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 3

• Identification and Characterization of STAC-BBB, an Engineered AAV Capsid That Exhibits Widespread Transduction of the Central Nervous System in Cynomolgus Macaques
  • Abstract No. 117
  • Oral Presentation – May 8; 4:00-4:15 pm ET
• A Highly Potent Engineered AAV Capsid, STAC-150, Enables High-Throughput AAV Production and Arrayed Epigenetic Regulator Screening Directly in Cultured Neurons
  • Abstract No. 351
  • Oral Presentation – May 10; 5:15-5:30 pm ET
• Optimal Drug Product Presentation and Container Closure Selection for AAV-Based Genomic Medicines
  • Abstract No. 547
  • Poster Presentation – May 8; 5:30-7:00 pm ET
• Development of Blood-Brain Barrier Penetrant AAVs through Receptor-Targeted Capsid Engineering
  • Abstract No. 985
  • Poster Presentation – May 9; 5:30-7:00 pm ET
• Unraveling Impact of Manufacturing Process-Related Stresses on AAV Stability, Aggregation, and DNA Release
  • Abstract No. 1032
  • Poster Presentation – May 9; 5:30-7:00 pm ET
• Process and Formulation Development for a Novel Blood-Brain Barrier Penetrant AAV Capsid
  • Abstract No. 1052
  • Poster Presentation – May 9; 5:30-7:00 pm ET

Next-Generation Genome Engineering

• Directed Evolution of Bxb1 for the Development of Modular Integrases (MInts)
  • Abstract No. 192
  • Oral Presentation – May 9; 4:00-4:15 pm ET
• Zinc Finger Mediated Repression and Replacement of MFN2 Leads to the Rescue of Cellular Disease Phenotype in CMT2A Patient-Derived Cells
  • Abstract No. 637
  • Poster Presentation – May 8; 5:30-7:00 pm ET
• Highly Specific Zinc Finger Proteins with Synthetic Target Sites Enable Self-Regulated Expression of Dosage-Sensitive Transgenes
  • Abstract No. 722
  • Poster Presentation – May 8; 5:30-7:00 pm ET
• Site-directed integration of large DNA sequences into endogenous sites in the human genome using engineered Modular Integrases (MInts)
  • Abstract No. 1680
  • Poster Presentation – May 10; 5:30-7:00 pm ET

All abstracts for the ASGCT Annual Meeting are available on ASGCT’s website.