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Q&A Genetic Modification .pdf

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Q&A: Genetic Modification

Express Yourself
Oxford Genetics and EBR team up to discuss cell engineering, genetic modification,
protein production and the next big breakthroughs in preclinical research
EBR: Oxford Genetics offers
numerous services in preclinical
R&D. Can you tell us a little about
some of these?
Paul Brooks: Oxford Genetics is a
synthetic biology company that
offers products and services to
support the discovery, development
and production of biologics, gene and
cell therapies. We have tremendous
expertise in designing DNA,
optimising expression of proteins,
improving viral delivery systems and
cell line development.
In your opinion, what is the
significance of the research
undertaken at Oxford Genetics
for the wider patient base, and
how does it contribute to the
production of new and
efficacious vaccines?
Oxford Genetics is addressing some of
the fundamental problems affecting
the biopharmaceutical industry in
relation to maximising productivity.
We are helping to achieve cost-effective
production of material for clinical
testing and commercial manufacture
from smaller batch sizes, which offers
a viable route to market for therapies
that might not otherwise make it.
The advent of CRISPR-Cas9 is
ground-breaking. Do you use this
technology and, if so, how has it
optimised your studies?
Our scientific team has extensive
experience in applying genomeediting technologies for cell line
engineering. We now intend to use
this technology to enhance and
refine our existing cellular models
for both protein production and
viral manufacture.


Ryan Cawood is Chief Executive Officer
and founder of Oxford Genetics. He
established the company in 2011 with
the aim to revolutionise DNA design,
optimisation and assembly in order to
enable molecular engineering to fulfil its
promise in synthetic biology and medicine.
Ryan’s research interests include vaccines
and treatments for infectious diseases
and cancer. He was inspired to create
Oxford Genetics after observing that
inefficient DNA design and assembly
often limit scientific progress. Ryan has
a degree in Genetics and a PhD from
Oxford University, UK.

Paul Brooks is Commercial Director at
Oxford Genetics. He recently joined the
company from Sigma-Aldrich, where he
was responsible for the commercialisation
of their functional genomic technologies –
including those for genome-editing. Over
the course of his career, Paul has held
various senior management positions in
both the US and Europe, and has overseen
the development, marketing and sales
of genomic technologies, products and
services. He holds a PhD in Molecular
Biology from the University of Manchester,
UK, and an MBA from the University of
Nottingham, UK.

Which other genetic modification
tools do you utilise at Oxford
Genetics, and how are the
data gathered from these
experiments employed?

In your opinion, why has geneediting been subject to so much
recent R&D?

Being a synthetic biology company, we
utilise a wide variety of molecular biology
techniques to ‘genetically modify’ cell lines.
A core aspect of our business is DNA design
and synthesis. Therefore, we regularly
introduce fully synthetic genes into the
genome of cell lines for the transient or
stable expression of recombinant proteins.

Ryan Cawood: Genome-editing has
been following the traditional product
lifecycle. It began more than a decade
ago with meganucleases, zinc finger
nucleases, transcriptor activator-like
effector nucleases and, now, we have
CRISPR. What we are currently witnessing
is the mass adoption that has been made
possible by the removal of the largest
barrier – namely price.

April 2017

Do you undertake any additional
research, such as protein
optimisation? How important
are your custom projects?

can overcome the challenges that
our clients face. The expertise gained
from these custom projects helps us to
further accelerate that process.

Oxford Genetics continues to invest
heavily in R&D to make improvements
to the production of biotherapeutics
– the optimisation of proteins is such
an area.

Based on your current knowledge,
will it soon be possible to cure
even inherited diseases?

By applying synthetic biology in a
streamlined workflow – which we
have refined over several years – we

If the inherited disease is solely due to
a mutated gene sequence that can be
replaced or repaired with the normal
sequence in the cells of the patient,
then yes: in theory, a cure is possible.

The challenge is that inherited diseases
like these represent only a small
number of the total.
What are some of the related
advances you anticipate in the
next few years?
The next big breakthroughs will come
from innovation in delivery, which will
permit more precise cellular targeting.
This would reduce toxicity from large
dose regimes, as well as diminish the
potential for off-target effects.

If the inherited disease is solely due to a mutated gene sequence that
can be replaced or repaired with the normal sequence in the cells of the patient,
then yes: in theory, a cure is possible



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