2 / 8
Page 12
Notes:
August 17-18, 2017 | Toronto, Canada
ANNUAL BIOTECHNOLOGY CONGRESS
allied
academies
Ann Biol Sci, 2017
ISSN: 2348-1927
A
vailability of synthetic mRNAs enabled progress in their
applications. Tremendous interest of private investors and
PHARMA has created a billion € business. AmpTec supports
new players by providing customized, high quality mRNA
products. Important features, technical options for high-
amount, high-quality mRNA synthesis and GMP-compliant
manufacturing will be presented. Specific mRNA features will
be presented for diverse applications like (i) mRNA-directed
expression of antigens in dendritic cells for vaccination projects
in oncogenesis, infectious disease and allergy prevention; (ii)
reprogramming of adult cells to induced pluripotent stem
cells with their subsequent differentiation to the desired cell
type; (iii) applications in gene therapy. A recent overview has
summarised applications and syn-mRNA quality requirements.
Syn-mRNAs can be generated by
in vitro
transcription (IVT) from
defined templates containing the synthetic gene of interest. In
principle, linearised plasmids (with a restriction enzyme) can
be used directly as templates in IVT reactions, However, this
procedure is hampered by several disadvantages: incomplete
plasmid cleavage results in variable amounts of very long and
undefined background transcripts; high amounts of plasmid
DNA introduce undesired bacterial components. Furthermore,
optimal mRNA activity depends on a very long, unmasked
poly(A) tail, like 120 A. However hompolymeric repeats
are prone to random deletions/elongations during plasmid
propagation inbacteria. Insteadof plasmids,weusewell defined
PCR-products as IVT-templates. This approach with examples
will be shown. Technical problems in IVT-based mRNA synthesis
and problem-solutions will be presented, plus a detailed list of
quality requirements for GMP-compliant synthetic mRNAs.
Speaker Biography
Guido Krupp, PhD, is the CEO and President of AmpTec GmbH. In 1981, he received PhD
degree from Würzburg University & Max-Planck-Institute Martinsried. From 1983 to
1987, he was a Post-doc at Yale University. From 1987 to 2002, he worked as Research
Group Leader at Kiel University. He is also the Founder of Artus GmbH (1998) & AmpTec
GmbH (2005) & KSK Diagnostics GmbH (2015). His primary area of research includes
nucleic acid technology with focus on RNA, plant pathogens (viroids), ribozymes and
telomerase. He has more than 60 publications, Editor of
Ribozyme Biochemistry &
Biotechnology
, and of
Telomeres, Telomerases & Cancer
, Editorial Board Member of
Biotechnology Annual Review
.
e:
krupp@amp-tec.comGuido Krupp
AmpTec GmbH, Germany
Synthetic mRNAs as optimised tools for stem cell generation and for manipulating
cellular phenotypes
Guido Krupp, Ann Biol Sci, 2017, 5:3
DOI: 10.21767/2348-1927-C1-001