PUBLICATIONS
CATCH UP WITH OUR LATEST PUBLICATIONS
Oral presentations from the American Society for Gene & Cell Therapy Annual Meeting – May 2025
Identification of Multiple Novel Blood-Brain-Barrier Receptors for CNS Gene Therapy and Other Drug Modalities via an Integrated AAV Capsid Engineering Platform – Download PDF
Systemic Gene Therapy CAP-002 Demonstrates Potential for Disease-Modifying Treatment of Seizures and Motor and Cognitive Deficits of STXBP1-DEE Using an Engineered, CNS-Targeted AAV – Download PDF
Systemic AAV Gene Therapy with Next Generation Engineered Capsid Demonstrates Expression Levels Supporting Potential Therapeutic Benefit for CNS, Cardiac, and Sensory Symptoms in Friedreich’s Ataxia – Download PDF
Posters from the American Society for Gene & Cell Therapy Annual Meeting – May 2025
Development of a Novel Automated Loading Approach Which Significantly Reduces Processing Time for Enriching Full AAV capsids Using Ultracentrifugation – Download PDF
Dual-Platform NGS for Comprehensive Characterization of Engineered rAAV Vector Integrity – Download PDF
CAP-003, a CNS-Targeted IV-delivered AAV Gene Therapy, Safely Increases Brain GCase in NHPs to Level Supporting Potential Normalization of Activity in PD-GBA Patients – Download PDF
rAAV Manufacturing Solutions: Strategic Designs of Engineered rAAV Two Plasmid Systems for Cost Effective Scaling and Product Safety (Abs 962) – Download PDF
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
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
Intravenous gene transfer throughout the brain of infant Old World primates using AAV
Chuapoco, Flytzanis, Goeden et al
biorxiv, January 2022