Adeno-associated virus (AAV)-based gene therapy products: What are toxicity studies in non-human primates showing us?

P Baldrick, B McIntosh, M Prasad - Regulatory Toxicology and …, 2023 - Elsevier
A number of adeno-associated virus (AAV)-based gene therapy products have entered
clinical development, with a few also reaching marketing approval. However, as our …

Current landscape and emerging opportunities of gene therapy with non-viral episomal vectors

SU Khan, MU Khan, MI Khan, F Kalsoom… - Current Gene …, 2023 - benthamdirect.com
Gene therapy has proven to be extremely beneficial in the management of a wide range of
genetic disorders for which there are currently no or few effective treatments. Gene transfer …

Intrathecal sc-AAV9-CB-GFP: systemic distribution predominates following single-dose administration in cynomolgus macaques

EK Meseck, G Guibinga, S Wang… - Toxicologic …, 2022 - journals.sagepub.com
Biodistribution of self-complementary adeno-associated virus-9 (scAAV9)–chicken β-actin
promoter–green fluorescent protein (GFP) was assessed in juvenile cynomolgus macaques …

An overview of nonclinical and clinical liver toxicity associated with AAV gene therapy

LO Whiteley - Toxicologic Pathology, 2023 - journals.sagepub.com
This article reviews the presentation given at the 2023 annual meeting of the Society of
Toxicologic Pathology (STP) on liver toxicity observed with adeno-associated viral vector …

Scientific and Regulatory Policy Committee technical review: biology and pathology of ganglia in animal species used for nonclinical safety testing

BM Bennet, ID Pardo, BT Assaf, E Buza… - Toxicologic …, 2023 - journals.sagepub.com
Dorsal root ganglia (DRG), trigeminal ganglia (TG), other sensory ganglia, and autonomic
ganglia may be injured by some test article classes, including anti-neoplastic …

Neuropathological findings in nonclinical species following administration of adeno-associated virus (AAV)-based gene therapy vectors

B Bolon, E Buza, E Galbreath, J Wicks… - Toxicologic …, 2024 - journals.sagepub.com
Adeno-associated virus (AAV) gene therapy vectors are an accepted platform for treating
severe neurological diseases. Test article (TA)-related and procedure-related …

Scientific and Regulatory Policy Committee points to consider: sampling, processing, evaluation, interpretation, and reporting of test article-related ganglion pathology …

BM Bennet, ID Pardo, BT Assaf, E Buza… - Toxicologic …, 2023 - journals.sagepub.com
Certain biopharmaceutical products consistently affect dorsal root ganglia, trigeminal
ganglia, and/or autonomic ganglia. Product classes targeting ganglia include antineoplastic …

Toxicologic neuropathology of novel biotherapeutics

DS Bangari, LG Lanigan, SD Cramer, JL Grieves… - 2023 - journals.sagepub.com
Biotherapeutic modalities such as cell therapies, gene therapies, nucleic acids, and proteins
are increasingly investigated as disease-modifying treatments for severe and life …

Pathology findings and in-life correlates in the nonclinical development of adeno-associated virus (AAV)-based retinal gene therapies

HS Booler, T Lejeune, O Turner… - Toxicologic …, 2024 - journals.sagepub.com
Adeno-associated virus (AAV)-based vectors are the most frequently used platform for
retinal gene therapy. Initially explored for the treatment of loss-of-function mutations …

Current Advances in Viral Nanoparticles for Biomedicine

X Sun, T Tian, Y Lian, Z Cui - ACS nano, 2024 - ACS Publications
Viral nanoparticles (VNPs) have emerged as crucial tools in the field of biomedicine.
Leveraging their biological and physicochemical properties, VNPs exhibit significant …