Publications
& Presentations

Molecular Therapy:
Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier

ASGCT 2025:

Tuesday, May 13th

Cross-Species BBB-Penetrant IV-Delivered AAV Gene Therapy Provides Broad and Robust CNS tau Lowering in Tauopathy Mouse Models and Non-Human Primate

One-Time Delivery of a Vectorized Anti-Amyloid Antibody for Increased and Sustained CNS Expression and Target Engagement

Wednesday, May 14th

Oral Presentation: Discovery of AAV9-Derived CNS Capsids Evading Pre-Existing Neutralizing Antibodies

Enabling Large Scale Implementation of Anion Exchange Chromatography for Full Capsid Enrichment of a Novel Adeno-Associated Viral Vector

An Alternative to Detergent Lysis: Promoting rAAV Release to Media by Optimizing Osmolality, pH and Harvest Timing

Thursday, May 15th

Oral Presentation: Intravenous Delivery of VY1706, a CNS Penetrant AAV Gene Therapy for Alzheimer’s Disease, Provides Broad Tau Lowering in NHP

Machine-Learning for AAV9 Mutant-Capsid Screening for Both Production and ALPL-Mediated Transduction Efficiency

Assessment of Two HEK293 Cell Line Cloning Strategies to Improve AAV Yield

ADPD 2025:
VY1706, A BBB-Penetrant, IV-delivered AAV Gene Therapy Provides Broad and Robust CNS tau Lowering in Tauopathy Mouse Models and Non-Human Primate

Discrimination of Anti-tau Antibodies Targeting Different tau Epitopes by a P301S Mouse Hippocampal Seeding Model of Tauopathy

ASGCT 2024:

Intravenous administration of BBB-penetrant, MAPT-Silencing, AAV gene therapy provides broad and robust CNS Tau lowering in tauopathy mouse models

Intravenous delivery of AAV gene therapy for the treatment of SOD1-ALS provides broad SOD1 lowering in NHP

Evaluation of cross-species expression across four species and cellular tropism of VCAP-102, an engineered blood-brain barrier-penetrating AAV derived capsid from TRACER Platform screens 

Identification and characterization of a highly conserved cell surface receptor utilized by engineered BBB-penetrant AAV capsids with enhanced brain tropism in non-human primates and mice

Oral Presentation: Continued directed evolution of VCAP-101 and VCAP-102 identifies second generation capsids with increased brain tropism in non-human primates and mice

Oral Presentation: Developability assessment of novel AAV capsids and payloads at early preclinical stage to enable development of AAV gene therapies

In Vivo Gene Therapy and Genome Editing Summit 2023:
Iterative Evolution of Cross-Species BBB-Penetrant Capsids​

ASGCT 2023:
Directed evolution of an AAV9 library identifies a capsid variant with enhanced brain tropism and liver de-targeting in non-human primates and mice following systemic administration

ASGCT 2023:
Discovery and Characterization of Novel CrossSpecies BBB-Penetrant Capsids

ESGCT 2022:
Dose-response Evaluation of 9P801, an Engineered AAV Capsid with High BBB Penetration and CNS Transduction in Non-human Primates

ESGCT 2022:
Identification of a Cell Surface Receptor Utilized by an Engineered BBB-Penetrant Capsid Family with Enhanced Brain Tropism in Non-Human Primates and Mice

ASGCT 2022:
Directed Evolution of AAV9 Peptide Display Libraries Identifies a Family of Cross-Species Variants with Enhanced Brain Tropism in Non-Human Primates and Mice Following Systemic Administration​

ASGCT 2022:
Development of AAV-GBA1 Gene Replacement Therapy for IV Delivery via Blood Brain Barrier Penetrant AAV Capsid

AAIC 2022:
Identification and Characterization of Novel Anti-tau Antibodies that Inhibit Tau-seed Mediated Pathology in a P301S Tauopathy Mouse Model of Alzheimer’s Disease and Tauopathies

Cell 2020:
Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning