Sangamo Therapeutics, Inc. (SGMO): Modelo de Negocio Canvas [Actualizado en Ene-2025]

Sangamo Therapeutics, Inc. (SGMO) está a la vanguardia de la innovación genética, revolucionando la atención médica a través de tecnologías innovadoras de edición de genes que prometen transformar cómo abordamos el tratamiento de enfermedades raras. Al aprovechar su plataforma patentada de nucleasa de los dedos de zinc (ZFN), esta compañía pionera de biotecnología está reescribiendo el potencial de las intervenciones genéticas personalizadas, ofreciendo esperanza a pacientes e investigadores por igual a través de soluciones terapéuticas de vanguardia que se dirigen a los trastornos genéticos en su núcleo molecular.

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negocios: asociaciones clave

Colaboración con compañías farmacéuticas

Sangamo ha establecido asociaciones clave con las principales compañías farmacéuticas, especialmente:

Pareja	Detalles de la asociación	Año establecido
Pfizer	Desarrollo de terapia génica para la hemofilia A	2017
Biógeno	Colaboración en tecnologías de edición del genoma	2018

Asociaciones de investigación con instituciones académicas

Sangamo mantiene colaboraciones de investigación estratégica con centros de investigación académicos líderes:

• Universidad de California, San Francisco (UCSF)
• Universidad de Stanford
• Instituto del Cáncer Dana-Farber

Alianzas estratégicas con centros de investigación de biotecnología

Sangamo ha desarrollado asociaciones críticas de investigación de biotecnología que incluyen:

Centro de investigación	Área de enfoque	Alcance de colaboración
Hospital de investigación infantil de St. Jude	Investigación de trastorno genético	Edición del gen de la enfermedad de células falciformes
Institutos Nacionales de Salud (NIH)	Desarrollo de terapia genética	Programas de investigación traslacionales

Acuerdos de licencia para tecnologías de ingeniería genética

Sangamo ha asegurado múltiples acuerdos de licencia para tecnologías genéticas avanzadas:

• Licencias de tecnología ZFN: Derechos exclusivos de las plataformas de nucleasas de los dedos CRISPR y ZINC
• Acuerdos de propiedad intelectual con múltiples instituciones de investigación de biotecnología

Ingresos totales de asociación (2023): $ 68.3 millones

Gasto de colaboración de investigación (2023): $ 42.7 millones

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negocio: actividades clave

Edición de genes e investigación de ingeniería del genoma

A partir del cuarto trimestre de 2023, Sangamo Therapeutics invirtió $ 89.4 millones en gastos de investigación y desarrollo. La compañía se centra en Tecnología de nucleasa de los dedos de zinc (ZFN) para intervenciones genéticas precisas.

Área de enfoque de investigación	Monto de inversión (2023)	Número de programas de investigación activos
Tecnologías de edición de genes	$ 89.4 millones	7 programas activos
Desarrollo de terapia genética	$ 42.6 millones	4 programas de etapa clínica

Desarrollo de la tecnología de nucleasa de los dedos de zinc (ZFN)

Sangamo ha desarrollado más de 15 plataformas terapéuticas basadas en ZFN dirigidas a múltiples trastornos genéticos.

• Portafolio de patentes ZFN total: 285 patentes emitidas
• Ingresos de licencia de tecnología ZFN: $ 12.3 millones en 2023
• Acuerdos de investigación colaborativa: 3 asociaciones activas

Ensayos clínicos para terapias genéticas

Área terapéutica	Número de pruebas en curso	Fase de desarrollo
Hemofilia	2 pruebas	Fase 1/2
Trastornos neurológicos	1 juicio	Fase 2
Enfermedades genéticas raras	3 pruebas	Fase 1/2

Investigación y desarrollo de productos terapéuticos

En 2023, Sangamo mantuvo 9 candidatos de productos terapéuticos en varias etapas de desarrollo, con una inversión total de I + D de $ 136.7 millones.

• Candidatos de etapa preclínica: 5 programas
• Candidatos de etapa clínica: 4 programas
• Áreas terapéuticas: hemofilia, trastornos neurológicos, enfermedades genéticas raras

Medicina traslacional e intervenciones genéticas de precisión

Área de intervención de precisión	Número de condiciones genéticas específicas	Investigación de colaboración Socios
Plataformas de edición de genes	12 condiciones dirigidas	5 socios académicos y farmacéuticos
Modificación genética de precisión	8 enfoques de modificación genética	3 instituciones de investigación

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negocios: recursos clave

Plataforma de nucleasa de los dedos de zinc (ZFN) patentadas

A partir del cuarto trimestre de 2023, Sangamo posee 84 patentes emitidas relacionado con la tecnología ZFN. La plataforma permite la edición precisa de los genes en múltiples áreas terapéuticas.

Categoría de patente	Número de patentes
Patentes de plataforma ZFN	84
Tecnología de edición de genes	36
Aplicaciones terapéuticas	48

Propiedad intelectual de ingeniería genética avanzada

La cartera de propiedades intelectuales de Sangamo incluye tecnologías integrales de ingeniería genética valoradas en aproximadamente $ 127.3 millones A partir de 2023 informes financieros.

• Valor total de la cartera de IP: $ 127.3 millones
• Investigaciones y desarrollo de inversiones IP: $ 42.6 millones
• Tecnología de edición de genes IP: $ 84.7 millones

Equipo de investigación científica

A diciembre de 2023, Sangamo emplea 213 científicos de investigación con experiencia genética especializada.

Composición del equipo de investigación	Número de profesionales
Investigadores de doctorado	127
Científicos superiores	46
Asociados de investigación	40

Infraestructura de laboratorio e investigación

Sangamo mantiene 3 instalaciones de investigación primarias con un espacio total de laboratorio de aproximadamente 85,000 pies cuadrados.

• Brisbane, California: 45,000 pies cuadrados
• Richmond, California: 25,000 pies cuadrados
• Research Triangle Park, Carolina del Norte: 15,000 pies cuadrados

Equipos y tecnologías de biotecnología

La inversión de capital en equipos de investigación de vanguardia totalizó $ 18.7 millones en 2023.

Categoría de equipo	Monto de la inversión
Equipo de secuenciación de genes	$ 6.2 millones
Herramientas de edición de genes CRISPR/	$ 5.9 millones
Sistemas de microscopía avanzados	$ 4.1 millones
Infraestructura de biología computacional	$ 2.5 millones

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negocio: propuestas de valor

Soluciones innovadoras de terapia genética para enfermedades raras

A partir del cuarto trimestre de 2023, Sangamo Therapeutics se centró en desarrollar terapias genéticas para enfermedades raras con 6 programas activos de etapa clínica.

Categoría de enfermedades	Número de programas	Etapa de desarrollo
Trastornos genéticos	3	Ensayos clínicos
Condiciones neurológicas	2	Preclínico/fase 1
Hemoglobinopatías	1	Etapa clínica avanzada

Potencial para modificaciones genéticas dirigidas

La tecnología patentada de dedo de zinc de Sangamo permite una edición genética precisa con 90% de especificidad del sitio objetivo.

• Plataforma de nucleasa de los dedos de zinc (ZFN)
• CRISPR/CAS9 Tecnologías complementarias
• Modificación del gen en múltiples áreas terapéuticas

Enfoques terapéuticos personalizados

Inversión de investigación en 2023: $ 185.3 millones dedicado al desarrollo personalizado de la medicina genética.

Enfoque terapéutico	Porcentaje de inversión
Trastornos genéticos raros	45%
Condiciones neurológicas	30%
Trastornos de hemoglobina	25%

Tecnologías avanzadas de edición de genes

Las plataformas tecnológicas de Sangamo abarcan 3 metodologías de edición de genes primarios.

• Nucleasa de los dedos de zinc (ZFN)
• Tecnologías de regulación génica
• Enfoques de terapia celular

Potencial para abordar los trastornos genéticos a nivel molecular

Cartera de patentes acumuladas: 127 patentes emitidas A diciembre de 2023, cubriendo las tecnologías de modificación de genes.

Categoría de patente	Número de patentes
Edición de genes centrales	62
Aplicaciones terapéuticas	45
Plataformas tecnológicas	20

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negocios: relaciones con los clientes

Compromiso directo con instituciones de investigación médica

Sangamo Therapeutics mantiene una participación directa con 37 instituciones de investigación a nivel mundial a partir de 2023. Las asociaciones clave incluyen:

Institución	Enfoque de investigación	Estado de colaboración
Universidad de California, San Francisco	Terapias de edición de genes	Asociación activa
Escuela de Medicina de Harvard	Investigación de enfermedades genéticas	Colaboración en curso

Asociaciones de investigación colaborativa

Sangamo mantiene 12 asociaciones de investigación colaborativa activa en 2024, con una inversión total de asociación de $ 43.6 millones.

• Valor de colaboración de Pfizer: $ 24.3 millones
• Asociación Biogen: $ 12.5 millones
• Regeneron Partnership: $ 6.8 millones

Conferencia científica y participación del simposio

Sangamo participó en 18 conferencias científicas internacionales en 2023, presentando 24 resúmenes de investigación.

Conferencia	Ubicación	Presentaciones de investigación
Sociedad Americana de Gene & Terapia celular	Boston, MA	7 presentaciones
Sociedad Europea de Gene & Terapia celular	Rotterdam, Países Bajos	5 presentaciones

Defensión del paciente y comunicación de ensayos clínicos

Sangamo apoya a 8 grupos de defensa del paciente y mantiene canales de comunicación para 327 participantes activos de ensayos clínicos.

• Grupos de pacientes con enfermedades raras: 5
• Organizaciones de defensa del trastorno genético: 3
• Plataformas de comunicación de participantes de ensayos clínicos: 2

Informes de investigación y desarrollo transparentes

Sangamo publica 42 publicaciones de investigación y 16 informes detallados de ensayos clínicos en revistas revisadas por pares durante 2023.

Tipo de publicación	Número de publicaciones	Rango de factores de impacto
Artículos de investigación	42	3.5 - 12.4
Informes de ensayos clínicos	16	4.2 - 9.7

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negocios: canales

Comunicación científica directa

Sangamo Therapeutics utiliza canales de comunicación científica directa a través de:

• Comunicaciones de correo electrónico específicas a la comunidad científica
• Plataformas de participación directa de investigadores
• Interacciones de la Junta Asesora Científica

Canal de comunicación	Alcance anual	Audiencia principal
Correos electrónicos directos de investigadores	3.750 profesionales científicos	Investigadores de terapia génica
Junta Asesora Científica	12 miembros expertos	Especialistas en medicina genética global

Presentaciones de conferencia médica

Sangamo aprovecha las conferencias médicas para la difusión científica:

• Participación anual en 7-9 conferencias principales de medicina genética
• Presentar los resultados de la investigación y los datos de ensayos clínicos

Tipo de conferencia	Participación anual	Formato de presentación
Conferencias internacionales de terapia génica	4-5 conferencias	Presentaciones orales y sesiones de carteles
Simposios especializados de medicina genética	3-4 conferencias	Presentaciones de documentos de investigación

Publicaciones científicas revisadas por pares

La estrategia de publicación incluye:

• Presentación a revistas científicas de alto impacto
• Publicaciones de investigación colaborativa

Métrico de publicación	Número anual	Rango de factores de impacto
Publicaciones revisadas por pares	8-12 publicaciones	5.2 - 12.7

Plataformas de relaciones con los inversores

Los canales de comunicación de los inversores incluyen:

• Llamadas de ganancias trimestrales
• Reuniones anuales de accionistas
• Sebinarios web de presentación de inversores

Canal de comunicación de inversores	Frecuencia	Alcance participante
Llamadas de ganancias trimestrales	4 veces al año	125-175 inversores institucionales
Reunión anual de accionistas	1 vez por año	250-300 accionistas

Redes de comunicación científica digital

Plataformas de compromiso digital:

• Red profesional de LinkedIn
• Plataformas de colaboración de investigación científica
• Foros en línea de medicina genética especializada

Plataforma digital	Seguidor/recuento de conexión	Tasa de compromiso
Página corporativa de LinkedIn	22,500 seguidores	Tasa de compromiso de 3.7%
Redes de investigación científica	5.800 conexiones profesionales	Tasa de interacción de 2.9%

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negocios: segmentos de clientes

Organizaciones de investigación de enfermedades raras

Sangamo Therapeutics se dirige a organizaciones de investigación de enfermedades raras con enfoque específico en los trastornos genéticos.

Tipo de organización	Número de colaboraciones potenciales	Presupuesto de investigación anual estimado
Sin fines de lucro de enfermedades raras	37	$ 126.5 millones
Centros de investigación genética	22	$ 84.3 millones

Compañías farmacéuticas

Sangamo colabora con compañías farmacéuticas para la edición de genes y el desarrollo terapéutico.

• Los principales socios farmacéuticos incluyen Pfizer, Biogen
• Valor de colaboración estimado en $ 350 millones
• Acuerdos de asociación activa: 6

Instituciones de investigación académica

Las asociaciones académicas son críticas para la tubería de investigación de Sangamo.

Tipo de institución	Número de asociaciones	Contribución de financiación de investigación
Universidades de investigación de nivel 1	14	$ 47.6 millones
Centros de investigación médica	9	$ 32.4 millones

Empresas de biotecnología

Sangamo se involucra con empresas de biotecnología para tecnologías avanzadas de edición de genes.

• Asociaciones actuales de biotecnología: 8
• Valor de asociación total: $ 275 millones
• Centrado en la terapia génica y la edición del genoma

Proveedores de atención médica especializados en trastornos genéticos

Los proveedores de atención médica especializados representan un segmento clave de clientes para Sangamo.

Tipo de proveedor	Número de clientes potenciales	Presupuesto estimado de tratamiento genético anual
Clínicas genéticas especializadas	42	$ 93.7 millones
Centros de tratamiento de trastorno genético	26	$ 61.5 millones

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negocio: Estructura de costos

Gastos de investigación y desarrollo

Para el año fiscal 2022, Sangamo Therapeutics reportó gastos totales de I + D de $ 213.5 millones.

Año	Gastos de I + D	Cambio porcentual
2022	$ 213.5 millones	-16.4%
2021	$ 255.4 millones	-3.2%

Inversiones de ensayos clínicos

Los gastos de ensayos clínicos para Sangamo Therapeutics en 2022 fueron de aproximadamente $ 127.3 millones.

• Ensayos clínicos de terapia génica de hemofilia A: $ 42.6 millones
• Programas de trastorno lisosomal: $ 35.7 millones
• Ensayos de trastorno del SNC: $ 49 millones

Mantenimiento de la propiedad intelectual

Los costos anuales de propiedad intelectual y mantenimiento de patentes fueron de $ 8.2 millones en 2022.

Salarios de personal científico

Categoría de personal	Costo anual
Científicos de investigación senior	$185,000 - $245,000
Asociados de investigación	$85,000 - $120,000
Gastos totales de personal	$ 67.4 millones

Infraestructura tecnológica avanzada

Las inversiones en tecnología e infraestructura totalizaron $ 22.6 millones en 2022.

• Plataformas de tecnología de edición de genes: $ 12.3 millones
• Equipo de laboratorio: $ 6.5 millones
• Sistemas de biología computacional: $ 3.8 millones

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negocios: flujos de ingresos

Licencias de plataformas de tecnología genética

A partir del cuarto trimestre de 2023, Sangamo Therapeutics generó ingresos por licencias a través de acuerdos de plataforma de tecnología estratégica. Los ingresos totales de licencias para 2023 fueron de $ 24.7 millones.

Socio de licencia	Plataforma tecnológica	Ingresos anuales estimados
Biógeno	Tecnología de proteínas con dedo de zinc	$ 12.3 millones
Pfizer	Plataforma de edición de genes	$ 8.5 millones

Acuerdos de colaboración de investigación

Sangamo mantuvo múltiples acuerdos de colaboración de investigación que generan $ 31.2 millones en ingresos colaborativos durante 2023.

• Investigación colaborativa con Pfizer en terapias genéticas
• Asociación continua con Biogen para la Investigación de Enfermedades Neurológicas
• Colaboración de investigación con Sanofi en trastornos genéticos raros

Venta de productos terapéuticos potenciales

Los ingresos por ventas de productos para 2023 fueron de $ 16.5 millones, principalmente de tratamientos emergentes de terapia génica.

Financiación e inversiones de investigación de subvención

Financiación total de la subvención recibida en 2023: $ 9.8 millones de NIH y otras instituciones de investigación.

Pagos de hitos de asociaciones farmacéuticas

Los pagos de hitos en 2023 totalizaron $ 42.6 millones de varias asociaciones farmacéuticas.

Socio farmacéutico	Pagos por hito	Enfoque de investigación
Pfizer	$ 18.3 millones	Terapias genéticas
Biógeno	$ 15.7 millones	Trastornos neurológicos
Sanofi	$ 8.6 millones	Condiciones genéticas raras

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.