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24th Biotechnology Congress: Research & Innovations
CRISPR Cas9 Technology and Genetic Engineering
Annual Congress on
October 24-25, 2018 | Boston, USA
Journal of Biotechnology & Biomaterials | ISSN: 2155-952X | Volume: 8
Implications of cGMP for CRISPR-Cas9 cellular therapy
Kimberley Buytaert-Hoefen
Parexel International, USA
Many current therapeutic treatments are not able to address the underlying cause of a disease, alter its course, or reverse
the damage that has already occurred. Cellular therapies offer the power of the human body to heal and regenerate itself.
Regulatory precedents for cellular therapy products continue to evolve for a widening array of the product of types. An exciting
newdiscovery of clustered regularly interspaced short palindromic repeat (CRISPR) technologywhen incorporatedwith cellular
therapy may lead to the cure of many diseases. CRISPR-Cas9 offers the hope of a cure for various maladies including genetic
diseases and cancers. The CRISPR-Cas9 medical cellular therapies involve removing cells from the body, modifying their DNA,
and administering them to the patient. These modified cells are able to either replace or attack diseased cells. Medical cellular
therapies are required to demonstrate quality, safety, and efficacy standards to obtain a marketing authorization. Medicinal
cellular therapy products are regulated as drugs, devices, and biological products, which adds the regulatory requirement of
manufacturing under cGMP conditions. With the high value of CRISPR-Cas9 source cell material, having ample amount for
process-development and validation of the manufacturing processes is an industry challenge. Furthermore, limited shelf life
and quantity of cells can complicate quality control testing and stability determinations. Defining critical quality attribute’s
(CQA’s) for these products and developing assays for their potency are essential to the commercialization of these cellular
therapy products. CRISPR-Cas9 source cells are characterized based on the presence of surface markers, size, and combinations
of attributes associated with cell source and mode of action. Due to their ability to alter DNA, CRISPR-Cas9 cellular therapies
offer the possibility to move beyond conventional disease treatment by addressing the underlying cause of disease, altering its
course, or reversing the damage that has already occurred. The transitions from discovery, to research and development, to
commercially manufactured products, brings the challenge of the regulatory requirements for incorporating cGMPs into the
collection, production, and delivery of these products. These developments will allow for CRISPR-Cas9 cellular therapy to
become increasingly available to patients and will offer new treatments and the hope to cure many diseases.
Biography
Buytaert-Hoefen obtained a Bachelor’s degree in Psychology at the State University of New York at Binghamton and then went on to complete her Master’s and
Doctorate degrees in Neuroscience at the University of Colorado at Boulder. She completed two post-doctoral fellowships at the University of Colorado Health
Sciences Center where she specialized in embryonic and adult stem cell research. She then entered the industry with a position as a Lead Scientist at Navigant
Biotechnologies. After which, she accepted a position as a Consumer Safety Officer at the FDA, where she specialized in pharmaceutical inspections with an
emphasis on biotechnology and sterile processing. Currently, as a Consultant at Parexel, she works closely with clients to develop and implement effective
compliance solutions in accordance with client needs. She performs GXP audits, conducts laboratory data review including chemistry, microbiology, and data
integrity assessments.
Kimberley Buytaert-Hoefen, J Biotechnol Biomater 2018, Volume 8
DOI: 10.4172/2155-952X-C4-097