Sangamo Therapeutics
Clinical-stage genomic medicine developer focused on engineering programmable DNA-targeting technologies and viral delivery systems to develop one-time or durable therapies for serious and rare diseases. Core activities include engineering of zinc-finger transcriptional regulators, serine integrase-based site-specific integration, AAV capsid engineering including blood-brain-barrier-penetrant capsids, preclinical safety and translational studies, and clinical development of gene therapies. Also conducts partnerships, technology transfer, and patient engagement programs.
Industries
Nr. of Employees
large (251-1000)
Sangamo Therapeutics
Patents
Products
Investigational AAV gene therapy candidate for Fabry disease (registrational Phase 1/2 program)
AAV-delivered gene therapy candidate under a registrational Phase 1/2 study with reported pharmacology, immunogenicity, renal and cardiac outcome presentations.
Investigational AAV gene therapy candidate for Hemophilia A (Phase 3 program)
AAV-based gene therapy candidate for Hemophilia A with Phase 3 clinical activity and long-term follow-up data reported; development conducted in collaboration with an external pharmaceutical partner.
Preclinical CNS-targeted epigenetic regulator candidates
Preclinical programs using engineered transcriptional repressors and blood–brain-barrier-penetrant delivery to lower disease-relevant CNS targets (examples include programs for small fiber neuropathy and prion disease).
Isaralgagene civaparvovec
A genomic medicine candidate for the treatment of Fabry Disease.
Isaralgagene civaparvovec (ST-920)
An investigational gene therapy using a recombinant AAV2/6 vector containing human GLA cDNA designed to produce continuous, liver-specific alpha-Gal-A expression for the treatment of Fabry disease.
giroctocogene fitelparvovec
A gene therapy candidate evaluated in the AFFINE Study, a Phase 3 study to evaluate clinical efficacy and safety in adult male participants with moderately severe or severe hemophilia A.
Investigational AAV gene therapy candidate for Fabry disease (registrational Phase 1/2 program)
AAV-delivered gene therapy candidate under a registrational Phase 1/2 study with reported pharmacology, immunogenicity, renal and cardiac outcome presentations.
Investigational AAV gene therapy candidate for Hemophilia A (Phase 3 program)
AAV-based gene therapy candidate for Hemophilia A with Phase 3 clinical activity and long-term follow-up data reported; development conducted in collaboration with an external pharmaceutical partner.
Preclinical CNS-targeted epigenetic regulator candidates
Preclinical programs using engineered transcriptional repressors and blood–brain-barrier-penetrant delivery to lower disease-relevant CNS targets (examples include programs for small fiber neuropathy and prion disease).
Isaralgagene civaparvovec
A genomic medicine candidate for the treatment of Fabry Disease.
Isaralgagene civaparvovec (ST-920)
An investigational gene therapy using a recombinant AAV2/6 vector containing human GLA cDNA designed to produce continuous, liver-specific alpha-Gal-A expression for the treatment of Fabry disease.
giroctocogene fitelparvovec
A gene therapy candidate evaluated in the AFFINE Study, a Phase 3 study to evaluate clinical efficacy and safety in adult male participants with moderately severe or severe hemophilia A.
Services
Design and execution of clinical programs including Phase 1/2 and registrational studies in rare diseases; long-term follow-up and clinical data generation.
Process and formulation development, stability and container/closure selection, and workflows to support scalable AAV production.
Out-licensing, strategic partnerships and alliance management to advance programs and expand reach through collaborations.
Design and execution of clinical programs including Phase 1/2 and registrational studies in rare diseases; long-term follow-up and clinical data generation.
Process and formulation development, stability and container/closure selection, and workflows to support scalable AAV production.
Out-licensing, strategic partnerships and alliance management to advance programs and expand reach through collaborations.
Expertise Areas
- Gene therapy development
- Programmable DNA-binding protein engineering (zinc-finger transcriptional regulation)
- AAV vector and capsid engineering
- Site-specific genome integration using integrases
Key Technologies
- Zinc-finger genome engineering
- Serine integrases for targeted integration
- Adeno-associated virus (AAV) vector engineering
- Blood-brain-barrier-penetrant capsid development
News & Updates
Announcement of selection of successful bidders following competitive asset auction and related asset sale agreements.
Announcement regarding asset sale agreements with external pharmaceutical companies.
Announcement of auction process for a clinical-stage chronic neuropathic pain program.
Press release announcing resumption of dosing in the Phase 3 AFFINE study evaluating an investigational AAV gene therapy for moderately severe to severe Hemophilia A; notes collaboration with an external pharmaceutical partner and transfer of manufacturing technology and IND.
Announcement describing updated corporate values and internal culture principles.
Announcement of appointment of new Chief Operating Officer, overseeing core functions including business development, alliance management, facilities, and information technology.
Announcement of selection of successful bidders following competitive asset auction and related asset sale agreements.
Announcement regarding asset sale agreements with external pharmaceutical companies.
Announcement of auction process for a clinical-stage chronic neuropathic pain program.
Press release announcing resumption of dosing in the Phase 3 AFFINE study evaluating an investigational AAV gene therapy for moderately severe to severe Hemophilia A; notes collaboration with an external pharmaceutical partner and transfer of manufacturing technology and IND.
Announcement describing updated corporate values and internal culture principles.
Announcement of appointment of new Chief Operating Officer, overseeing core functions including business development, alliance management, facilities, and information technology.