Publications
& Presentations

ASGCT 2024:

Friday, May 10th

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

Thursday, May 9th 

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

Establishment of a predictive transcytosis model to recapitulate capsid-receptor interaction and phenotype of BBB-penetrant AAV variants

Discovery of TRACER AAV capsids escaping pre-existing neutralizing antibodies 

Machine learning for AAV production-fitness modeling

Comparing CsCl density gradient ultracentrifugation and anion exchange chromatography for the enrichment of full adeno-associated viral (AAV) vectors

Development of HEK293 cell line for optimal production of Novel capsids with enhanced brain tropism

Wednesday, May 8th 

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

High-resolution quantitative analysis of multiple AAV capsids in rodent and primate models using multiplexed reporter protein tagging platform 

ADPD 2024:
Intravenous administration of BBB-penetrant AAV containing primary artificial microRNA targeting tau reduces tau broadly and robustly in hTau mouse brain

Pharmacokinetics and tolerability of VY-TAU01, an anti-tau antibody for the treatment of Alzheimer’s disease, in P301S mouse and nonhuman primate

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

BioProcessing Summit 2023:
Rapid HEK Platform Scale Up to 200 L Using kLa Modeling

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

ASGCT 2023:
Stepwise Evolution of the AAV5-Derived Capsid VCAP-100 Identifies Novel Variants with Improved CNS Transduction and Liver Detargeting Following Systemic Injection

ASGCT 2023:
An Evolved AAV Variant with Enhanced Brain and Spinal Cord Tropism and Translation across Primate Species

ADPD 2023:
Selection of an Anti-tau Antibody Candidate Targeting Pathological Tau for the Treatment of Alzheimer’s Disease

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:
Intravenous Delivery of AAV Gene Therapy Provides Broad SOD1 Knockdown in the Spinal Cord and Robust Efficacy in a Mouse Model of SOD1-ALS

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