The Perspective of DMPK on Recombinant Adeno-Associated Virus-Based Gene Therapy: Past Learning, Current Support, and Future Contribution

Current progress and limitations of AAV mediated delivery of protein therapeutic genes and the importance of developing quantitative pharmacokinetic/pharmacodynamic (PK/PD) models

(2021) Ekram Ahmed Chowdhury et al.   ADVANCED DRUG DELIVERY REVIEWS

Evolving AAV-delivered therapeutics towards ultimate cures

(2021) Xiangjun He et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

2020 White Paper on Recent Issues in Bioanalysis: Vaccine Assay Validation, qPCR Assay Validation, QC for CAR-T Flow Cytometry, NAb Assay Harmonization and ELISpot Validation (Part 3 – Recommendations on Immunogenicity Assay Strategies, NAb Assays, Biosimilars and FDA/EMA Immunogenicity Guidance/Guideline, Gene & Cell Therapy and Vaccine Assays)

(2021) Bart Corsaro et al.   Bioanalysis

Rational Clinical Dose Selection of Adeno‐Associated Virus‐Mediated Gene Therapy Based on Allometric Principles

(2021) Fei Tang et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Current and Next Steps Toward Prediction of Human Dose for Gene Therapy Using Translational Dose‐Response Studies

(2021) Sergey Aksenov et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

IQ consortium perspective: complementary LBA and LC–MS in protein therapeutics bioanalysis and biotransformation assessment

(2020) Surinder Kaur et al.   Bioanalysis

Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

(2020) Giuseppe Ronzitti et al.   Frontiers in Immunology

Translational Potential of Immune Tolerance Induction by AAV Liver-Directed Factor VIII Gene Therapy for Hemophilia A

(2020) Benjamin J. Samelson-Jones et al.   Frontiers in Immunology

Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future

(2020) Agnieszka Łoboda et al.   Pharmacological Reports

Hybrid LC–MS as a powerful tool for supporting protein bioanalysis in gene and cell therapies

(2020) Dong Wei et al.   Bioanalysis

T-Cell Dependent Immunogenicity of Protein Therapeutics Pre-clinical Assessment and Mitigation–Updated Consensus and Review 2020

(2020) Vibha Jawa et al.   Frontiers in Immunology

Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?

(2020) Karim Bey et al.   Molecular Therapy-Methods & Clinical Development

High-resolution histological landscape of AAV DNA distribution in cellular compartments and tissues following intramuscular and intravenous injection

(2020) Junling Zhao et al.   Molecular Therapy-Methods & Clinical Development

Activity of Transgene-Produced B-Domain Deleted Factor VIII in Human Plasma Following AAV5 Gene Therapy

(2020) Steffen Rosen et al.   BLOOD

The Impact of Product and Process Related Critical Quality Attributes on Immunogenicity and Adverse Immunological Effects of Biotherapeutics

(2020) Yi Wen et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

Current Clinical Applications of In Vivo Gene Therapy with AAVs

(2020) Jerry R. Mendell et al.   MOLECULAR THERAPY

qPCR and qRT-PCR analysis: Regulatory points to consider when conducting biodistribution and vector shedding studies

(2020) Haiyan Ma et al.   Molecular Therapy-Methods & Clinical Development

The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy

(2019) Brian R. Long et al.   Molecular Therapy-Methods & Clinical Development

Enhanced Factor IX Activity Following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 wildtype Human Factor IX in Non-human Primates

(2019) Elisabeth A. Spronck et al.   Molecular Therapy-Methods & Clinical Development

Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells

(2019) Jamie L. Shirley et al.   MOLECULAR THERAPY

Bioanalysis of adeno-associated virus gene therapy therapeutics: regulatory expectations

(2019) Boris Gorovits et al.   Bioanalysis

Engineering the AAV capsid to evade immune responses

(2019) Christopher Barnes et al.   CURRENT OPINION IN BIOTECHNOLOGY

2019 White Paper on Recent Issues in Bioanalysis: FDA Immunogenicity Guidance, Gene Therapy, Critical Reagents, Biomarkers and Flow Cytometry Validation (Part 3 – Recommendations on 2019 FDA Immunogenicity Guidance, Gene Therapy Bioanalytical Challenges, Strategies for Critical Reagent Management, Biomarker Assay Validation, Flow Cytometry Validation & CLSI H62)

(2019) Steven Piccoli et al.   Bioanalysis

Critical assessment of digital PCR for the detection and quantification of genetically modified organisms

(2018) Tigst Demeke et al.   ANALYTICAL AND BIOANALYTICAL CHEMISTRY

Optimized Adeno-Associated Virus-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques

(2018) Jenny Greig et al.   HUMAN GENE THERAPY

Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice

(2018) Stuart Bunting et al.   MOLECULAR THERAPY

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques

(2018) Juliette Hordeaux et al.   Molecular Therapy-Methods & Clinical Development

Considerations for Preclinical Safety Assessment of Adeno-Associated Virus Gene Therapy Products

(2018) Basel T. Assaf et al.   TOXICOLOGIC PATHOLOGY

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Direct Liquid Chromatography/Mass Spectrometry Analysis for Complete Characterization of Recombinant Adeno-Associated Virus Capsid Proteins

(2017) Xiaoying Jin et al.   Human Gene Therapy Methods

Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria

(2016) Delia D’Avola et al.   JOURNAL OF HEPATOLOGY

A Bottom-Up Whole-Body Physiologically Based Pharmacokinetic Model to Mechanistically Predict Tissue Distribution and the Rate of Subcutaneous Absorption of Therapeutic Proteins

(2015) Katherine L. Gill et al.   AAPS Journal

Immunogenicity assessment of biotherapeutic products: An overview of assays and their utility

(2015) Meenu Wadhwa et al.   BIOLOGICALS

Quantification of proteins by flow cytometry: Quantification of human hepatic transporter P-gp and OATP1B1 using flow cytometry and mass spectrometry

(2015) Karen Hogg et al.   METHODS

Guidelines for the automated evaluation of Elispot assays

(2015) Sylvia Janetzki et al.   Nature Protocols

Safety, Biodistribution, and Efficacy of an AAV-5 Vector Encoding Human Interferon-Beta (ART-I02) Delivered via Intra-Articular Injection in Rhesus Monkeys with Collagen-Induced Arthritis

(2015) Lisette Bevaart et al.   Human Gene Therapy Clinical Development

Recommendations for Validation of LC-MS/MS Bioanalytical Methods for Protein Biotherapeutics

(2014) Rand Jenkins et al.   AAPS Journal

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

The immune epitope database (IEDB) 3.0

(2014) Randi Vita et al.   NUCLEIC ACIDS RESEARCH

Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates

(2014) Darren A. Murrey et al.   Human Gene Therapy Clinical Development

The Digital MIQE Guidelines: Minimum Information for Publication of Quantitative Digital PCR Experiments

(2013) J. F. Huggett et al.   CLINICAL CHEMISTRY

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

(2013) S J Gray et al.   GENE THERAPY

Safety and Liver Transduction Efficacy of rAAV5-cohPBGD in Nonhuman Primates: A Potential Therapy for Acute Intermittent Porphyria

(2013) Astrid Pañeda et al.   HUMAN GENE THERAPY

Immunogenicity of mAbs in non-human primates during nonclinical safety assessment

(2013) Peter J.K. van Meer et al.   mAbs

Systemic Errors in Quantitative Polymerase Chain Reaction Titration of Self-Complementary Adeno-Associated Viral Vectors and Improved Alternative Methods

(2012) Paolo Fagone et al.   Human Gene Therapy Methods

Toll-like receptor 2-mediated innate immune response in human nonparenchymal liver cells toward adeno-associated viral vectors

(2011) Marianna Hösel et al.   HEPATOLOGY

Capsid-specific T-cell Responses to Natural Infections With Adeno-associated Viruses in Humans Differ From Those of Nonhuman Primates

(2011) Hua Li et al.   MOLECULAR THERAPY

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Innate Immune Responses to AAV Vectors

(2011) Geoffrey L. Rogers et al.   Frontiers in Microbiology

The MIQE Guidelines: Minimum Information for Publication of Quantitative Real-Time PCR Experiments

(2009) S. A. Bustin et al.   CLINICAL CHEMISTRY

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

(2009) Jiangao Zhu et al.   JOURNAL OF CLINICAL INVESTIGATION

Gene Therapy Using Adeno-Associated Virus Vectors

(2008) S. Daya et al.   CLINICAL MICROBIOLOGY REVIEWS

Enhancing transduction of the liver by adeno-associated viral vectors

(2008) A C Nathwani et al.   GENE THERAPY