Sangamo Therapeutics, Inc.
Sangamo Therapeutics is a pioneering genomic medicine company dedicated to transforming patients' lives by developing innovative therapies for severe diseases with high unmet needs. They leverage proprietary zinc finger technology and collaborate with global pharmaceutical partners to create cures that could replace symptom management treatments. Their mission emphasizes translating groundbreaking science into life-changing medicines, with a focus on serious conditions such as neurodegenerative diseases, rare genetic disorders, and other severe illnesses. The company is committed to responsible business practices, diversity, equity, inclusion, and employee safety, aiming to develop cures for diseases one gene at a time.
Industries
Nr. of Employees
large (251-1000)
Sangamo Therapeutics, Inc.
Products
Investigational AAV-delivered gene therapy candidate for Fabry disease (registrational study)
An investigational AAV-based gene therapy program for Fabry disease that has progressed to a registrational clinical study.
Investigational gene therapy candidate for Hemophilia A
A clinical-stage gene therapy program for hemophilia A administered via AAV vectors and advanced through Phase clinical development.
Gene therapy candidate for idiopathic small fiber neuropathy (iSFN)
A clinical program addressing small fiber neuropathy using an investigational genomic medicine approach in early clinical development.
Epigenetic gene regulation therapies for neurodegenerative and neuropathic targets
Preclinical and early translational therapeutic candidates using transcriptional repressors and activators to modulate expression of CNS disease genes.
Modular Integrase platform for targeted integration
A platform enabling precise, site-directed integration of therapeutic payloads into the genome to support durable expression and compact payload designs.
Investigational AAV-delivered gene therapy candidate for Fabry disease (registrational study)
An investigational AAV-based gene therapy program for Fabry disease that has progressed to a registrational clinical study.
Investigational gene therapy candidate for Hemophilia A
A clinical-stage gene therapy program for hemophilia A administered via AAV vectors and advanced through Phase clinical development.
Gene therapy candidate for idiopathic small fiber neuropathy (iSFN)
A clinical program addressing small fiber neuropathy using an investigational genomic medicine approach in early clinical development.
Epigenetic gene regulation therapies for neurodegenerative and neuropathic targets
Preclinical and early translational therapeutic candidates using transcriptional repressors and activators to modulate expression of CNS disease genes.
Modular Integrase platform for targeted integration
A platform enabling precise, site-directed integration of therapeutic payloads into the genome to support durable expression and compact payload designs.
Services
AAV capsid engineering and CNS delivery platform development
Engineering and selection of AAV capsids optimized for blood–brain barrier penetration and CNS transduction, including receptor-targeted capsid design and directed-evolution approaches.
AAV manufacturing and process development
Process development for recombinant AAV production and purification, including baculovirus–Sf9 manufacturing workflows, yield optimization, capsid ratio control, and formulation selection for drug product.
Preclinical development and in vivo efficacy testing
Design and execution of preclinical studies in rodent and nonhuman primate models to assess biodistribution, target engagement, efficacy (including survival endpoints), and safety of genomic therapeutics.
Assay development and bioanalytics for immunogenicity and product quality
Development and validation of immunoassays to detect anti-transgene and neutralizing antibodies, and analytical methods for assessing vector purity and product-related impurities.
Clinical program conduct and regulatory support
Execution of clinical studies across early phases and registrational trials for rare genetic diseases and provision of support for regulatory submissions and interactions.
Bioethics and patient engagement services
Development of enhanced informed consent materials, long-term medical monitoring plans, and health information return practices tailored for genomic medicine clinical trials.
AAV capsid engineering and CNS delivery platform development
Engineering and selection of AAV capsids optimized for blood–brain barrier penetration and CNS transduction, including receptor-targeted capsid design and directed-evolution approaches.
AAV manufacturing and process development
Process development for recombinant AAV production and purification, including baculovirus–Sf9 manufacturing workflows, yield optimization, capsid ratio control, and formulation selection for drug product.
Preclinical development and in vivo efficacy testing
Design and execution of preclinical studies in rodent and nonhuman primate models to assess biodistribution, target engagement, efficacy (including survival endpoints), and safety of genomic therapeutics.
Assay development and bioanalytics for immunogenicity and product quality
Development and validation of immunoassays to detect anti-transgene and neutralizing antibodies, and analytical methods for assessing vector purity and product-related impurities.
Clinical program conduct and regulatory support
Execution of clinical studies across early phases and registrational trials for rare genetic diseases and provision of support for regulatory submissions and interactions.
Bioethics and patient engagement services
Development of enhanced informed consent materials, long-term medical monitoring plans, and health information return practices tailored for genomic medicine clinical trials.
Expertise Areas
- Gene therapy development
- Viral vector engineering and AAV capsid design
- CNS-targeted delivery and blood–brain barrier penetration
- Epigenetic regulation and transcriptional modulation
Key Technologies
- Zinc finger genome engineering
- Modular integrases for targeted integration
- AAV capsid engineering and directed evolution
- Baculovirus–Sf9 AAV production systems