annualized eGFR Slope (1-year follow-up)	3.061 mL/min/1.73m2/year	23 patients with at least one-year follow-up (as of Feb 2025 data)
Patients Withdrawn from ERT	All 18 patients who began on ERT	Patients who have withdrawn and remain off ERT
Durable $\alpha$-Gal A Expression	Maintained for up to 4.5 years	Longest treated patient

Patients suffering from chronic neurological disorders (e.g., small fiber neuropathy)

Sangamo Therapeutics, Inc. is expanding into neurology, marking its transition to a clinical-stage neurology company with the initiation of the Phase 1/2 STAND study for chronic neuropathic pain (ST-503), which targets intractable pain due to idiopathic small fiber neuropathy (iSFN). This represents an entry into a substantial market, estimated at $10 billion.

The operational focus for this segment is moving quickly from site activation to patient dosing.

• Phase 1/2 STAND study sites activated: Two clinical sites as of November 2025.
• First patient dosing expected: In the fall of 2025 or 'coming months' as of November 2025.
• Preliminary proof of efficacy data anticipated: Q4 2026.

Also, Sangamo Therapeutics, Inc. is advancing CTA-enabling activities for its prion disease program (ST-506), with a Clinical Trial Application (CTA) submission expected in Q1 2026.

Major pharmaceutical and biotech companies seeking gene therapy technology

This segment is crucial for non-dilutive funding and validating Sangamo Therapeutics, Inc.'s proprietary technology, especially its novel neurotropic adeno-associated virus (AAV) capsid platform, STAC-BBB. These partners are interested in licensing the delivery technology or co-developing pipeline assets.

The financial impact of these collaborations is clear in the revenue figures:

• Lilly Agreement: Received an $18 million upfront license fee in April 2025 for the first target.
• Lilly Potential Value: Eligible to earn up to $1.4 billion in milestone payments plus royalties across up to five CNS targets.
• Pfizer Payment: Received $6 million in October 2025 from Pfizer Inc. for exercising a buyout option on certain zinc finger modified cell lines.
• Q1 2025 Revenue: Totaled $6.4 million, with $5.0 million attributed to the Pfizer sublicense transfer and $1.0 million from a Sigma-Aldrich license agreement.
• Total Collaborations: Sangamo Therapeutics, Inc. signed its third STAC-BBB license agreement (with Lilly).

The reliance on these deals is highlighted by the need for a Fabry commercialization agreement or other non-dilutive capital to secure runway beyond Q1 2026.

Specialist physicians and treatment centers for rare and neurological diseases

These are the clinical gatekeepers and prescribers who will ultimately use the therapies developed for the patient segments above. Sangamo Therapeutics, Inc. engages this group through data presentation at key medical congresses and by establishing clinical trial sites.

Engagement points for this segment in 2025 included:

• Presenting Fabry data at the 21st Annual WORLDSymposium on February 6, 2025.
• Presenting pipeline advances at the 28th ASGCT Annual Meeting on May 13-17, 2025.
• Presenting detailed Fabry data at the 15th ICIEM2025 on September 2-6, 2025.
• The STAND pain study has secured nine clinical sites.

Financially, the company's operating expenses reflect the costs of engaging this segment for clinical development. GAAP operating expenses for Q3 2025 were $36.1 million, with non-GAAP operating expenses at $33.0 million. Cash and cash equivalents as of September 30, 2025, were $29.6 million.

Sangamo Therapeutics, Inc. (SGMO) - Canvas Business Model: Cost Structure

You're looking at the core expenditures that fuel Sangamo Therapeutics, Inc.'s push to bring genomic medicines, particularly in neurology, to market. The cost structure is heavily weighted toward the science itself, which is typical for a clinical-stage biotech.

High research and development (R&D) expenses for clinical trials.

The R&D engine is the primary cost driver, reflecting the necessary investment in advancing the pipeline, especially isaralgagene civaparvovec (ST-920) and the STAND study for chronic neuropathic pain. For the six months ended June 30, 2025, GAAP R&D expenses were $53.10 million. This is down from $60.10 million for the same period in 2024, suggesting some initial cost optimization, though clinical trial costs remain substantial. The company is focused on a neurology-centric business, which dictates where these R&D dollars are allocated.

Here's a look at the operating expense components for the first half of 2025 versus 2024:

Expense Category (GAAP, in millions)	Six Months Ended June 30, 2025	Six Months Ended June 30, 2024
Research and development	$53.10	$60.10
General and administrative	$19.10	$23.80
Impairment of long-lived assets	$0.00	$5.50
Total operating expenses (Partial Year)	$72.20 (Calculated: $53.10 + $19.10)	$89.40 (Calculated: $60.10 + $23.80 + $5.50)

2025 GAAP total operating expenses guided between $135 million and $155 million.

Sangamo Therapeutics, Inc. has guided its full-year 2025 GAAP total operating expenses to be in the range of $135 million to $155 million. To give you a clearer picture of the underlying operational spend, the company also provides a non-GAAP view, which excludes certain non-cash or non-recurring items. The non-GAAP projection for 2025 is between $125 million and $145 million. These figures include estimates for non-cash stock-based compensation of about $7 million and depreciation and amortization costs of roughly $3 million.

Manufacturing and BLA readiness costs for ST-920.

Advancing ST-920 for Fabry disease toward a potential Biologics License Application (BLA) submission, targeted as early as the first quarter of 2026, necessitates specific spending. You see this reflected as an increase in clinical and manufacturing expenses, which partially offset overall expense decreases in the third quarter of 2025. This spending is critical for Chemistry, Manufacturing and Controls (CMC) readiness required by the FDA for accelerated approval.

General and administrative costs for a lean, neurology-focused business.

The G&A component reflects the cost of running the business, including executive, finance, and legal functions, while supporting the neurology focus. For the first six months of 2025, GAAP G&A expenses were $19.10 million. This is a reduction from $23.80 million in the first half of 2024, aligning with the stated intention to operate a lean business model. The company is actively pursuing cost-saving measures to maximize efficiency.

Intellectual property maintenance and licensing fees.

Costs associated with protecting the company's innovations, such as patent maintenance and fees related to existing licensing agreements, are part of the structure. In the third quarter of 2025, lower licensing and patent-related expenses were noted as a factor contributing to the year-over-year decrease in GAAP operating expenses. Furthermore, the company secured a $6 million license fee from Pfizer in October 2025 from a 2008 agreement, which helps offset burn rate, though this is revenue, not an expense item.

The cash position as of September 30, 2025, was $29.6 million, which, combined with expected proceeds, is projected to fund operations into the first quarter of 2026. Finance: draft 13-week cash view by Friday.

Sangamo Therapeutics, Inc. (SGMO) - Canvas Business Model: Revenue Streams

You're looking at how Sangamo Therapeutics, Inc. (SGMO) brings in cash, which is heavily weighted toward non-product revenue streams at this stage. It's all about partnerships and development milestones right now, which is typical for a company deep in the development pipeline.

Upfront license payments form a key component of near-term recognized revenue. For instance, Sangamo Therapeutics received an $18 million upfront license fee from Eli Lilly in April 2025 for the first target under their STAC-BBB capsid license agreement. Looking at the cumulative picture as of the third quarter of 2025, the company has received approximately $910.0 million in total from upfront licensing fees, milestone payments, and proceeds from the sale of common stock to collaborators.

The real potential upside, however, sits in the milestone payments tied to their existing collaborations. Sangamo Therapeutics has the opportunity to earn up to $5.9 billion in potential future milestone payments from ongoing collaborations, in addition to product royalties. This potential is derived from several key agreements, which you can see broken down below.

Collaboration Partner	Potential Future Milestone Payments
Eli Lilly and Company	Up to $1.4 billion across five CNS targets
Astellas Gene Therapies, Inc.	Up to $1.3 billion across five neurological disease targets
Genentech, Inc.	Up to $1.9 billion
Total Potential Milestones	Up to $4.6 billion

It's important to note that the total potential milestone figure of $4.6 billion is the sum of the individual potential maximums from these key partners. Still, the actual realization of these funds depends entirely on the partners successfully completing clinical development and achieving regulatory and commercial success, so there's defintely risk involved.

Another source of non-dilutive cash flow comes from the exercise of buyout options by partners. In October 2025, Sangamo Therapeutics received $6.0 million from Pfizer Inc. when Pfizer exercised a buyout option for a license to use certain zinc finger modified cell lines under a 2008 agreement. This is a clean, one-time cash infusion.

To bridge operational gaps and fund the pipeline, Sangamo Therapeutics also relies on equity financing. The company announced the pricing of a $23 million underwritten registered direct equity offering in May 2025, which was intended to extend the cash runway into the third quarter of 2025. Separately, the company also raised approximately $21 million in net proceeds from an underwritten registered equity offering reported in August 2025.

The final, long-term revenue component involves future royalties. Sangamo Therapeutics is eligible to earn tiered royalties on net product sales from several partnered programs, including those with Eli Lilly, Astellas, and Genentech. These royalties represent the potential for passive income streams once any of the partnered products actually reach the market.

• Tiered royalties on net product sales from partnered programs.
• Potential milestone payments up to $4.6 billion total from existing collaborations.
• Upfront fee of $18 million received from Eli Lilly in Q1 2025.
• Buyout fee of $6.0 million received from Pfizer in October 2025.
• Proceeds from equity offerings, including a $23 million gross proceeds offering in Q1 2025.

Finance: draft 13-week cash view by Friday.