Juliette Hordeaux

Juliette Hordeaux

Company: University of Pennsylvania

Job title: Senior Director, Translational Research & Lysosomal Storage Disease

Seminars:

Q&A Panel Discussion: Overcoming Toxicity & Immunogenicity Challenges 8:55 am

• Does it depend on the disorder? • What are best preclinical models for toxicology risk assessment? • Reviewing preclinical data for CNS and systemic toxicity • How does this translate into humans? • Discussing immunogenicity concerns for patientsRead more

day: Day Two

Dorsal Root Ganglia Pathology in AAV Preclinical Studies: Meta-Analysis & Mitigation Strategy 8:05 am

• The administration of adeno-associated virus (AAV) vectors to nonhuman primates (NHP) can lead to dorsal root ganglion (DRG) pathology • A meta-analysis including 256 NHP shows that 83% of animals that were administered AAV through the cerebrospinal fluid (CSF), and 32% of those that received an intravenous (IV) injection developed some DRG pathology and…Read more

day: Day Two

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