Sangamo Therapeutics to Present Neurology-Focused Pre-Clinical Data From Its Epigenetic Regulation, Capsid Delivery and Genome Engineering Platforms at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) - Seite 2

Additional poster presentations at the ASGCT Annual Meeting will showcase the use of zinc finger activators and repressors for the potential treatment of neurological diseases such as prion disease, tauopathies, Charcot-Marie-Tooth disease type 1A (CMT1A), Dravet Syndrome, SOD1-mediated amyotrophic lateral sclerosis (ALS), Phelan-McDermid syndrome, Parkinson’s disease, Angelman syndrome, and other CNS disorders. Sangamo will also present additional data from its AAV capsid engineering platform SIFTER and its efforts for integrase evolution and utilization.

ASGCT Annual Meeting Presentations and Invited Sessions

Epigenetic Regulation for the Central Nervous System

• Restoration of Normal Gene and Protein Expression in Mouse and Human Disease Models of SCN2A Haploinsufficiency Using Zinc Finger Activators
  • Abstract No. 636
  • Poster Presentation – May 8; 5:30-7:00 pm ET
• A Zinc Finger Activator Platform to Restore Normal Gene & Protein Expression in Cellular Models of Dravet Syndrome
  • Abstract No. 642
  • Poster Presentation – May 8; 5:30-7:00 pm ET
• SNCA Gene Repression Mediated by Zinc Finger Repressors (ZFRs) as a Therapeutic Approach for Parkinson's Disease
  • Abstract No. 1120
  • Poster Presentation – May 9; 5:30-7:00 pm ET
• UBE3A Gene Activation Mediated by Zinc Finger Activators (ZFAs) as a Therapeutic Approach for Angelman Syndrome
  • Abstract No. 1121
  • Poster Presentation – May 9; 5:30-7:00 pm ET
• Whole CNS Human Tau Knockdown for the Potential Treatment of Alzheimer’s Disease and Other Tauopathies
  • Abstract No. 1126
  • Poster Presentation – May 9; 5:30-7:00 pm ET
• SOD1 Gene Repression Mediated by Zinc Finger Repressors (ZFRs) as a Therapeutic Approach for SOD1-Mediated ALS
  • Abstract No. 1597
  • Poster Presentation – May 10; 5:30-7:00 pm ET
• PMP22 Gene Repression Mediated by Zinc Finger Repressors (ZFRs) as a Therapeutic Approach for CMT1A
  • Abstract No. 1600
  • Poster Presentation – May 10; 5:30-7:00 pm ET
• Shank3 Gene Activation Mediated by Zinc Finger Transcriptional Activators (ZFA) as a Therapeutic Approach for Phelan-McDermid Syndrome
  • Abstract No. 1605
  • Poster Presentation – May 10; 5:30-7:00 pm ET
• Development of a Robust Zinc Finger Activation Platform for Treatment of Neurological Disorders
  • Abstract No. 1609
  • Poster Presentation – May 10; 5:30-7:00 pm ET
• Epigenetic Regulation of Human Prion Expression as a Potential One-Time Treatment for Prion Disease
  • Abstract No. 1616
  • Poster Presentation – May 10; 5:30-7:00 pm ET

Viral Engineering for the Central Nervous System