Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 14 Sangamo abstracts for presentation at the 26^th ASGCT Annual Meeting being held May 16-20, 2023, in-person in Los Angeles, CA and in a virtual format. 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.

“The data to be presented at ASGCT 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 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 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 Annual Meeting are available on ASGCT’s website.

About Sangamo Therapeutics

Sangamo Therapeutics is a clinical-stage biopharmaceutical company with a robust genomic medicines pipeline. Using ground-breaking science, including our proprietary zinc finger genome engineering technology and manufacturing expertise, Sangamo aims to create new genomic medicines for patients suffering from diseases for which existing treatment options are inadequate or currently don’t exist. To learn more, visit www.sangamo.com and connect with us on LinkedIn and Twitter.

Sangamo Forward-Looking Statements

This press release contains forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to Sangamo’s technologies, the presentation of data from various therapeutic and research programs and the potential of these programs to demonstrate therapeutic benefit and transform the lives of patients. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the research development process, including the results of clinical trials; the regulatory approval process for product candidates; and the potential for technological developments that obviate technologies used by Sangamo. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business. These risks and uncertainties are described more fully in our Securities and Exchange Commission filings and reports, including in our Annual Report on Form 10-K for the year ended December 31, 2022. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

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