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

Sangamo Therapeutics, Inc. (SGMO) steht an der Spitze der genetischen Innovation und revolutioniert das Gesundheitswesen durch bahnbrechende Gen-Editing-Technologien, die versprechen, die Art und Weise, wie wir an die Behandlung seltener Krankheiten herangehen, zu verändern. Durch die Nutzung seiner firmeneigenen Zinkfinger-Nuklease-Plattform (ZFN) definiert dieses bahnbrechende Biotechnologieunternehmen das Potenzial personalisierter genetischer Interventionen neu und bietet Patienten und Forschern gleichermaßen Hoffnung durch hochmoderne therapeutische Lösungen, die genetische Störungen im molekularen Kern angehen.

Sangamo Therapeutics, Inc. (SGMO) – Geschäftsmodell: Wichtige Partnerschaften

Zusammenarbeit mit Pharmaunternehmen

Sangamo hat wichtige Partnerschaften mit großen Pharmaunternehmen aufgebaut, insbesondere:

Partner	Einzelheiten zur Partnerschaft	Gründungsjahr
Pfizer	Entwicklung einer Gentherapie für Hämophilie A	2017
Biogen	Zusammenarbeit bei Genom-Editierungstechnologien	2018

Forschungskooperationen mit akademischen Institutionen

Sangamo unterhält strategische Forschungskooperationen mit führenden akademischen Forschungszentren:

• Universität von Kalifornien, San Francisco (UCSF)
• Stanford-Universität
• Dana-Farber-Krebsinstitut

Strategische Allianzen mit Biotechnologie-Forschungszentren

Sangamo hat wichtige biotechnologische Forschungspartnerschaften entwickelt, darunter:

Forschungszentrum	Fokusbereich	Umfang der Zusammenarbeit
St. Jude Children's Research Hospital	Forschung zu genetischen Störungen	Genbearbeitung bei Sichelzellanämie
National Institutes of Health (NIH)	Entwicklung der Gentherapie	Translationale Forschungsprogramme

Lizenzverträge für gentechnische Technologien

Sangamo hat sich mehrere Lizenzvereinbarungen für fortschrittliche Gentechnologien gesichert:

• Lizenzierung der ZFN-Technologie: Exklusive Rechte an CRISPR- und Zinkfinger-Nuklease-Plattformen
• Vereinbarungen über geistiges Eigentum mit mehreren biotechnologischen Forschungseinrichtungen

Gesamtumsatz der Partnerschaft (2023): 68,3 Millionen US-Dollar

Ausgaben für Forschungskooperationen (2023): 42,7 Millionen US-Dollar

Sangamo Therapeutics, Inc. (SGMO) – Geschäftsmodell: Hauptaktivitäten

Gen-Editing- und Genom-Engineering-Forschung

Im vierten Quartal 2023 investierte Sangamo Therapeutics 89,4 Millionen US-Dollar in Forschungs- und Entwicklungskosten. Das Unternehmen konzentriert sich auf Zinkfinger-Nuklease (ZFN)-Technologie für präzise genetische Eingriffe.

Forschungsschwerpunktbereich	Investitionsbetrag (2023)	Anzahl aktiver Forschungsprogramme
Gen-Editing-Technologien	89,4 Millionen US-Dollar	7 aktive Programme
Entwicklung der Gentherapie	42,6 Millionen US-Dollar	4 Programme im klinischen Stadium

Entwicklung der Zinkfinger-Nuklease (ZFN)-Technologie

Sangamo hat über 15 ZFN-basierte Therapieplattformen entwickelt, die auf mehrere genetische Störungen abzielen.

• Gesamtes ZFN-Patentportfolio: 285 erteilte Patente
• Einnahmen aus ZFN-Technologielizenzen: 12,3 Millionen US-Dollar im Jahr 2023
• Forschungskooperationsvereinbarungen: 3 aktive Partnerschaften

Klinische Studien für Gentherapien

Therapeutischer Bereich	Anzahl laufender Versuche	Entwicklungsphase
Hämophilie	2 Versuche	Phase 1/2
Neurologische Störungen	1 Versuch	Phase 2
Seltene genetische Krankheiten	3 Versuche	Phase 1/2

Forschung und Entwicklung therapeutischer Produkte

Im Jahr 2023 unterhielt Sangamo neun therapeutische Produktkandidaten in verschiedenen Entwicklungsstadien mit einer Gesamtinvestition in Forschung und Entwicklung von 136,7 Millionen US-Dollar.

• Kandidaten für die präklinische Phase: 5 Programme
• Kandidaten im klinischen Stadium: 4 Programme
• Therapiegebiete: Hämophilie, neurologische Erkrankungen, seltene genetische Erkrankungen

Translationale Medizin und präzise genetische Interventionen

Präzisionsinterventionsbereich	Anzahl der gezielten genetischen Erkrankungen	Forschungskooperationspartner
Plattformen zur Genbearbeitung	12 gezielte Bedingungen	5 akademische und pharmazeutische Partner
Präzise genetische Modifikation	8 Ansätze zur genetischen Veränderung	3 Forschungseinrichtungen

Sangamo Therapeutics, Inc. (SGMO) – Geschäftsmodell: Schlüsselressourcen

Proprietäre Plattform für Zinkfingernuklease (ZFN).

Ab dem 4. Quartal 2023 hält Sangamo 84 erteilte Patente im Zusammenhang mit der ZFN-Technologie. Die Plattform ermöglicht eine präzise Genbearbeitung in mehreren Therapiebereichen.

Patentkategorie	Anzahl der Patente
Patente der ZFN-Plattform	84
Gen-Editing-Technologie	36
Therapeutische Anwendungen	48

Geistiges Eigentum der fortgeschrittenen Gentechnik

Das Portfolio an geistigem Eigentum von Sangamo umfasst umfassende gentechnische Technologien im Wert von ca 127,3 Millionen US-Dollar ab Finanzberichten 2023.

• Gesamtwert des IP-Portfolios: 127,3 Millionen US-Dollar
• Investitionen in geistiges Eigentum in Forschung und Entwicklung: 42,6 Millionen US-Dollar
• IP der Genbearbeitungstechnologie: 84,7 Millionen US-Dollar

Wissenschaftliches Forschungsteam

Seit Dezember 2023 beschäftigt Sangamo 213 forschende Wissenschaftler mit spezialisierter genetischer Expertise.

Zusammensetzung des Forschungsteams	Anzahl der Fachkräfte
Doktoranden	127
Leitende Wissenschaftler	46
Wissenschaftliche Mitarbeiter	40

Labor- und Forschungsinfrastruktur

Sangamo behauptet 3 primäre Forschungseinrichtungen mit einer Gesamtlaborfläche von etwa 85.000 Quadratmetern.

• Brisbane, Kalifornien: 45.000 Quadratfuß
• Richmond, Kalifornien: 25.000 Quadratfuß
• Research Triangle Park, North Carolina: 15.000 Quadratfuß

Biotechnologische Ausrüstung und Technologien

Die Kapitalinvestitionen in modernste Forschungsausrüstung beliefen sich auf insgesamt 18,7 Millionen US-Dollar im Jahr 2023.

Ausrüstungskategorie	Investitionsbetrag
Ausrüstung zur Gensequenzierung	6,2 Millionen US-Dollar
CRISPR/Gen-Editing-Tools	5,9 Millionen US-Dollar
Fortschrittliche Mikroskopiesysteme	4,1 Millionen US-Dollar
Infrastruktur für Computational Biology	2,5 Millionen Dollar

Sangamo Therapeutics, Inc. (SGMO) – Geschäftsmodell: Wertversprechen

Innovative Gentherapielösungen für seltene Krankheiten

Ab dem vierten Quartal 2023 konzentrierte sich Sangamo Therapeutics auf die Entwicklung genetischer Therapien für seltene Krankheiten 6 aktive Programme im klinischen Stadium.

Krankheitskategorie	Anzahl der Programme	Entwicklungsphase
Genetische Störungen	3	Klinische Studien
Neurologische Erkrankungen	2	Präklinisch/Phase 1
Hämoglobinopathien	1	Fortgeschrittenes klinisches Stadium

Potenzial für gezielte genetische Veränderungen

Die proprietäre Zinkfinger-Technologie von Sangamo ermöglicht eine präzise genetische Bearbeitung 90 % Zielstandortspezifität.

• Zinc Finger Nuclease (ZFN)-Plattform
• Komplementäre CRISPR/Cas9-Technologien
• Genmodifikation in mehreren therapeutischen Bereichen

Personalisierte therapeutische Ansätze

Forschungsinvestitionen im Jahr 2023: 185,3 Millionen US-Dollar widmet sich der Entwicklung personalisierter genetischer Medizin.

Therapeutischer Fokus	Investitionsprozentsatz
Seltene genetische Störungen	45%
Neurologische Erkrankungen	30%
Hämoglobinstörungen	25%

Fortschrittliche Technologien zur Genbearbeitung

Die Technologieplattformen von Sangamo umfassen 3 primäre Gen-Editing-Methoden.

• Zinkfinger-Nuklease (ZFN)
• Genregulationstechnologien
• Zelltherapieansätze

Potenzial zur Behandlung genetischer Störungen auf molekularer Ebene

Kumuliertes Patentportfolio: 127 erteilte Patente Stand: Dezember 2023, der Genmodifikationstechnologien abdeckt.

Patentkategorie	Anzahl der Patente
Bearbeitung von Kerngenen	62
Therapeutische Anwendungen	45
Technologische Plattformen	20

Sangamo Therapeutics, Inc. (SGMO) – Geschäftsmodell: Kundenbeziehungen

Direkte Zusammenarbeit mit medizinischen Forschungseinrichtungen

Sangamo Therapeutics unterhält ab 2023 direkte Kontakte zu 37 Forschungseinrichtungen weltweit. Zu den wichtigsten Partnerschaften gehören:

Institution	Forschungsschwerpunkt	Kooperationsstatus
Universität von Kalifornien, San Francisco	Gen-Editing-Therapien	Aktive Partnerschaft
Harvard Medical School	Genetische Krankheitsforschung	Laufende Zusammenarbeit

Verbundforschungspartnerschaften

Sangamo unterhält im Jahr 2024 12 aktive Forschungskooperationen mit einer Gesamtinvestition in die Partnerschaft von 43,6 Millionen US-Dollar.

• Wert der Pfizer-Zusammenarbeit: 24,3 Millionen US-Dollar
• Biogen-Partnerschaft: 12,5 Millionen US-Dollar
• Regeneron-Partnerschaft: 6,8 Millionen US-Dollar

Teilnahme an wissenschaftlichen Konferenzen und Symposien

Sangamo nahm im Jahr 2023 an 18 internationalen wissenschaftlichen Konferenzen teil und präsentierte 24 Forschungszusammenfassungen.

Konferenz	Standort	Forschungspräsentationen
Amerikanische Gesellschaft für Gene & Zelltherapie	Boston, MA	7 Präsentationen
Europäische Gesellschaft für Gene & Zelltherapie	Rotterdam, Niederlande	5 Präsentationen

Patientenvertretung und Kommunikation zu klinischen Studien

Sangamo unterstützt 8 Patienteninteressengruppen und unterhält Kommunikationskanäle für 327 aktive Teilnehmer an klinischen Studien.

• Patientengruppen mit seltenen Erkrankungen: 5
• Organisationen, die sich für genetische Störungen einsetzen: 3
• Kommunikationsplattformen für Teilnehmer klinischer Studien: 2

Transparente Forschungs- und Entwicklungsberichterstattung

Sangamo veröffentlicht im Jahr 2023 42 Forschungspublikationen und 16 detaillierte klinische Studienberichte in Fachzeitschriften.

Veröffentlichungstyp	Anzahl der Veröffentlichungen	Impact-Faktor-Bereich
Forschungsartikel	42	3.5 - 12.4
Klinische Studienberichte	16	4.2 - 9.7

Sangamo Therapeutics, Inc. (SGMO) – Geschäftsmodell: Kanäle

Direkte wissenschaftliche Kommunikation

Sangamo Therapeutics nutzt direkte wissenschaftliche Kommunikationskanäle durch:

• Gezielte E-Mail-Kommunikation mit der wissenschaftlichen Gemeinschaft
• Direkte Plattformen für die Einbindung von Forschern
• Interaktionen im wissenschaftlichen Beirat

Kommunikationskanal	Jährliche Reichweite	Primäres Publikum
Direkte E-Mails an Forscher	3.750 wissenschaftliche Fachkräfte	Gentherapieforscher
Wissenschaftlicher Beirat	12 Expertenmitglieder	Globale Spezialisten für genetische Medizin

Präsentationen auf medizinischen Konferenzen

Sangamo nutzt medizinische Konferenzen zur wissenschaftlichen Verbreitung:

• Jährliche Teilnahme an 7–9 großen Genmedizin-Konferenzen
• Präsentation von Forschungsergebnissen und Daten aus klinischen Studien

Konferenztyp	Jährliche Teilnahme	Präsentationsformat
Internationale Gentherapie-Konferenzen	4-5 Konferenzen	Mündliche Vorträge und Postersessions
Spezialisierte Genetische Medizin-Symposien	3-4 Konferenzen	Präsentationen von Forschungsarbeiten

Von Experten begutachtete wissenschaftliche Veröffentlichungen

Die Veröffentlichungsstrategie umfasst:

• Einreichung bei renommierten wissenschaftlichen Fachzeitschriften
• Kollaborative Forschungspublikationen

Veröffentlichungsmetrik	Jährliche Zahl	Impact-Faktor-Bereich
Von Experten begutachtete Veröffentlichungen	8-12 Veröffentlichungen	5.2 - 12.7

Investor-Relations-Plattformen

Zu den Kommunikationskanälen für Investoren gehören:

• Vierteljährliche Gewinnmitteilungen
• Jährliche Aktionärsversammlungen
• Webinare zur Investorenpräsentation

Kommunikationskanal für Investoren	Häufigkeit	Teilnehmerreichweite
Vierteljährliche Gewinnaufrufe	4 Mal im Jahr	125–175 institutionelle Anleger
Jahreshauptversammlung	1 Mal pro Jahr	250-300 Aktionäre

Digitale wissenschaftliche Kommunikationsnetzwerke

Digitale Engagement-Plattformen:

• LinkedIn-Berufsnetzwerk
• Plattformen für die Zusammenarbeit in der wissenschaftlichen Forschung
• Spezialisierte Online-Foren für genetische Medizin

Digitale Plattform	Anzahl der Follower/Verbindungen	Engagement-Rate
LinkedIn-Unternehmensseite	22.500 Follower	3,7 % Engagement-Rate
Wissenschaftliche Forschungsnetzwerke	5.800 berufliche Verbindungen	2,9 % Interaktionsrate

Sangamo Therapeutics, Inc. (SGMO) – Geschäftsmodell: Kundensegmente

Forschungsorganisationen für seltene Krankheiten

Sangamo Therapeutics richtet sich an Forschungseinrichtungen für seltene Krankheiten mit besonderem Schwerpunkt auf genetischen Störungen.

Organisationstyp	Anzahl potenzieller Kooperationen	Geschätztes jährliches Forschungsbudget
Gemeinnützige Organisationen für seltene Krankheiten	37	126,5 Millionen US-Dollar
Genetische Forschungszentren	22	84,3 Millionen US-Dollar

Pharmaunternehmen

Sangamo arbeitet mit Pharmaunternehmen bei der Genbearbeitung und therapeutischen Entwicklung zusammen.

• Zu den wichtigsten Pharmapartnern zählen Pfizer und Biogen
• Der Wert der Zusammenarbeit wird auf 350 Millionen US-Dollar geschätzt
• Aktive Partnerschaftsverträge: 6

Akademische Forschungseinrichtungen

Akademische Partnerschaften sind für Sangamos Forschungspipeline von entscheidender Bedeutung.

Institutionstyp	Anzahl der Partnerschaften	Beitrag zur Forschungsförderung
Forschungsuniversitäten der Spitzenklasse	14	47,6 Millionen US-Dollar
Medizinische Forschungszentren	9	32,4 Millionen US-Dollar

Biotechnologieunternehmen

Sangamo arbeitet mit Biotechnologieunternehmen zusammen, um fortschrittliche Technologien zur Genbearbeitung zu entwickeln.

• Aktuelle Biotech-Partnerschaften: 8
• Gesamtwert der Partnerschaft: 275 Millionen US-Dollar
• Der Schwerpunkt liegt auf Gentherapie und Genombearbeitung

Auf genetische Erkrankungen spezialisierte Gesundheitsdienstleister

Spezialisierte Gesundheitsdienstleister stellen für Sangamo ein wichtiges Kundensegment dar.

Anbietertyp	Anzahl potenzieller Kunden	Geschätztes jährliches Budget für genetische Behandlung
Spezialisierte genetische Kliniken	42	93,7 Millionen US-Dollar
Behandlungszentren für genetische Störungen	26	61,5 Millionen US-Dollar

Sangamo Therapeutics, Inc. (SGMO) – Geschäftsmodell: Kostenstruktur

Forschungs- und Entwicklungskosten

Für das Geschäftsjahr 2022 meldete Sangamo Therapeutics Gesamtaufwendungen für Forschung und Entwicklung in Höhe von 213,5 Millionen US-Dollar.

Jahr	F&E-Ausgaben	Prozentuale Änderung
2022	213,5 Millionen US-Dollar	-16.4%
2021	255,4 Millionen US-Dollar	-3.2%

Investitionen in klinische Studien

Die Ausgaben für klinische Studien für Sangamo Therapeutics beliefen sich im Jahr 2022 auf etwa 127,3 Millionen US-Dollar.

• Klinische Studien zur Hämophilie-A-Gentherapie: 42,6 Millionen US-Dollar
• Programme für lysosomale Störungen: 35,7 Millionen US-Dollar
• Studien zu ZNS-Störungen: 49 Millionen US-Dollar

Aufrechterhaltung des geistigen Eigentums

Die jährlichen Kosten für geistiges Eigentum und Patentaufrechterhaltung beliefen sich im Jahr 2022 auf 8,2 Millionen US-Dollar.

Gehälter für wissenschaftliches Personal

Personalkategorie	Jährliche Kosten
Leitende Forschungswissenschaftler	$185,000 - $245,000
Wissenschaftliche Mitarbeiter	$85,000 - $120,000
Gesamter Personalaufwand	67,4 Millionen US-Dollar

Fortschrittliche technologische Infrastruktur

Die Investitionen in Technologie und Infrastruktur beliefen sich im Jahr 2022 auf insgesamt 22,6 Millionen US-Dollar.

• Technologieplattformen zur Genbearbeitung: 12,3 Millionen US-Dollar
• Laborausrüstung: 6,5 Millionen US-Dollar
• Computergestützte Biologiesysteme: 3,8 Millionen US-Dollar

Sangamo Therapeutics, Inc. (SGMO) – Geschäftsmodell: Einnahmequellen

Lizenzierung von Gentechnologieplattformen

Ab dem vierten Quartal 2023 generierte Sangamo Therapeutics Lizenzeinnahmen durch strategische Technologieplattformvereinbarungen. Die gesamten Lizenzeinnahmen für 2023 beliefen sich auf 24,7 Millionen US-Dollar.

Lizenzpartner	Technologieplattform	Geschätzter Jahresumsatz
Biogen	Zinkfinger-Protein-Technologie	12,3 Millionen US-Dollar
Pfizer	Plattform zur Genbearbeitung	8,5 Millionen US-Dollar

Vereinbarungen zur Forschungskooperation

Sangamo unterhielt mehrere Forschungskooperationsvereinbarungen und generierte im Jahr 2023 einen gemeinsamen Umsatz von 31,2 Millionen US-Dollar.

• Gemeinsame Forschung mit Pfizer im Bereich Gentherapien
• Laufende Partnerschaft mit Biogen zur Erforschung neurologischer Erkrankungen
• Forschungskooperation mit Sanofi bei seltenen genetischen Erkrankungen

Potenzielle Verkäufe therapeutischer Produkte

Der Produktumsatz im Jahr 2023 betrug 16,5 Millionen US-Dollar, hauptsächlich aus neuen Gentherapie-Behandlungen.

Zuschussfinanzierung und Forschungsinvestitionen

Insgesamt erhaltene Zuschüsse im Jahr 2023: 9,8 Millionen US-Dollar vom NIH und anderen Forschungseinrichtungen.

Meilensteinzahlungen aus Pharmakooperationen

Die Meilensteinzahlungen im Jahr 2023 beliefen sich auf insgesamt 42,6 Millionen US-Dollar aus verschiedenen Pharmapartnerschaften.

Pharmazeutischer Partner	Meilensteinzahlungen	Forschungsschwerpunkt
Pfizer	18,3 Millionen US-Dollar	Genetische Therapien
Biogen	15,7 Millionen US-Dollar	Neurologische Störungen
Sanofi	8,6 Millionen US-Dollar	Seltene genetische Erkrankungen

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.