So there's a large number
of diseases
across many TAs
that are being considered
for gene therapy.
However, the most activity
that's being observed
and perhaps where it's
gonna be most relevant
in the near term
is for orphan diseases
that are mediated
by specific mutations.
So in terms of challenges
people are facing,
there's the traditional
development pathway challenges.
Some of these gene therapies
have been associated
with side effects
that need to be balanced
and make sure people know
how to manage them
or perhaps eliminate it
as technology
continues to evolve.
There's the challenge
of actually demonstrating
an efficacy benefit
for the patient
from a pharmaceutical
development perspective,
and pretty much all molecules
including small molecules
and biologics face that.
What's new with gene therapy
is that we may be--
the whole value proposition
really depends
on a single treatment
that leads to a functional cure.
So that means patients
don't get redosed.
Oftentimes, most pharma models
are based on patients
treated repeatedly
in a particular cadence
every day,
every couple weeks, every month,
every couple months.
With gene therapy,
it could be one time.
Gene therapy's gonna require
technical innovation,
but in all likelihood,
it'll require innovation
with business models.
And by that
what we mean is
that companies are gonna need
to think about
how they're gonna engage
with regulatory agencies,
ministries of health,
payers at the end of the day
to try to drive the alignments.
The single upfront lump sum
payment's an alternative,
that's the way it works today
for every dose.
Whether payers
and other agencies
are gonna want to make
that commitment
given the limited track record
remains to be seen,
and maybe they're willing
to offer a price
but pharmaceutical companies
are gonna want
to extract more value,
if at all possible,
if they're in fact
delivering a cure
for a disease that may cause
$400,000, $500,000 a year
in the U.S. to manage
a particular patient.
From our perspective,
gene therapy
is probably here to stay.
It's starting to reach
the market,
starting to reach
late stage development,
there's a lot of excitement.
Investors, pharma companies,
many players in the space
are following
very, very closely
and the reason for that is
it has a huge potential
in terms of the impact
it could have with patients.
So that is
tremendous opportunity
for patients
at the end of the day,
but it's a new modality that has
very unique circumstances
associated with it,
in particular,
this whole concept
of potential functional cure
after one dose
which is gonna be
a very different issue
for pharma companies and payers
and other stakeholders
to deal with.
So with that excitement
there's gonna be some challenges
as to how do we make gene
therapies work for everybody
from a clinical patient
commercial perspective
and that's where a lot
of the innovation's
gonna happen going forward.
