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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