FDA Guidance on Preclinical Proof-of-Concept Studies for Human Gene Therapy for Neurodegenerative Disease
Preclinical evidence to support a prospect of direct benefit is most important when clinical evidence of effectiveness is not available from adult subjects with the same disease. Further details for general considerations in preclinical studies of these investigational GT products are available in a separate guidance document. The Food and Drug Administration issued draft guidance for "Human Gene Therapy for Neurodegenerative Diseases" for the development of a preclinical program for an investigational GT product intended for the treatment of neurodegenerative disease.
A preclinical program that is tailored to the investigational product and planned early-phase clinical trial contributes to the characterization of the product’s benefit/risk profile for the intended patient population. The overall objectives of a preclinical program for a GT product include: