BioPharm International - March 2021

BioPharm International - Regulatory Sourcebook - March 2021

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12 BioPharm International eBook March 2021 Gene Therapies Target Neurodegenerative Diseases A growing pipeline of gene therapy candidates aimed at treating neurodegenerative diseases has prompted new guidance. M uch of gene therapy development is targeting neurodegenerative diseases, among other difficult-to-treat indica- tions. Responding to a growing pipe- line of gene therapy candidates, FDA released a new draft guidance in January 2021 (1) focused on gene therapies being developed to treat neurode- generative diseases. The new draft guidance focuses on product devel- opment, preclinical testing, and clinical trial design as well as discusses marketing approval pathways for investigational gene therapy products for neurodegen- erative disease indications. CMC CONSIDERATIONS The guidance for general chemistry, manufacturing, and controls (CMC), which take into account prod- uct manufacturing, testing, and release of gene ther- apy products for neurodegenerative diseases remain the same as those for other gene therapy products (2). But some aspects of gene therapy products for neu- rodegenerative diseases may require additional CMC considerations, such as challenges related to the route of administration, the volume of product that can be administered, the delivery device, and the study population size (1). The new draft guidance also recommends that the gene therapy product's critical quality attributes (CQAs) and critical process parameters (CPPs) be fully evaluated during the early clinical development phase and appropriate controls implemented. FDA's rea- soning is that, since early-phase clinical studies of neurodegenerative diseases often involve small study populations and the focus is on safety assessments, natali_mis - FELIZA MIRASOL Regulatory Sourcebook Quality

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