Consideration for Program Design in Nonclinical Development of Gene Therapy
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Overview of Gene Therapy with a focus on AAV based therapies. We will look at major differences in preclinical safety evaluations, such as small molecule vs gene therapy. Some of the key study components will be reviewed with the audience for knowledge of FDA requirements to get to an IND approved study design. Gene editing, biodistribution assay development, along with updates and program designs in the history of preclinical development in 15 years of gene therapy at Lovelace Biomedical.
How are preclinical programs run for gene therapy?
What does FDA require to approve your study?
What is the history of gene therapy and updates on running studies in the field?
How does it all come together to get to the IND stage?