Gene Therapies Target Neurodegenerative Diseases

Published on: 
BioPharm International, BioPharm International-03-15-2021, Volume 2021 eBook, Issue 1
Pages: 12–13

A growing pipeline of gene therapy candidates aimed at treating neurodegenerative diseases has prompted new guidance.

Much of gene therapy development is targeting neurodegenerative diseases, among other difficult-to-treat indications. Responding to a growing pipeline of gene therapy candidates, FDA released a new draft guidance in January 2021, Human Gene Therapy for Neurodegenerative Diseases, focused on gene therapies being developed to treat neurodegenerative diseases. The new draft guidance focuses on product development, preclinical testing, and clinical trial design as well as discusses marketing approval pathways for investigational gene therapy products for neurodegenerative disease indications.

Read this article in BioPharm International's Regulatory Sourcebook March 2021 eBook.


Article Details

BioPharm International
eBook: Regulatory Sourcebook, March 2021
March 2021
Pages: 12–13


When referring to this article, please cite it as F. Mirasol, “Gene Therapies Target Neurodegenerative Diseases," BioPharm International Regulatory Sourcebook eBook (March 2021).