Gene therapy has emerged as one of the
most promising therapeutic fields in recent years, particularly for
neurodegenerative disorders. The GENOV Innovation Unit at the Paris Brain
Institute is dedicated to advancing gene and cell therapy strategies for conditions
such as ALS, Parkinson's disease, and Alzheimer's disease, as well as rare
lysosomal storage disorders, including mucopolysaccharidosis and metachromatic
leukodystrophy. With a strong translational focus, the team develops therapies
from early proof-of-concept studies through large animal models and into
clinical applications. Their work involves comparing and optimizing multiple
AAV vectors, as well as evaluating different routes of administration. To generate
robust data for regulatory submissions, they require highly reproducible
analysis processes, particularly for biodistribution and RNA expression readouts.
Join us for this
webinar, where featured speakers from the GENOV Innovation Unit will share how
they established a streamlined analysis pipeline by integrating the Maxwell®
automated DNA/RNA extraction system to enhance reproducibility. Accurate
biodistribution assessment is crucial in the development of gene therapy. It confirms
that therapeutic vectors reach the intended target tissues or cells, informs
dosing to achieve the right balance of efficacy and safety, and provides
essential evidence that links gene presence to biological effects. Moreover,
precise biodistribution data are required for regulatory approval, help
mitigate long-term risks such as insertional mutagenesis, and ultimately
support the safe and effective translation of gene therapies to patients.
In this webinar, you will learn about:
- Advances in gene and cell
therapy for neurodegenerative and rare disorders
- How the Maxwell® automated
nucleic acid extraction system supports reproducible biodistribution and
RNA expression analysis
Insights into how
accurate biodistribution ensures evaluation of treatment safety, efficacy, and
regulatory compliance.
Thursday,
2 October, 2025
Watch The Webinar
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