The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution
Over the past decade, in vivo gene replacement therapy has significantly advanced,
resulting in market approval of numerous therapeutics predominantly relying on adeno …
resulting in market approval of numerous therapeutics predominantly relying on adeno …
Role of FoxP3+ Regulatory T Cells in Modulating Immune Responses to Adeno-Associated Virus Gene Therapy
M Muñoz-Melero, M Biswas - Human Gene Therapy, 2024 - liebertpub.com
Adeno-associated virus (AAV) gene therapy is making rapid strides owing to its wide range
of therapeutic applications. However, development of serious immune responses to the …
of therapeutic applications. However, development of serious immune responses to the …
Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A
E Symington, S Rangarajan, W Lester, B Madan… - …, 2024 - Wiley Online Library
Introduction Valoctocogene roxaparvovec is an adeno‐associated virus vector serotype 5
(AAV5)‐mediated gene therapy approved for severe haemophilia A (HA). Aim To report the …
(AAV5)‐mediated gene therapy approved for severe haemophilia A (HA). Aim To report the …
[HTML][HTML] Immune responses to central nervous system directed adeno-associated virus gene therapy: Does direct CNS delivery make a difference?
AL Harkins, PP Ambegaokar, AM Keeler - Neurotherapeutics, 2024 - Elsevier
Adeno-associated virus (AAV) mediated gene therapy is a leading gene delivery platform
with potential to transform the landscape of treatment for neurological disorders. While AAV …
with potential to transform the landscape of treatment for neurological disorders. While AAV …
Essential role of pre-existing humoral immunity in TLR9-mediated type I IFN response to recombinant AAV vectors in human whole blood
NS Alakhras, CA Moreland, LC Wong, P Raut… - Frontiers in …, 2024 - frontiersin.org
Recombinant adeno-associated virus (AAV) vectors have emerged as the preferred platform
for gene therapy of rare human diseases. Despite the clinical promise, host immune …
for gene therapy of rare human diseases. Despite the clinical promise, host immune …
Clinical Pharmacology Perspective on Development of Adeno‐Associated Virus Vector‐Based Retina Gene Therapy
JL Ford, E Karatza, H Mody… - Clinical …, 2024 - Wiley Online Library
Adeno‐associated virus (AAV) vector‐based gene therapy is an innovative modality being
increasingly investigated to treat diseases by modifying or replacing defective genes or …
increasingly investigated to treat diseases by modifying or replacing defective genes or …
Human cell surface-AAV interactomes identify LRP6 as blood-brain-barrier transcytosis receptor and immune cytokine IL3 as AAV9 binder
Adeno-associated viruses (AAVs) are foundational gene delivery tools for basic science and
clinical therapeutics. However, lack of mechanistic insight, especially for engineered vectors …
clinical therapeutics. However, lack of mechanistic insight, especially for engineered vectors …
Immunogenicity Risk Assessment of Process-Related Impurities in An Engineered T Cell Receptor Cellular Product
J Mora, D Forman, J Hu, A Ijantkar, J Gokemeijer… - Journal of …, 2024 - Elsevier
Cell therapies such as genetically modified T cells have emerged as a promising and viable
treatment for hematologic cancers and are being aggressively pursued for a wide range of …
treatment for hematologic cancers and are being aggressively pursued for a wide range of …
Development of an Enzyme-Linked Immunosorbent Spot Assay for the Assessment of Adeno-Associated Virus Peptides to Examine Immune Safety
SR Krivoshik, L Dzielak, AR Masters, J Hall… - Human Gene …, 2024 - liebertpub.com
Adeno-associated virus (AAV)-based gene therapies have shown promise as novel
treatments for rare genetic disorders such as hemophilia A and spinal muscular atrophy …
treatments for rare genetic disorders such as hemophilia A and spinal muscular atrophy …
2023 White Paper on Recent Issues in Bioanalysis: ISR for ADA Assays, the Rise of dPCR vs qPCR, International Reference Standards for Vaccine Assays, Anti-AAV …
J Mora, R Palmer, L Wagner, B Wu, M Partridge… - Bioanalysis, 2024 - Taylor & Francis
The 17th Workshop on Recent Issues in Bioanalysis (17th WRIB) took place in Orlando, FL,
USA on June 19–23, 2023. Over 1000 professionals representing pharma/biotech …
USA on June 19–23, 2023. Over 1000 professionals representing pharma/biotech …