On May 2, 2023 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported that the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) has accepted 14 Sangamo abstracts for presentation at the 26th ASGCT (Free ASGCT Whitepaper) Annual Meeting being held May 16-20, 2023, in-person in Los Angeles, CA and in a virtual format (Press release, Sangamo Therapeutics, MAY 2, 2023, View Source [SID1234630861]). Presentations will focus on the progression of Sangamo’s pre-clinical programs, including data from the prioritized neurology programs Nav1.7 and Prion, innovations in the epigenetic regulation platform and advances in AAV capsid engineering for delivery.

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"The data to be presented at ASGCT (Free ASGCT Whitepaper) reflect important advances in areas core to our strategy, including epigenetic regulation for neurological disorders and engineered AAV capsids to potentially address the issues of delivery," said Jason Fontenot, Ph.D., Chief Scientific Officer at Sangamo. "We are excited to share such a depth of pre-clinical data and look forward to presenting the innovation that continues to emerge from our research efforts, to potentially deliver transformative medicines to patients in need."

Data to be presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting include an oral presentation from Sangamo’s flagship neurology epigenetic regulation program Nav1.7 to treat chronic neuropathic pain. This presentation will showcase how Sangamo is using an optimized zinc finger epigenetic repressor to specifically target and seek to reduce Nav1.7 expression in dorsal root ganglions to inhibit pain sensation in diseases of chronic neuropathic pain. Beginning with Nav1.7-associated small fiber neuralgia, with an estimated prevalence of at least 43,000 patients in the US, future success in this indication could subsequently enable the broadened use of this therapy to other neuropathic pain indications, regardless of the cause of the pain.

Additional presentations at the ASGCT (Free ASGCT Whitepaper) Annual Meeting will showcase how Sangamo is advancing its proprietary zinc finger platform development efforts, including the use of zinc finger transcriptional regulators designed to reduce the expression of the Prion protein for the treatment of Creutzfeldt-Jakob disease. Sangamo will also present data from its proprietary AAV capsid discovery platform SIFTER, which enables the engineering of AAV capsids with the potential for highly improved central nervous system transduction efficiency, along with broader presentations showcasing Sangamo’s latest genomic engineering platform evolution.

ASGCT Annual Meeting Presentations and Invited Sessions

Epigenetic Regulation for the Central Nervous System

Zinc Finger Transcriptional Regulator Mediated Repression of SCN9A Gene as a Therapeutic Approach for Painful Peripheral Neuropathies
Abstract No. 32
Oral Presentation – May 17; 3:45-4:00 pm PT
Engineered Zinc Finger Transcriptional Regulators Specifically Reduce Prion Expression and Extend Survival in an Aggressive Prion Disease Model
Abstract No. 490
Poster Presentation – May 17; 5:30-7:00 pm PT
Cell-Type Specific Reduction of Prion Expression in Neurons and Astrocytes Using Engineered Zinc Finger Transcriptional Regulators
Abstract No. 1561
Poster Presentation – May 19; 5:30-7:00 pm PT
Intercellular Zinc Finger Protein Delivery for Cross-Corrective Epigenetic Regulation in the CNS
Abstract No. 1485
Poster Presentation – May 19; 5:30-7:00 pm PT
Gene Activation Mediated by Zinc Finger Transcriptional Regulators (ZF-TRs) as a Therapeutic Approach for CNS Disorders
Abstract No. 1057
Poster Presentation – May 18; 5:30-7:00 pm PT
Viral Engineering for the Central Nervous System

Fitness Maturation of Engineered AAV Capsids STAC-102 and STAC-103 Enhances Central Nervous System Transduction after CSF Administration in Cynomolgus Macaques
Abstract No. 1698
Poster Presentation – May 19; 5:30-7:00 pm PT
Evolution of Blood-Brain Barrier Penetrant AAV Capsids in Non-Human Primates Using a Multiplexed Transcription Dependent Capsid Engineering Platform
Abstract No. 1203
Poster Presentation – May 18; 5:30-7:00 pm PT
Process and Platform Development for Production and Purification of CNS-Tropic Engineered AAV Capsids
Abstract No. 1460
Poster Presentation – May 19; 5:30-7:00 pm PT
Strategic Formulation Development for AAV Delivered Gene Therapies – a Case Study
Abstract No. 1186
Poster Presentation – May 18; 5:30-7:00 pm PT
Genomic Engineering Platform Evolution

Engineered Zinc Finger Transcriptional Regulators Enable Robust and Reliable Epigenetic Regulation in the Mouse Brain
Abstract No. 682
Poster Presentation – May 17; 5:30-7:00 pm PT
Zinc Finger-Transcriptional Repressors Against Immune Checkpoint Molecules to Improve Anti-Tumor Activity of Gene-Modified T Cells
Abstract No. 1680
Poster Presentation – May 19; 5:30-7:00 pm PT
A Robust and Flexible Baculovirus-Insect Cell System for AAV Vector Production with Improved Yield, Capsid Ratios and Potency
Abstract No. 471
Poster Presentation – May 17; 5:30-7:00 pm PT
Assays to Support Clinical Programs

Overcoming the Effect of Previous Enzyme Replacement Therapy on the Detection of Anti-Transgene Protein Antibodies After Treatment with Gene Therapy
Abstract No. 727
Poster Presentation – May 17; 5:30-7:00 pm PT
Development of a Competitive Ligand-Binding Assay to Detect Neutralizing Antibodies Against Chimeric Antigen Receptor of Regulatory T Cells
Abstract No. 891
Poster Presentation – May 18; 5:30-7:00 pm PT
All abstracts for the ASGCT (Free ASGCT Whitepaper) Annual Meeting are available on ASGCT (Free ASGCT Whitepaper)’s website.