4.7 Review

IgG Fusion Proteins for Brain Delivery of Biologics via Blood-Brain Barrier Receptor-Mediated Transport

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Chemistry, Medicinal

Kinetics of Blood-Brain Barrier Transport of Monoclonal Antibodies Targeting the Insulin Receptor and the Transferrin Receptor

William M. Pardridge

Summary: Biologic drugs are large molecule pharmaceuticals that cannot cross the blood-brain barrier. They can be re-engineered for BBB transport as IgG fusion proteins. The kinetics of BBB transport can be quantified with mathematical models.

PHARMACEUTICALS (2022)

Article Chemistry, Multidisciplinary

Transferrin Receptor Binding BBB-Shuttle Facilitates Brain Delivery of Anti-Aβ-Affibodies

Rebecca Faresjo et al.

Summary: Affibodies targeting amyloid-beta (A beta) show potential as therapeutic and diagnostic agents in Alzheimer's disease. Fusion of Affibodies with the blood-brain barrier shuttle scFv8D3 improves brain delivery and retention. However, the specificity of affibodies for A beta deposits needs further confirmation.

PHARMACEUTICAL RESEARCH (2022)

Article Biotechnology & Applied Microbiology

A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II

Torayuki Okuyama et al.

Summary: The study demonstrated that pabinafusp alfa is a potentially effective treatment for both the central and peripheral symptoms of MPS-II patients, with the ability to cross the blood-brain barrier to reach the central nervous system. In the 52-week clinical trial, the drug showed relatively good efficacy and safety profiles.

MOLECULAR THERAPY (2021)

Article Biotechnology & Applied Microbiology

Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil

Roberto Giugliani et al.

Summary: The novel drug pabinafusp alpha has shown potential in crossing the blood-brain barrier and addressing neurodegenerative diseases, with favorable clinical outcomes in MPS-II patients.

MOLECULAR THERAPY (2021)

Article Genetics & Heredity

Nonclinical safety evaluation of pabinafusp alfa, an anti-human transferrin receptor antibody and iduronate-2-sulfatase fusion protein, for the treatment of neuronopathic mucopolysaccharidosis type II

Ryuji Yamamoto et al.

Summary: Pabinafusp alfa is a fusion protein developed to target central nervous system-related symptoms in patients with MPS II. Nonclinical safety assessment showed that it did not exhibit effector functions or significant toxicological changes in animal studies, indicating minimal impact on the iron transport system. These findings suggest that pabinafusp alfa is expected to be safe for long-term use in individuals with MPS II.

MOLECULAR GENETICS AND METABOLISM REPORTS (2021)

Article Genetics & Heredity

Examination of a blood-brain barrier targeting 8-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis

Michael J. Przybilla et al.

Summary: GM1-gangliosidosis is a lysosomal disease characterized by a deficiency in hydrolase 8-galactosidase, leading to neurological deterioration. A novel fusion enzyme mTfR-GLB1 was designed to cross the blood-brain barrier and showed efficacy in improving enzyme activity in the liver and spleen. However, behavioral tests indicated limited improvement in treated mice, and there was no significant increase in enzyme activity in the brain or reduction in ganglioside content.

MOLECULAR GENETICS AND METABOLISM REPORTS (2021)

Article Chemistry, Medicinal

Mathematical Models of Blood-Brain Barrier Transport of Monoclonal Antibodies Targeting the Transferrin Receptor and the Insulin Receptor

William M. Pardridge et al.

Summary: This study examines the receptor-mediated transcytosis of monoclonal antibodies targeting transferrin receptor or insulin receptor at the blood-brain barrier, using mathematical models to analyze and predict the transport process. The optimal rates of receptor association with the targeted antibodies are highlighted as crucial factors determining the efficiency of transcytosis.

PHARMACEUTICALS (2021)

Article Cell Biology

Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice

Julie C. Ullman et al.

SCIENCE TRANSLATIONAL MEDICINE (2020)

Article Multidisciplinary Sciences

Bi-functional IgG-lysosomal enzyme fusion proteins for brain drug delivery

Ruben J. Boado et al.

SCIENTIFIC REPORTS (2019)

Article Clinical Neurology

Hematologic safety of chronic brain-penetrating erythropoietin dosing in APP/PS1 mice

Jiahong Sun et al.

ALZHEIMERS & DEMENTIA-TRANSLATIONAL RESEARCH & CLINICAL INTERVENTIONS (2019)

Article Medicine, Research & Experimental

Brain Penetrating Bifunctional Erythropoietin-Transferrin Receptor Antibody Fusion Protein for Alzheimer's Disease

Rudy Chang et al.

MOLECULAR PHARMACEUTICS (2018)

Article Medicine, Research & Experimental

Blood-Brain Barrier Penetrating Biologic TNF-α Inhibitor for Alzheimer's Disease

Rudy Chang et al.

MOLECULAR PHARMACEUTICS (2017)

Review Pharmacology & Pharmacy

CSF, blood-brain barrier, and brain drug delivery

William M. Pardridge

EXPERT OPINION ON DRUG DELIVERY (2016)

Article Medicine, Research & Experimental

Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo

Michael B. Tropak et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2016)

Article Biochemistry & Molecular Biology

Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan

Cara M. Weismann et al.

HUMAN MOLECULAR GENETICS (2015)

Article Biochemical Research Methods

IgG-Enzyme Fusion Protein: Pharmacokinetics and Anti-Drug Antibody Response in Rhesus Monkeys

Ruben J. Boado et al.

BIOCONJUGATE CHEMISTRY (2013)

Article Cell Biology

Addressing Safety Liabilities of TfR Bispecific Antibodies That Cross the Blood-Brain Barrier

Jessica A. Couch et al.

SCIENCE TRANSLATIONAL MEDICINE (2013)

Article Endocrinology & Metabolism

Brain protection from stroke with intravenous TNFα decoy receptor-Trojan horse fusion protein

Rachita K. Sumbria et al.

JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM (2012)

Article Pharmacology & Pharmacy

Brain-Penetrating IgG-Iduronate 2-Sulfatase Fusion Protein for the Mouse

Qing-Hui Zhou et al.

DRUG METABOLISM AND DISPOSITION (2012)

Article Biochemical Research Methods

Delivery of a Peptide Radiopharmaceutical to Brain with an IgG-Avidin Fusion Protein

Qing-Hui Zhou et al.

BIOCONJUGATE CHEMISTRY (2011)

Article Biotechnology & Applied Microbiology

Expression in CHO Cells and Pharmacokinetics and Brain Uptake in the Rhesus Monkey of an IgG-Iduronate-2-Sulfatase Fusion Protein

Jeff Zhiqiang Lu et al.

BIOTECHNOLOGY AND BIOENGINEERING (2011)

Article Biochemistry & Molecular Biology

Quantitative targeted absolute proteomics of human blood-brain barrier transporters and receptors

Yasuo Uchida et al.

JOURNAL OF NEUROCHEMISTRY (2011)

Article Medicine, Research & Experimental

Receptor-Mediated Abeta Amyloid Antibody Targeting to Alzheimer's Disease Mouse Brain

Qing-Hui Zhou et al.

MOLECULAR PHARMACEUTICS (2011)

Article Medicine, Research & Experimental

Reversal of Lysosomal Storage in Brain of Adult MPS-I Mice with Intravenous Trojan Horse-Iduronidase Fusion Protein

Ruben J. Boado et al.

MOLECULAR PHARMACEUTICS (2011)

Article Pharmacology & Pharmacy

Neuroprotection with a Brain-Penetrating Biologic Tumor Necrosis Factor Inhibitor

Qing-Hui Zhou et al.

JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2011)

Article Pharmacology & Pharmacy

Chronic Dosing of Mice with a Transferrin Receptor Monoclonal Antibody-Glial-Derived Neurotrophic Factor Fusion Protein

Qing-Hui Zhou et al.

DRUG METABOLISM AND DISPOSITION (2011)

Article Biochemical Research Methods

Genetic Engineering of a Bifunctional IgG Fusion Protein with Iduronate-2-Sulfatase

Jeff Zhiqiang Lu et al.

BIOCONJUGATE CHEMISTRY (2010)

Article Pharmacology & Pharmacy

Monoclonal Antibody-Glial-Derived Neurotrophic Factor Fusion Protein Penetrates the Blood-Brain Barrier in the Mouse

Qing-Hui Zhou et al.

DRUG METABOLISM AND DISPOSITION (2010)

Article Biotechnology & Applied Microbiology

Selective targeting of a TNFR decoy receptor pharmaceutical to the primate brain as a receptor-specific IgG fusion protein

Ruben J. Boado et al.

JOURNAL OF BIOTECHNOLOGY (2010)

Article Pharmacology & Pharmacy

Genetic engineering of IgG-glucuronidase fusion proteins

Ruben J. Boado et al.

JOURNAL OF DRUG TARGETING (2010)

Article Pharmacology & Pharmacy

Drug Targeting of Erythropoietin Across the Primate Blood-Brain Barrier with an IgG Molecular Trojan Horse

Ruben J. Boado et al.

JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2010)

Article Medicine, Research & Experimental

Re-Engineering Erythropoietin as an IgG Fusion Protein That Penetrates the Blood-Brain Barrier in the Mouse

Qing-Hui Zhou et al.

MOLECULAR PHARMACEUTICS (2010)

Review Neurosciences

Structure and function of the blood-brain barrier

N. Joan Abbott et al.

NEUROBIOLOGY OF DISEASE (2010)

Article Biotechnology & Applied Microbiology

Engineering and Expression of a Chimeric Transferrin Receptor Monoclonal Antibody for Blood-Brain Barrier Delivery in the Mouse

Ruben J. Boado et al.

BIOTECHNOLOGY AND BIOENGINEERING (2009)

Review Neurosciences

Immunotherapy, Vascular Pathology, and Microhemorrhages in Transgenic Mice

Donna M. Wilcock et al.

CNS & NEUROLOGICAL DISORDERS-DRUG TARGETS (2009)

Article Anesthesiology

Effects of Etanercept and Minocycline in a rat model of spinal cord injury

Fabien Marchand et al.

EUROPEAN JOURNAL OF PAIN (2009)

Article Biotechnology & Applied Microbiology

AGT-181: Expression in CHO cells and pharmacokinetics, safety, and plasma iduronidase enzyme activity in Rhesus monkeys

Ruben J. Boado et al.

JOURNAL OF BIOTECHNOLOGY (2009)

Article Endocrinology & Metabolism

Neurodegenerative lysosomal storage disease in European Burmese cats with hexosaminidase β-subunit deficiency

Allison M. Bradbury et al.

MOLECULAR GENETICS AND METABOLISM (2009)

Review Biochemical Research Methods

Re-engineering biopharmaceuticals for delivery to brain with molecular Trojan horses

William M. Pardridge

BIOCONJUGATE CHEMISTRY (2008)

Article Biotechnology & Applied Microbiology

GDNF fusion protein for targeted-drug delivery across the human blood-brain barrier

Ruben J. Boado et al.

BIOTECHNOLOGY AND BIOENGINEERING (2008)

Article Psychiatry

Depression, comorbidities and the TNF-α system

H. Himmerich et al.

EUROPEAN PSYCHIATRY (2008)

Article Endocrinology & Metabolism

Sanfilippo syndrome: A mini-review

M. J. Valstar et al.

JOURNAL OF INHERITED METABOLIC DISEASE (2008)

Article Biotechnology & Applied Microbiology

Genetic engineering of a lysosomal enzyme fusion protein for targeted delivery across the human blood-brain barrier

Ruben J. Boado et al.

BIOTECHNOLOGY AND BIOENGINEERING (2008)

Article Chemistry, Multidisciplinary

Intravenous siRNA of brain cancer with receptor targeting and avidin-biotin technology

Chun-Fang Xia et al.

PHARMACEUTICAL RESEARCH (2007)

Article Immunology

Biological and clinical effects of anti-TNFα treatment

G. Valesini et al.

AUTOIMMUNITY REVIEWS (2007)

Article Endocrinology & Metabolism

Murine model (Galnstm(C76S)slu) of MPS IVA with missense mutation at the active site cysteine conserved among sulfatase proteins

Shunji Tomatsu et al.

MOLECULAR GENETICS AND METABOLISM (2007)

Article Biochemistry & Molecular Biology

Neuronal ceroid lipofuscinoses therapeutic strategies: Past, present and future

Judith A. Hobert et al.

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2006)

Article Multidisciplinary Sciences

FGF-2 promotes neurogenesis and neuroprotection and prolongs survival in a transgenic mouse model of Huntington's disease

KL Jin et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)

Review Neurosciences

Motoneurons crave glial cell line-derived neurotrophic factor

MC Bohn

EXPERIMENTAL NEUROLOGY (2004)

Review Clinical Neurology

Enzyme-replacement therapy for metabolic storage disorders

RO Brady et al.

LANCET NEUROLOGY (2004)

Review Biochemistry & Molecular Biology

Gene therapy progress and prospects: gene therapy of lysosomal storage disorders

SH Cheng et al.

GENE THERAPY (2003)

Article Multidisciplinary Sciences

An anti-transferrin receptor-avidin fusion protein exhibits both strong proapoptotic activity and the ability to deliver various molecules into cancer cells

PP Ng et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)

Article Biochemistry & Molecular Biology

Stimulation of acid sphingomyelinase activity by lysosomal lipids and sphingolipid activator proteins

T Linke et al.

BIOLOGICAL CHEMISTRY (2001)