Placeholder Banner

BIO Comments on FDA’s Draft Guidance on Human Gene Therapy for Neurodegenerative Diseases

April 6, 2021

On Tuesday, April 6th, BIO submitted comments on the U.S. Food and Drug Administration (FDA)’s January 2021 draft industry guidance on “Human Gene Therapy for Neurodegenerative Diseases.” In its comments, BIO stated that while the guidance covers many critical development issues for gene therapies, it is not specific to gene therapies for neurodegenerative diseases, and further requests that the Agency consider the potential impact of this guidance on gene therapy programs outside of neurodegenerative diseases. To this end, BIO encourages the issuance of (1) broad guidance on gene therapies that includes information that is applicable across diseases and (2) publication of focused and brief disease-specific guidance to provide information to Sponsors on disease-specific issues, as appropriate. Since aspects of this guidance may be relevant to cell therapies, BIO recommends that FDA develop a guidance or Q&A document that is quickly and easily updated, and consolidates cell and gene therapy policy with broad applicability into a single document. In the full comment letter, BIO also provides specific comments on the following topics: Gene Therapies and Accelerated Approval, Preclinical Studies, Chemistry Manufacturing and Controls (CMC), and Clinical Studies. 

Download Full Comments Below
BIO Comments on FDA’s Draft Guidance on Human Gene Therapy for Neurodegenerative Diseases
Discover More
On Thursday, June 10th, 2021, the U.S. Food and Drug Administration (FDA) published a draft guidance, Core Patient-Reported Clinical Outcomes in Cancer Clinical Trials, providing recommendations to sponsors on the collection of a core set of patient…
The Biotechnology Innovation Organization (BIO) is the world's largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30…
On Wednesday, July 14th, BIO submitted comments on FDA’s Guidances: 1) Remote Interactive Evaluations of Drug Manufacturing and Bioresearch Monitoring Facilities During the COVID-19 Public Health Emergency Guidance; 2) Updated Manufacturing, Supply…