Some of the challenges to develop gene therapies include identifying delivery methods to achieve appropriate biodistribution profiles, determining dose range, testing safety, and assessing efficacy using translationally relevant assays.
In this webinar, Neeraj Kumar Tiwari, Ph.D., Technical Information Scientist, discusses overcoming these challenges, specifically for neurodevelopmental and neurodegenerative diseases.
Topics covered include:
- Common gene therapy-based products tested in preclinical models and in clinical trials including ASO, AAV, and CRISPR-based therapeutics.
- Characterization of delivery methods to circumvent the blood brain barrier, using translational relevant assays unique to neurodevelopmental and neurodegenerative diseases.
- Sample study designs JAX In Vivo Pharmacology Services uses routinely for the preclinical assessment of gene-based therapeutic activity.