Human Gene Therapy - April 2023 - 271

SHORT COURSE IN AAV MANUFACTURING
271
sionals and university students. Instructors with at least
some experience working in the biopharmaceutical industry
are needed.
CONCLUSIONS AND FUTURE
OPPORTUNITIES
BTEC has developed and delivered a hands-on
course (Hands-On cGMP Biomanufacturing of Vectors
for Gene Therapy) that covers numerous topics related
to viral vector manufacturing. These topics include an
overview of gene therapy products, fundamental concepts
underlying the operations used to manufacture
these products (both upstream and downstream), process
parameters that must be controlled, assays used for
product intermediate and drug product testing, and
validation of these assays. Since its inception in May
2019, nearly 80 participants have attended the course's
seven offerings. Postcourse evaluations show that
overall participant satisfaction with the program is high
and that the course provided participants with the information
and skills needed to effectively carry out their
jobs. Demand for the course remains high, and BTEC
will continue to offer it as part of its professional development
program in the foreseeable future.
Because of BTEC's success with the course, the gene
therapy training network (GTTN) has been initiated with
support from the National Institute for Innovation in
Biopharmaceutical Manufacturing (NIIMBL) to organize
efforts to transfer this and other gene therapy courses to
other institutions. To date, the Hands-On cGMP Biomanufacturing
of Vectors for Gene Therapy course has
been transferred to the National Center for Therapeutic
Manufacturing (NCTM) at the Texas A&M Engineering
Experiment Station. They piloted the course in October
2021 and have since offered it to 25 participants, with
more than 100 participants anticipated to take the course
(or an abbreviated lecture-only version) by the end of
2022. Similar to the BTEC version of the course, the
response to the NCTM program has been extremely
positive.
Both BTEC and NCTM continue to work with partners
interested in joining GTTN to exchange course content on
topics related to viral vector manufacturing. Furthermore,
BTEC has also incorporated elements of the course into its
academic programs for undergraduates and graduate students
to prepare them for opportunities in the rapidly expanding
gene therapy industry.
Finally, we believe that more focused training in any of
the areas covered in this course is warranted. BTEC has
recently completed the development and initial delivery of
a course titled Analytical Methods for Gene Therapy
Vector Characterization and Testing, which focuses on
analytical methods for viral vector process intermediate
and final product testing. Likewise, individual courses
that look specifically at upstream or downstream processing,
including the design and development of these
processes, are needed and are likely to see high demand
based on our experience in professional development
programs. Based on the feedback BTEC received from
course participants, we believe that training on the topic
of plasmid design and plasmid production (required for
transient transfection to produce viral vectors) would
also be of great interest.
ACKNOWLEDGMENTS
The authors thank Thomas Parker, formerly with
MilliporeSigma and now at BridgeBio, who worked with
BTEC to develop the depth filtration laboratory described
here, Chris Cummings for support in delivering
the course, and transferring it to NCTM, Suleiman
Sweilem for support during the course delivery and
Brian Mosley (now at BridgeBio) for his contribution to
developing and delivering the analytical portion of the
course.
AUTHORS' CONTRIBUTIONS
L.O.: Conceptualization (equal); methodology (equal);
investigation (equal); and writing-original draft (equal).
C.B.: Conceptualization (supporting); investigation
(equal); and writing-review and editing (equal). S.S.:
Investigation (equal) and writing-review and editing
(equal). C.S.-M.: Conceptualization (equal); methodology
(equal); investigation (equal); and writing-original draft
(equal). J.B.: Funding acquisition (supporting); project administration
(equal); and writing-original draft (supporting).
D.S.: Project administration (equal) and writing-original
draft (supporting). G.G.: Funding acquisition (lead); conceptualization
(equal); methodology (equal); supervision
(lead); and writing-original draft (equal).
ETHICS STATEMENT
Development of this course did not include studies on
human subjects, human data or tissue, or animals.
AUTHOR DISCLOSURE
No competing financial interests exist.
FUNDING INFORMATION
The development of this course was supported by
a Project Award Agreement from the National Institute for
Innovation inManufacturing Biopharmaceuticals (NIIMBL)
and financial assistance award 70NANB17H002 from the
U.S. Department of Commerce, National Institute of Standards
and Technology. Course development also benefited
from the financial support of the North Carolina Biotechnology
Center.
Human Gene Therapy - April 2023

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