Takeda eBook - 10
protein that you are producing theoretically can have
post-translational modifications. I do not think we have
gotten to that complexity or understanding of that
complexity in vivo.
Mike Baratta: Just trying to express either a protein
that is in some mutated form and replacing it, or produce
a functional enzyme. We are just focusing on that, and
have not gone to the area of how the post-translational
modifications might affect that type of drug product.
" I think there is an emerging opportunity
of incorporating PTMs into a CDx space.
I think there may be one opportunity
where a mass spec platform has been
used as a CDX platform to support a
companion diagnostic."
- Mike Baratta
Chris Anderson: If we are talking about the development of these therapeutics, and we are talking about rare
the precision medicine development. Fully understand-
diseases and precision medicine, if you will, can anyone
ing the nature of PTMs for the individual patient is one
on the panel talk about how a better understanding of
of the most important steps to find the most suitable
PTMs as biomarkers can drive precision medicine in the
treatment.
future?
Chris Anderson: We have talked about understanding
Stephen Zicha: One of the strategies many companies
a disease, disease progression, and the development of
are employing now in the dementia field is trying to
new therapeutics to go along with that. How might the
go earlier and earlier on in terms of diagnosis. But,
expanding knowledge of post-translational modifica-
like I mentioned, a lot of these can only be confirmed
tions as biomarkers be incorporated into development
postmortem. So if you have the ability to actually define
of clinical diagnostics, post-approval, to help select the
a disease by a specific post-translation modified version
most appropriate therapy for individual patients?
of Tau and diagnose it earlier on, I think that speaks to
precision medicine.
Stephen Zicha: Well it is sort of unprecedented. It
definitely adds an additional layer of things that we as
Karen Cao: In order to explore more, tools such as
pharmaceutical companies should be looking out for
analytical technology, software, bioinformatics are still
as an opportunity to develop a companion diagnostic
the keys. Since almost all proteins in the human body are
or a clinical diagnostic. We started off talking just about
directly affected by some form of PTM and they are all
the intrinsic protein, if you will, and just being able to
unique. PTMs may play a role on precision medicine. For
develop sensitive and quantifiable assays to measure
individual rare diseases, besides checking patient's intact
that. Now you throw on the post-translational modifica-
DNA, RNA and protein, we may check the corresponding
tions, and that is an additional layer that we should be
PTM forms as well. Once PTM biomarker is identified,
aware of. I have not seen it done in practice, but there
just like all the other biomarkers, we can use it in drug
definitely are opportunities.
development.
Mike Baratta: I think there is an emerging opportunity
Moucun Yuan: If we know the individual pattern for
of incorporating PTMs into a CDx space. I think there
the PTM, we can design a therapy to target the biolog-
may be one opportunity where a mass spec platform has
ical disorder via the dysfunctions caused by different
been used as a CDx platform to support a companion
protein PTMs. This may result in better outcomes for
diagnostic. Another point is the cost associated with it
10
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Takeda eBook
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