Overcoming Challenges in Preclinical Gene Therapy Testing for Neurodevelopmental and Neurodegenerative Diseases

Summary

Developing gene therapies for neurodevelopmental and neurodegenerative disorders presents unique challenges that vary by disease. The Jackson Laboratory (JAX) maintains the world’s largest portfolio of genetically humanized neuro mouse models and has extensive expertise in conducting FDA-aligned studies of biodistribution, efficacy, and safety testing using these models.

In this webinar, scientists from JAX’s In Vivo Preclinical Neuro Services team will discuss how our well-characterized, clinically relevant models and custom research assays can help you overcome key challenges in gene therapy development. They’ll share case studies demonstrating how JAX’s platforms have successfully supported gene therapy programs from early discovery through IND-supporting studies.

Topics covered will include:

• Key phenotypic and molecular biomarkers used in model selection and efficacy assessment
• Vector administration routes—IV, ICV, IT, and IP—and their implications for CNS targeting
• Biodistribution analysis across CNS and peripheral tissues using qPCR and immunohistochemistry
• Sample in vivo study designs demonstrating vector efficacy and therapeutic impact
• The role of early iPSC-derived neural cell assays in preclinical gene therapy discovery

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