Sangamo Therapeutics to Showcase Breakthrough Prion Disease Treatment at ASGCT Annual Meeting
核心洞察
Sangamo will present data on its novel zinc finger repressor therapy for prion disease (搜索) at the ASGCT Presidential Symposium, demonstrating sustained brain-wide protein suppression in mice and non-human primates.
The company's innovative approach combines zinc finger epigenetic regulators with its proprietary STAC-BBB delivery system, potentially offering a one-time intravenous treatment for this currently incurable neurodegenerative disease.
Additional presentations will highlight Sangamo's advances in chronic pain treatment targeting Nav1.7, second-generation AAV capsid engineering, and manufacturing innovations for CNS delivery systems.
Sangamo Therapeutics announced today that it will present significant advances in its neurology pipeline at the upcoming 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place May 13-17, 2025, in New Orleans, Louisiana.
The genomic medicine company will deliver nine presentations, including three oral presentations highlighting its zinc finger repressor (ZFR) technology for treating neurological diseases. Most notably, Sangamo's potential breakthrough treatment for prion disease (搜索) will be featured in the prestigious Presidential Symposium.
Groundbreaking Prion Disease Treatment Approach
Two presentations will showcase Sangamo's innovative approach to treating prion disease (搜索), a fatal and currently incurable neurodegenerative condition. The company has developed a zinc finger repressor targeting the prion gene, delivered using its proprietary STAC-BBB capsid, which can cross the blood-brain barrier after intravenous administration.
"We are particularly excited to be presenting, in the prestigious Presidential Symposium, our potent combination of epigenetic regulation and capsid delivery capabilities as an anticipated one-time intravenous treatment for prion disease (搜索)," said Sandy Macrae, Chief Executive Officer of Sangamo Therapeutics.
The data demonstrates profound survival benefits in disease mouse models and sustained, brain-wide suppression of prion protein (搜索) expression in both mouse and non-human primate models. This supports the potential of Sangamo's approach as a one-time therapeutic intervention for prion disease (搜索), which currently has no effective treatments.
Chronic Pain Treatment Advances
Sangamo will also present data on ST-503, a zinc finger repressor targeting the gene encoding Nav1.7, for treating intractable chronic neuropathic pain (搜索). The presentation will detail how this therapy achieved selective and potent repression of Nav1.7 in human sensory neurons and non-human primate dorsal root ganglion nociceptors following intrathecal injection.
This approach could provide a significant advancement for patients suffering from chronic pain conditions that are resistant to current therapies.
Innovations in AAV Capsid Engineering and Manufacturing
Additional poster presentations will highlight Sangamo's advances in adeno-associated virus (AAV) capsid engineering and manufacturing for central nervous system delivery. These include:
- Second-generation STAC-BBB variants with enhanced delivery to the central nervous system
- Receptor-targeted AAVs designed to penetrate the blood-brain barrier
- Manufacturing innovations to enhance yield, purity, and stability
- Improved analytical assessment methods for AAV products
"We are proud to be showcasing our latest pipeline advances at ASGCT, including three platform presentations, reflecting our mission to develop innovative neurology genomic medicines to treat debilitating neurological diseases," Macrae added.
Next-Generation Genome Engineering
Sangamo will also present updated data from its protein-guided Modular Integrase (MINT) platform, which enables engineering of large gene-sized pieces of DNA. This technology allows for compact therapeutic payloads and efficient targeted integration in T cells, expanding the potential applications of Sangamo's genomic medicine approach.
Presentation Schedule
The company's presentations at ASGCT will include:
Neurology Epigenetic Regulation
- "Sustained Brain-wide Reduction of Prion via Zinc Finger Repressors in Mice and Nonhuman Primates as a Potential One-Time Treatment for Prion Disease" - Presidential Symposium, May 14
- "Preclinical Development of an AAV-delivered Zinc Finger Transcriptional Repressor Targeting the Prion Gene as a Novel Epigenetic Gene Therapy for Prion Disease" - May 17
- "AAV-Mediated Delivery of an Engineered Zinc Finger Lead to Selective and Potent Repression of Nav1.7 in Human Sensory Neurons and Nonhuman Primates DRG Nociceptors Following Intrathecal Injection" - May 17
AAV Engineering and Production
- "Fitness Maturation of STAC-BBB Yields Second-Generation Capsid Variants with Enhanced Delivery to the Central Nervous System" - May 15
- "Characterization of Receptor-Targeted Blood-Brain Barrier Penetrant AAV Capsids" - May 15
- "The Impact of Empty Capsids on AAV Manufacturing and Strategies for Enhancing Yield, Purity, and Stability in the Production of a Novel Blood-Brain Barrier Penetrant AAV Capsid" - May 14
- "Recombinant Adeno-Associated Virus (rAAV) Production in Spodoptera Frugiperda (Sf9) Cells: Viral Cathepsin Mediated Capsid Cleavage and Mitigation Strategies" - May 13
- "Assessment of Adeno-Associated Virus (AAV) Purity by Capillary Electrophoresis-Based Western" - May 15
Next-Generation Genome Engineering
- "A Protein-Guided Modular Integrase (MINT) Platform Enables Compact Therapeutic Payloads and Efficient Targeted Integration in T Cells" - May 13
About Sangamo Therapeutics
Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options.
The company believes its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders, and that its capsid discovery platform can expand delivery beyond currently available intrathecal delivery capsids, including in the central nervous system.
Sangamo's approach combines precise gene targeting with innovative delivery methods, potentially offering new hope for patients with conditions that have previously been considered untreatable.