Sangamo Therapeutics to Present Neurology Pipeline Advances at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

Sangamo is using groundbreaking science and novel technology to replace today’s symptomatic treatments with tomorrow’s genomic cures.


NEUROLOGY PIPELINE - WHOLLY OWNED
Indication Preclinical Phase 1/2 Pivotal Partner
Idiopathic Small Fiber Neuropathy (ST-503)
None
Prion Disease
None
Undisclosed neurology target(s)
None
NEUROLOGY PIPELINE - PARTNERED
Indication Preclinical Phase 1/2 Pivotal Partner
Genentech
Genentech logo
Undisclosed neurology target
Cargo Icon Capsid Icon
Genentech
Genentech logo
Undisclosed neurology target
Capsid Icon
Astellas
Astellas logo
Undisclosed neurology target
Capsid Icon
Lilly
Lilly logo
Alexion
alexion logo
Takeda
takeda logo
OTHER PROGRAMS
Indication Preclinical Phase 1/2 Pivotal Partner
Hemophilia A (Giroctogene fitelparvovec)
Pfizer
pfizer logo

*until April 21, 2025

Fabry Disease (Isaralgagene civaparvovec)
None
Cargo Icon Cargo
Capsid Icon Capsid

Clinical Programs

Clinical Trials

Learn more about our approach to clinical trials.

Sangamo is conducting several clinical trials to evaluate the safety, tolerability, and potential efficacy of its genomic medicines, some of which we lead and some of which are led by our global pharmaceutical company collaboration partners.

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Collaborations

We seek collaborations that can extend or complement our leading genomic medicine capabilities

Sangamo’s novel technology and depth of scientific expertise make us a partner of choice among leading global pharmaceutical companies. Whether through in-licensing, out-licensing, scientific collaborations or advancing our corporate development priorities, our collaborations extend the reach of our technology and accelerate the development of genomic medicines. These are some of the ways we deliver on our commitment to get new treatments to patients in need.


Our Partners

Sangamo and Alexion are collaborating for the development and commercialization of therapeutics using our zinc finger transcription factors (ZF-TFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene.

Astellas and Sangamo have entered into a worldwide exclusive license agreement allowing Astellas to use Sangamo’s novel proprietary neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, for up to five neurological disease targets.

nChromaBio (formerly Chroma Medicine) has a research evaluation, option and license agreement with Sangamo to explore zinc finger proteins (ZFPs) for epigenetic editing in specified targets outside of the central nervous system.

Sangamo has a global epigenetic regulation and capsid delivery license agreement with Genentech to develop novel genomic medicines for neurodegenerative diseases, focused on the tau gene, which is critically involved in Alzheimer’s disease and other tauopathies, as well as a second undisclosed neurology target.

Lilly and Sangamo have entered into a worldwide exclusive license agreement allowing Lilly to use Sangamo’s novel proprietary neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, for up to five potential disease targets of the central nervous system.

Image: Pfizer Logo

Pfizer and Sangamo have an exclusive, global collaboration and license agreement to develop and commercialize gene therapies for hemophilia A.

partner-logo-prevail

Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, has a research evaluation and option agreement with Sangamo to evaluate certain proprietary novel engineered cerebrospinal fluid-administered capsids with enhanced nervous system delivery, generated through Sangamo’s capsid engineering platform, SIFTER.

Image: Takeda Logo

Takeda and Sangamo are developing therapeutics for Huntington's disease utilizing our epigenetic regulation platform.

Interested in collaborating with us?

Contact Stephanie Seiler, Head of Business Development and Alliance Management

Contact Stephanie