12 BioPharm International eBook March 2021 www.biopharminternational.com
Gene Therapies Target
Neurodegenerative Diseases
A growing pipeline of gene therapy candidates aimed at
treating neurodegenerative diseases has prompted new guidance.
M
uch of gene therapy development is
targeting neurodegenerative diseases,
among other difficult-to-treat indica-
tions. Responding to a growing pipe-
line of gene therapy candidates, FDA
released a new draft guidance in January 2021 (1) focused
on gene therapies being developed to treat neurode-
generative diseases.
The new draft guidance focuses on product devel-
opment, preclinical testing, and clinical trial design
as well as discusses marketing approval pathways for
investigational gene therapy products for neurodegen-
erative disease indications.
CMC CONSIDERATIONS
The guidance for general chemistry, manufacturing,
and controls (CMC), which take into account prod-
uct manufacturing, testing, and release of gene ther-
apy products for neurodegenerative diseases remain
the same as those for other gene therapy products (2).
But some aspects of gene therapy products for neu-
rodegenerative diseases may require additional CMC
considerations, such as challenges related to the
route of administration, the volume of product that
can be administered, the delivery device, and the
study population size (1).
The new draft guidance also recommends that
the gene therapy product's critical quality attributes
(CQAs) and critical process parameters (CPPs) be fully
evaluated during the early clinical development phase
and appropriate controls implemented. FDA's rea-
soning is that, since early-phase clinical studies of
neurodegenerative diseases often involve small study
populations and the focus is on safety assessments,
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FELIZA MIRASOL
Regulatory Sourcebook Quality
