Sangamo Therapeutics to Highlight Neurology Pipeline Innovations with Nine Presentations at 28th ASGCT Annual Meeting
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company focused on developing transformative therapies for serious neurological conditions, announced that nine of its scientific abstracts have been accepted for presentation at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT). The meeting will take place May 13–17, 2025, in New Orleans, Louisiana, with both in-person and virtual attendance options.
The company’s presentations will spotlight significant advancements across its neurology pipeline, including cutting-edge developments in zinc finger-based epigenetic regulation, engineered capsid delivery systems, and modular integrase (MINT) technology.
“We are honored to share our latest neurology-focused innovations at this year’s ASGCT meeting,” said Sandy Macrae, Chief Executive Officer of Sangamo Therapeutics. “With three platform presentations, including one selected for the prestigious Presidential Symposium, we’re excited to demonstrate the potential of our zinc finger technology and delivery platforms to drive meaningful progress in treating devastating neurological disorders. This reflects the dedication of our teams and our commitment to pushing the boundaries of genomic medicine.”
Three oral presentations will feature Sangamo’s pioneering work using zinc finger repressors (ZFRs) for targeted epigenetic modulation of neurological disease genes. Two of these presentations—including the one in the Presidential Symposium—will detail the combination of a ZFR targeting the prion gene with Sangamo’s STAC-BBB, a neurotropic capsid engineered for intravenous delivery. This therapeutic strategy has shown significant survival benefits and broad, durable suppression of prion protein expression in both rodent and nonhuman primate models, positioning it as a promising one-time treatment approach for prion disease—a currently untreatable and fatal neurodegenerative condition.
The third oral presentation will cover ST-503, a ZFR candidate targeting the Nav1.7 gene, designed for the treatment of chronic, intractable neuropathic pain through intrathecal administration.
In addition to the oral sessions, Sangamo will present several posters highlighting advances in adeno-associated virus (AAV) capsid engineering for improved central nervous system (CNS) delivery. These include next-generation STAC-BBB variants, receptor-targeted AAV constructs, and process innovations aimed at enhancing vector yield, purity, stability, and analytical characterization.
The company will also unveil updated data on its Modular Integrase (MINT) platform, a novel protein-guided approach designed to facilitate precise integration of large gene-sized DNA sequences—potentially broadening the reach of genomic engineering applications.
Collectively, these presentations underscore Sangamo’s continued leadership in advancing genomic medicine platforms for challenging neurological diseases.
ASGCT Annual Meeting Presentations and Invited Sessions
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
- Abstract No. 2
- Oral Presentation – May 14; 11:30-11:45 am CT
- Session Title: Presidential Symposium
- Preclinical Development of an AAV-delivered Zinc Finger Transcriptional Repressor Targeting the Prion Gene as a Novel Epigenetic Gene Therapy for Prion Disease
- Abstract No. 389
- Oral Presentation – May 17; 8:45-9:00 am CT
- Session Title: Pharmacology/Toxicology Studies and Analytics/Assay Development Session II
- 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
- Abstract No. 369
- Oral Presentation – May 17; 8:45-9:00 am CT
- Session Title: Translational Approaches: Gene Therapy of Neurological Diseases in Large Animal Models
AAV Engineering and Production for the Central Nervous System
- Fitness Maturation of STAC-BBB Yields Second-Generation Capsid Variants with Enhanced Delivery to the Central Nervous System
- Abstract No. 1909
- Poster Presentation – May 15; 5:30-7:00 pm CT
- Characterization of Receptor-Targeted Blood-Brain Barrier Penetrant AAV Capsids
- Abstract No. 1896
- Poster Presentation – May 15; 5:30-7:00 pm CT
- 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
- Abstract No. 1464
- Poster Presentation – May 14; 5:30-7:00 pm CT
- Recombinant Adeno-Associated Virus (rAAV) Production in Spodoptera Frugiperda (Sf9) Cells: Viral Cathepsin Mediated Capsid Cleavage and Mitigation Strategies
- Abstract No. 987
- Poster Presentation – May 13; 6:00-7:30 pm CT
- Assessment of Adeno-Associated Virus (AAV) Purity by Capillary Electrophoresis-Based Western
- Abstract No. 1814
- Poster Presentation – May 15; 5:30-7:00 pm CT
Next-Generation Genome Engineering
- A Protein-Guided Modular Integrase (MINT) Platform Enables Compact Therapeutic Payloads and Efficient Targeted Integration in T Cells
- Abstract No. 648
- Poster Presentation – May 13; 6:00-7:30 pm CT
