Resources

Publications

2026

Davis C et al. (ASGCT 2026) Defining pharmacologically relevant animal models for characterizing human transferrin receptor-binding AAVs, Abstract #1519.

Steinsapir, A et al. (ASGCT 2026) Establishing scalable TfR1 CapX manufacturing platform to accelerate multiple CNS gene therapy programs, Abstract #2237.

Wiltman, V et al. (ASGCT 2026) Open Aperture: An express MTA framework enabling academic evaluation of an hTfR1-targeting AAV capsid for systemic CNS delivery, Abstract #2236.

2025

Santiago-Ortiz et al. (2025) Fit-for-purpose analytical considerations to support the clinical readiness of engineered AAV vectors. Cell & Gene Therapy Insights. 2025 Dec 16; 11(11), 1369–1

Davis, C et al. Intravenous Delivery of a CNSPenetrant AAV Gene Therapy Provides Survival Benefit in a TSC1 Disease Model, Abstract #1531.

Santiago-Ortiz J et al. (ASGCT 2025) Fit-for-Purpose Analytics for Novel Engineered Capsids: Advancing Precision in AAV Characterization, Abstract #1320.

Santiago-Ortiz J et al. (ASGCT 2025) FDA-Aligned Strategy for AAV-Based TSC1 Gene Therapy: Advancing Preclinical Development Without Non-Human Primates, Abstract #305.

Steinsapir A et al. (ASGCT 2025) Redefining TSC2 Gene Therapy: Development of a Novel Split Intein Payload and a Translational Potency Assay, Abstract #1060.

Steinsapir A et al. (ASGCT 2025) Revolutionizing Neurometabolic Gene Therapy: The TfR1 CapX Platform and a Primate-Free Regulatory Pathway, Abstract #1900.

Wiltman V et al. Machine Learning-Enhanced Design of Experiments (DoE) for Optimizing AAV Plasmid Ratios in Gene Therapy Manufacturing, Abstract #378.

2024

Huang Q et al. (2024) An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery. Science, 384(6701), 1220-1227.

2023

Huang Q et al. An AAV capsid reprogrammed to bind human Transferrin Receptor mediates brain-wide gene delivery. BioRxiv 2023.12.20.572615

2022

Huang Q et al. (2022) Targeting AAV vectors to the CNS via de novo engineered capsid-receptor interactions. BioRxiv 10.31.514553.

2019

Hrvatin S et al. (2019) A scalable platform for the development of cell-type-specific viral drivers. eLife 8:e48089.

Huang Q et al. (2019) Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids. PLOS One. 2019 Nov 14;14(11):e0225206.

2018

Challis RC et al. Widespread and targeted gene expression by systemic AAV vectors: Production, purification, and administration. BioRxiv 246405.