Oral presentation from American Academy of Neurology (AAN) Annual Meeting – April 2025

Systemic AAV Gene Therapy Using a CNS-targeted Engineered Capsid Significantly Increases GCase Activity to Support the Potential Treatment of PD-GBA – Download PDF

Oral presentation from International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) – April 2025

Systemic AAV Gene Therapy with CNS-Targeted Engineered Capsids Significantly Increases GCase Activity to Support the Potential Treatment of PD-GBA – Download PDF

Oral presentation from the Muscular Dystrophy Association Conference Mar 2025

CAP-004: Systemic AAV Gene Therapy with Engineered Capsids for Treatment of CNS and Cardiac Symptoms in Friedreich’s Ataxia – Download PDF

Poster from the American Epilepsy Society Annual Meeting – Dec 2024

Systemic Gene Therapy with Engineered AAV Demonstrates Preclinical Efficacy and Safety Supporting a Disease-Modifying Treatment for STXBP1 Developmental and Epileptic Encephalopathy – Download PDF

Poster from the International Congress for Ataxia Research – Nov 2024

Systemic AAV Gene Therapy with Next Generation Engineered Capsids for Treatment of CNS and Cardiac Symptoms in Friedreich’s Ataxia – Download PDF

Poster from the Neuroscience 2024 Society for Neuroscience Annual Meeting – Oct 2024

Systemic AAV Gene Therapy with CNS-Targeted Engineered Capsids Achieves Significant GCase Activity Increases in the Primate Brain to Support Potential Treatment of PD-GBA – Download PDF

Oral presentations from the American Society for Gene & Cell Therapy Annual Meeting – May 2024

Systemic AAV Gene Therapy with CNS-Targeted Engineered Capsids Achieves Significant GCase Activity Increases in the Primate Brain to Support the Potential Treatment of GBA-PD – Download PDF

Directed Evolution of AAV9 Libraries in Non-Human Primates Identifies a Capsid Family with Enhanced Central Nervous System Tropism and Liver De-Targeting Following Systemic Delivery – Download PDF

Posters from the American Society for Gene & Cell Therapy Annual Meeting – May 2024

Directed Evolution of AAV2 Libraries Yields Capsids with Improved Performance in the Central Nervous System and Cross-Species Translatability – Download PDF

Alternative Plasmid Designs Including Two Plasmid Transfection Systems for Improved Production and Packaging of Engineered AAV Capsids – Download PDF

Characterization of Engineered AAV Capsids from Different HEK293 Cell Culture Fractions, Crude Lysate Versus Cell Pellet Material – Download PDF

Separation of Empty and Full Engineered Adeno-Associated Virus Capsids Using a Weak Anion Exchanger – Download PDF

CAP-002: Systemic AAV Gene Therapy with Next Generation Capsids for Treatment of STXBP1 Encephalopathy – Download PDF

Adeno-associated viral vectors for functional intravenous gene transfer throughout the non-human primate brain

Chuapoco, Flytzanis, Goeden, et al
Nature Nanotechnology, July 2023

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Posters from the American Society for Gene & Cell Therapy Annual Meeting – 2023

• Optimization of a Versatile Downstream Process for Multiple Novel AAV Capsids That Demonstrates Improved Recovery – Download PDF
• Exploring upstream process conditions to improve productivity for novel AAV Capsids – Download PDF
• Development and Comparative Assessment of a Rapid, High-Throughput Method for Accurate Quantification of Host Cell DNA Impurities in Final Drug Product and Process Intermediates – Download PDF

Posters from the American Society for Gene & Cell Therapy Annual Meeting – 2022

• Optimization of a scalable upstream process for production of novel AAV capsids – Download PDF
• Manufacturability and product quality of novel AAV capsids optimized for specific targeting using capsid library selection in non-human primates – Download PDF
• Next-generation automated AAV engineering platform for rapid identification of efficient and specific capsids in non-human primates – Download PDF
• Multiple generations of capsids engineered in NHPs yield improved performance in the central nervous system – Download PDF
• Combinatorial engineering across multiple surface exposed loops of AAV2 and AAV9 yields capsids with high degrees of enrichment and specificity for target tissues – Download PDF

AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset

Goertsen, Flytzanis, Goeden et al
Nature Neuroscience, January 2022

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Intravenous gene transfer throughout the brain of infant Old World primates using AAV

Chuapoco, Flytzanis, Goeden et al
biorxiv, January 2022

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