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 …

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 …

Dorsal root ganglion toxicity after AAV intra-CSF delivery of a RNAi expression construct into non-human primates and mice

ZCE Hawley, ID Pardo, S Cao, MI Zavodszky, F Casey… - Molecular Therapy, 2025 - cell.com
Dorsal root ganglion (DRG) toxicity has been consistently reported as a potential safety
concern after delivery of adeno-associated viruses (AAVs) containing gene-replacement …

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 …

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 …

Toxicologic Pathology Forum Opinion: Rational Approaches to Expanded Neurohistopathology Evaluation for Nonclinical General Toxicity Studies and Juvenile …

B Bolon - Toxicologic Pathology, 2023 - journals.sagepub.com
Existing nervous system sampling and processing “best practices” for nonclinical general
toxicity studies (GTS) were designed to assess test articles with unknown, no known, or well …

Toxicologic Pathology Forum: Opinion on Interpretive Challenges for Procedure-Related Effects Associated With Direct Central Nervous System Delivery of …

LD Berman-Booty, SK Klein, C Mazur… - Toxicologic …, 2023 - journals.sagepub.com
Direct delivery of therapeutics to the central nervous system (CNS) greatly expands
opportunities to treat neurological diseases but is technically challenging. This opinion …