Human gene therapy for neurodegenerative diseases: guidance for industry

Human gene therapy for neurodegenerative diseases: guidance for industry

Collection:
Health Policy and Services Research
Contributor(s):
United States. Department of Health and Human Services, issuing body.
United States. Food and Drug Administration, issuing body.
Center for Biologics Evaluation and Research (U.S.), issuing body.
Publication:
Silver Spring, MD : Center for Biologics Evaluation and Research, October 2022
Language(s):
English
Format:
Text
Subject(s):
Clinical Studies as Topic
Genetic Therapy -- legislation & jurisprudence
Government Regulation
Neurodegenerative Diseases -- therapy
United States
United States. Food and Drug Administration
Genre(s):
Guideline
Technical Report
Abstract:
This guidance provides recommendations to sponsors developing human gene therapy (GT) products for neurodegenerative diseases affecting adult and pediatric patients. Neurodegenerative diseases are a heterogeneous group of disorders characterized by progressive degeneration of the structure and function of the central nervous system or peripheral nervous system. These diseases vary in etiology, prevalence, diagnosis, and management, and include genetic as well as age-related diseases. 2 This guidance focuses on considerations for product development, preclinical testing, and clinical trial design. This guidance finalizes the draft guidance of the same title dated January 2021. In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidances means that something is suggested or recommended, but not required.
Copyright:
The National Library of Medicine believes this item to be in the public domain. (More information)
Extent:
1 online resource (1 PDF file (i, 16 pages))
NLM Unique ID:
9918504488006676 (See catalog record)
Permanent Link:
http://resource.nlm.nih.gov/9918504488006676