Sangamo Therapeutics, Inc. (SGMO): Modelo de Negócios Canvas [Jan-2025 Atualizado]

A Sangamo Therapeutics, Inc. (SGMO) fica na vanguarda da inovação genética, revolucionando a assistência médica através de tecnologias inovadoras de edição de genes que prometem transformar a maneira como abordamos o tratamento de doenças raras. Ao alavancar sua plataforma proprietária de nuclease de dedos de zinco (ZFN), esta empresa pioneira de biotecnologia está reescrevendo o potencial de intervenções genéticas personalizadas, oferecendo esperança a pacientes e pesquisadores por meio de soluções terapêuticas de ponta que visam distúrbios genéticos em seu núcleo molecular.

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negócios: Parcerias -chave

Colaboração com empresas farmacêuticas

A Sangamo estabeleceu as principais parcerias com as principais empresas farmacêuticas, principalmente:

Parceiro	Detalhes da parceria	Ano estabelecido
Pfizer	Desenvolvimento da terapia genética para hemofilia A	2017
Biogênio	Colaboração em tecnologias de edição de genoma	2018

Parcerias de pesquisa com instituições acadêmicas

Sangamo mantém colaborações estratégicas de pesquisa com os principais centros de pesquisa acadêmica:

• Universidade da Califórnia, São Francisco (UCSF)
• Universidade de Stanford
• Instituto de Câncer Dana-Farber

Alianças estratégicas com centros de pesquisa de biotecnologia

Sangamo desenvolveu parcerias críticas de pesquisa de biotecnologia, incluindo:

Centro de Pesquisa	Área de foco	Escopo de colaboração
Hospital de Pesquisa Infantil de St. Jude	Pesquisa de Transtorno Genético	Edição de genes da doença de células falciformes
Institutos Nacionais de Saúde (NIH)	Desenvolvimento da terapia genética	Programas de pesquisa translacional

Acordos de licenciamento para tecnologias de engenharia genética

Sangamo garantiu vários acordos de licenciamento para tecnologias genéticas avançadas:

• Licenciamento de tecnologia ZFN: Direitos exclusivos das plataformas CRISPR e de nuclease de dedos de zinco
• Acordos de propriedade intelectual com várias instituições de pesquisa de biotecnologia

Receita total de parceria (2023): US $ 68,3 milhões

Despesas de colaboração de pesquisa (2023): US $ 42,7 milhões

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negócios: Atividades -chave

Edição de genes e pesquisa de engenharia de genoma

A partir do quarto trimestre de 2023, a Sangamo Therapeutics investiu US $ 89,4 milhões em despesas de pesquisa e desenvolvimento. A empresa se concentra em Tecnologia de nuclease de dedos de zinco (ZFN) Para intervenções genéticas precisas.

Área de foco de pesquisa	Valor do investimento (2023)	Número de programas de pesquisa ativos
Tecnologias de edição de genes	US $ 89,4 milhões	7 programas ativos
Desenvolvimento da terapia genética	US $ 42,6 milhões	4 programas de estágio clínico

Desenvolvimento da tecnologia de nuclease de dedos de zinco (ZFN)

A Sangamo desenvolveu mais de 15 plataformas terapêuticas baseadas em ZFN direcionadas a vários distúrbios genéticos.

• Portfólio total de patentes ZFN: 285 patentes emitidas
• Receita de licenciamento de tecnologia ZFN: US $ 12,3 milhões em 2023
• Acordos de pesquisa colaborativa: 3 parcerias ativas

Ensaios clínicos para terapias genéticas

Área terapêutica	Número de ensaios em andamento	Fase de desenvolvimento
Hemofilia	2 ensaios	Fase 1/2
Distúrbios neurológicos	1 Trial	Fase 2
Doenças genéticas raras	3 ensaios	Fase 1/2

Pesquisa e Desenvolvimento de Produtos Terapêuticos

Em 2023, Sangamo manteve 9 candidatos a produtos terapêuticos em vários estágios de desenvolvimento, com um investimento total em P&D de US $ 136,7 milhões.

• Candidatos de estágio pré -clínico: 5 programas
• Candidatos em estágio clínico: 4 programas
• Áreas terapêuticas: hemofilia, distúrbios neurológicos, doenças genéticas raras

Medicina translacional e intervenções genéticas de precisão

Área de intervenção de precisão	Número de condições genéticas direcionadas	Parceiros de colaboração de pesquisa
Plataformas de edição de genes	12 condições direcionadas	5 parceiros acadêmicos e farmacêuticos
Modificação genética de precisão	8 abordagens de modificação genética	3 instituições de pesquisa

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negócios: Recursos -chave

Plataforma proprietária de nuclease de dedos de zinco (ZFN)

A partir do quarto trimestre 2023, Sangamo segura 84 patentes emitidas Relacionado à tecnologia ZFN. A plataforma permite edição precisa de genes em várias áreas terapêuticas.

Categoria de patentes	Número de patentes
Patentes da plataforma ZFN	84
Tecnologia de edição de genes	36
Aplicações terapêuticas	48

Propriedade intelectual de engenharia genética avançada

O portfólio de propriedade intelectual de Sangamo inclui tecnologias abrangentes de engenharia genética avaliadas em aproximadamente US $ 127,3 milhões a partir de 2023 relatórios financeiros.

• Valor total do portfólio IP: US $ 127,3 milhões
• Pesquisa e desenvolvimento Investimentos de IP: US $ 42,6 milhões
• Tecnologia de edição de genes IP: US $ 84,7 milhões

Equipe de pesquisa científica

Em dezembro de 2023, Sangamo emprega 213 cientistas de pesquisa com experiência genética especializada.

Composição da equipe de pesquisa	Número de profissionais
Pesquisadores de doutorado	127
Cientistas seniores	46
Associados de pesquisa	40

Infraestrutura de laboratório e pesquisa

Sangamo mantém 3 instalações de pesquisa primárias com espaço total de laboratório de aproximadamente 85.000 pés quadrados.

• Brisbane, Califórnia: 45.000 pés quadrados
• Richmond, Califórnia: 25.000 pés quadrados
• Pesquisa Triangle Park, Carolina do Norte: 15.000 pés quadrados

Equipamentos e tecnologias de biotecnologia

Investimento de capital em equipamentos de pesquisa de ponta totalizados US $ 18,7 milhões em 2023.

Categoria de equipamento	Valor do investimento
Equipamento de sequenciamento de genes	US $ 6,2 milhões
Ferramentas de edição do CRISPR/genes	US $ 5,9 milhões
Sistemas de microscopia avançada	US $ 4,1 milhões
Infraestrutura de biologia computacional	US $ 2,5 milhões

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negócios: proposições de valor

Soluções inovadoras de terapia genética para doenças raras

A partir do quarto trimestre 2023, a terapêutica de Sangamo se concentrou no desenvolvimento de terapias genéticas para doenças raras com 6 programas ativos em estágio clínico.

Categoria de doença	Número de programas	Estágio de desenvolvimento
Distúrbios genéticos	3	Ensaios clínicos
Condições neurológicas	2	Pré -clínico/Fase 1
Hemoglobinopatias	1	Estágio clínico avançado

Potencial para modificações genéticas direcionadas

A tecnologia proprietária de dedos de zinco de Sangamo permite edição genética precisa com 90% de especificidade do local -alvo.

• Plataforma de nuclease de dedos de zinco (ZFN)
• Tecnologias complementares CRISPR/CAS9
• Modificação de genes em várias áreas terapêuticas

Abordagens terapêuticas personalizadas

Investimento de pesquisa em 2023: US $ 185,3 milhões dedicado ao desenvolvimento personalizado da medicina genética.

Foco terapêutico	Porcentagem de investimento
Distúrbios genéticos raros	45%
Condições neurológicas	30%
Distúrbios da hemoglobina	25%

Tecnologias avançadas de edição de genes

As plataformas de tecnologia de Sangamo abrangem 3 metodologias primárias de edição de genes.

• Nuclease de dedos de zinco (ZFN)
• Tecnologias de regulação de genes
• Abordagens de terapia celular

Potencial para abordar os distúrbios genéticos em nível molecular

Portfólio cumulativo de patentes: 127 patentes emitidas Em dezembro de 2023, abrangendo tecnologias de modificação de genes.

Categoria de patentes	Número de patentes
Edição de genes central	62
Aplicações terapêuticas	45
Plataformas tecnológicas	20

Sangamo Therapeutics, Inc. (SGMO) - Modelo de Negócios: Relacionamentos do Cliente

Engajamento direto com instituições de pesquisa médica

A Sangamo Therapeutics mantém o envolvimento direto com 37 instituições de pesquisa globalmente a partir de 2023. As parcerias -chave incluem:

Instituição	Foco na pesquisa	Status de colaboração
Universidade da Califórnia, São Francisco	Terapias de edição de genes	Parceria ativa
Escola de Medicina de Harvard	Pesquisa de doenças genéticas	Colaboração em andamento

Parcerias de pesquisa colaborativa

A Sangamo mantém 12 parcerias de pesquisa colaborativa ativa em 2024, com investimento total em parceria de US $ 43,6 milhões.

• Valor da colaboração da Pfizer: US $ 24,3 milhões
• Parceria Biogen: US $ 12,5 milhões
• Regeneron Partnership: US $ 6,8 milhões

Conferência Científica e Participação do Simpósio

Sangamo participou de 18 conferências científicas internacionais em 2023, apresentando 24 resumos de pesquisa.

Conferência	Localização	Apresentações de pesquisa
Sociedade Americana de Gene & Terapia celular	Boston, MA	7 apresentações
Sociedade Europeia de Gene & Terapia celular	Roterdã, Holanda	5 apresentações

Advocacia do paciente e comunicação de ensaios clínicos

A Sangamo suporta 8 grupos de defesa de pacientes e mantém canais de comunicação para 327 participantes ativos de ensaios clínicos.

• Grupos de pacientes com doenças raras: 5
• Organizações de defesa do distúrbio genético: 3
• Plataformas de comunicação de participantes do ensaio clínico: 2

Relatórios transparentes de pesquisa e desenvolvimento

Sangamo publica 42 publicações de pesquisa e 16 relatórios detalhados de ensaios clínicos em periódicos revisados ​​por pares durante 2023.

Tipo de publicação	Número de publicações	Faixa de fatores de impacto
Artigos de pesquisa	42	3.5 - 12.4
Relatórios de ensaios clínicos	16	4.2 - 9.7

Sangamo Therapeutics, Inc. (SGMO) - Modelo de Negócios: Canais

Comunicação científica direta

A Sangamo Therapeutics utiliza canais diretos de comunicação científica através de:

• Comunicações de e -mail direcionadas para a comunidade científica
• Plataformas diretas de engajamento do pesquisador
• Interações do Conselho Consultivo Científico

Canal de comunicação	Alcance anual	Público principal
E -mails diretos do pesquisador	3.750 profissionais científicos	Pesquisadores de terapia genética
Conselho Consultivo Científico	12 membros especializados	Especialistas em medicina genética global

Apresentações da conferência médica

Sangamo aproveita conferências médicas para disseminação científica:

• Participação anual em 7-9 principais conferências de medicina genética
• Apresentando resultados de pesquisa e dados de ensaios clínicos

Tipo de conferência	Participação anual	Formato de apresentação
Conferências de terapia genética internacional	4-5 Conferências	Apresentações orais e sessões de pôsteres
Simpósios de Medicina Genética Especializada	3-4 Conferências	Apresentações em papel de pesquisa

Publicações científicas revisadas por pares

A estratégia de publicação inclui:

• Submissão a periódicos científicos de alto impacto
• Publicações de pesquisa colaborativa

Métrica de publicação	Número anual	Faixa de fatores de impacto
Publicações revisadas por pares	8-12 publicações	5.2 - 12.7

Plataformas de relações com investidores

Os canais de comunicação dos investidores incluem:

• Chamadas de ganhos trimestrais
• Reuniões anuais de acionistas
• Webinars de apresentação de investidores

Canal de comunicação do investidor	Freqüência	Alcance dos participantes
Chamadas de ganhos trimestrais	4 vezes por ano	125-175 Investidores institucionais
Reunião Anual dos Acionistas	1 tempo por ano	250-300 acionistas

Redes de comunicação científica digital

Plataformas de engajamento digital:

• Rede Profissional do LinkedIn
• Plataformas de colaboração de pesquisa científica
• Fóruns online de medicina genética especializados

Plataforma digital	Contagem de seguidores/conexão	Taxa de engajamento
Página corporativa do LinkedIn	22.500 seguidores	Taxa de engajamento de 3,7%
Redes de pesquisa científica	5.800 conexões profissionais	Taxa de interação de 2,9%

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

Organizações de pesquisa de doenças raras

A Sangamo Therapeutics tem como alvo organizações de pesquisa de doenças raras com foco específico em distúrbios genéticos.

Tipo de organização	Número de possíveis colaborações	Orçamento de pesquisa anual estimado
Doenças raras organizações sem fins lucrativos	37	US $ 126,5 milhões
Centros de Pesquisa Genética	22	US $ 84,3 milhões

Empresas farmacêuticas

A Sangamo colabora com empresas farmacêuticas para edição de genes e desenvolvimento terapêutico.

• Os principais parceiros farmacêuticos incluem Pfizer, Biogen
• Valor de colaboração estimado em US $ 350 milhões
• Acordos de parceria ativa: 6

Instituições de pesquisa acadêmica

As parcerias acadêmicas são críticas para o pipeline de pesquisa de Sangamo.

Tipo de instituição	Número de parcerias	Pesquise contribuição de financiamento
Universidades de pesquisa de nível 1	14	US $ 47,6 milhões
Centros de Pesquisa Médica	9	US $ 32,4 milhões

Empresas de biotecnologia

Sangamo se envolve com empresas de biotecnologia para tecnologias avançadas de edição de genes.

• Parcerias de biotecnologia atuais: 8
• Valor total da parceria: US $ 275 milhões
• Focado na terapia genética e na edição do genoma

Provedores de saúde especializados em distúrbios genéticos

Os prestadores de serviços de saúde especializados representam um segmento importante de clientes para Sangamo.

Tipo de provedor	Número de clientes em potencial	Orçamento anual de tratamento genético estimado
Clínicas genéticas especializadas	42	US $ 93,7 milhões
Centros de Tratamento de Transtornos Genéticos	26	US $ 61,5 milhões

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negócios: estrutura de custos

Despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2022, a Sangamo Therapeutics registrou despesas totais de P&D de US $ 213,5 milhões.

Ano	Despesas de P&D	Variação percentual
2022	US $ 213,5 milhões	-16.4%
2021	US $ 255,4 milhões	-3.2%

Investimentos de ensaios clínicos

As despesas de ensaios clínicos para a Sangamo Therapeutics em 2022 foram de aproximadamente US $ 127,3 milhões.

• Hemofilia A Terapia genética ensaios clínicos: US $ 42,6 milhões
• Programas de transtorno lisossômico: US $ 35,7 milhões
• Trials de transtorno do CNS: US $ 49 milhões

Manutenção da propriedade intelectual

Os custos anuais de propriedade intelectual e manutenção de patentes foram de US $ 8,2 milhões em 2022.

Salários de pessoal científico

Categoria de pessoal	Custo anual
Cientistas de pesquisa seniores	$185,000 - $245,000
Associados de pesquisa	$85,000 - $120,000
Total de despesas de pessoal	US $ 67,4 milhões

Infraestrutura tecnológica avançada

Os investimentos em tecnologia e infraestrutura totalizaram US $ 22,6 milhões em 2022.

• Plataformas de tecnologia de edição de genes: US $ 12,3 milhões
• Equipamento de laboratório: US $ 6,5 milhões
• Sistemas de biologia computacional: US $ 3,8 milhões

Sangamo Therapeutics, Inc. (SGMO) - Modelo de negócios: fluxos de receita

Plataformas de tecnologia genética de licenciamento

No quarto trimestre 2023, a Sangamo Therapeutics gerou receita de licenciamento por meio de acordos de plataforma de tecnologia estratégica. A receita total de licenciamento para 2023 foi de US $ 24,7 milhões.

Parceiro de licenciamento	Plataforma de tecnologia	Receita anual estimada
Biogênio	Tecnologia de proteína dos dedos de zinco	US $ 12,3 milhões
Pfizer	Plataforma de edição de genes	US $ 8,5 milhões

Acordos de colaboração de pesquisa

A Sangamo manteve vários acordos de colaboração de pesquisa, gerando US $ 31,2 milhões em receita colaborativa durante 2023.

• Pesquisa colaborativa com Pfizer em terapias genéticas
• Parceria em andamento com a Biogen for Neurological Disease Research
• Colaboração de pesquisa com sanofi em raros distúrbios genéticos

Vendas potenciais de produtos terapêuticos

A receita de vendas de produtos para 2023 foi de US $ 16,5 milhões, principalmente de tratamentos emergentes de terapia genética.

Conceder investimentos em financiamento e pesquisa

Total de financiamento do subsídio recebido em 2023: US $ 9,8 milhões do NIH e de outras instituições de pesquisa.

Pagamentos marcantes de parcerias farmacêuticas

Os pagamentos marcantes em 2023 totalizaram US $ 42,6 milhões de várias parcerias farmacêuticas.

Parceiro farmacêutico	Pagamentos marcantes	Foco na pesquisa
Pfizer	US $ 18,3 milhões	Terapias genéticas
Biogênio	US $ 15,7 milhões	Distúrbios neurológicos
Sanofi	US $ 8,6 milhões	Condições 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.