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Hi. This is Tineke
van den Hoorn.
Nonclinical assessor at
the Dutch Medicines
evaluation Board.
I will talk about ICH S12.
This is a guidance on
the Nonclinical
Biodistribution Considerations
specifically for gene
therapy product.
The final version of
this guideline was
adopted 14th of March 2023.
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I will guide you through
this guideline and I will do
that by the help
of the structure
of the guideline itself.
It's structured in six
parts or six chapters.
Here with this
slide I start with
the introduction
of the guidance.
The first slide is
on the objective.
The guidance
concerns non clinical
biodistribution studies
for gene therapy products.
It talks about the
overall design
of Nonclinical Biodistribution
assessments or the studies.
It deals with the
interpretation and
the application of the
biodistribution data and
this biodistribution
data supports
the nonclinical
development program
for the development of
the gene therapy product.
It also aids the design
of clinical trials.
Another objective
is the facilitation
of the development of
gene therapy products and
to consider 3R principles.
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The other part of the
introduction is about the scope.
Which products are in the
scope of this guidance?
That are gene therapy products
that mediate their effect
by the expression,
transcription or translation of
a transferred genetic material.
The genetic material can
either be purified nucleic
acids like plasmids and RNA
can be microorganisms
like viruses,
bacteria or fungi that
are genetically
modified to express
a transgene including products
that edit the host genome.
Gene editing which is done
via gene editing enzymes
and a guide RNA.
It might also include
ex vivo genetically
modified human cells
also products that are
intended to alter the
host cell genome in
vivo without specific
transcription or translation.
That means the
delivery of a nuclease
an editing enzyme and a guide
RNA by non viral methods
like a ibtnano particle and it
concerns also oncolytic
viruses that are not
genetically modified
per se but are intended
to edit the tumor micro
environment by being oncolytic.
Although not all countries or
all regions have oncolytic virus
classified as gene
therapy product.
