
FDA Gives Guidance on Gene Therapy for Neurodegenerative Diseases
The draft guidance document offers developers of these treatments information regarding product development, preclinical testing, and clinical trial design.
On Jan. 5, 2021, FDA published
Specifically, the guidance addresses chemistry, manufacturing, and controls (CMC) considerations and states aspects of GT products for neurodegenerative diseases may have particular CMC considerations. The guidance emphasizes product design should be based on current scientific knowledge and GT products should be designed to reduce inflammatory immune responses. Considerations for drug product purity, identity, and strength are also emphasized.
The guidance also discusses aspects of preclinical studies, clinical trials, expedited programs, and communications with FDA.
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