Expert Opinion on Biological Therapy,
Journal Year:
2022,
Volume and Issue:
22(9), P. 1193 - 1208
Published: Sept. 2, 2022
Ocular
gene
therapy
represents
fertile
ground
for
rapid
innovation,
with
ever-expanding
therapeutic
strategies,
molecular
targets,
and
indications.Potential
indications
ocular
have
classically
focused
on
inherited
retinal
disease
(IRD)
but
more
recently
include
acquired
diseases,
such
as
neovascular
age-related
macular
degeneration,
geographic
atrophy,
diabetic
retinopathy.
strategies
proliferated
recently,
augmentation,
inactivation,
editing,
RNA
modulation,
gene-independent
augmentation.
Viral
vector
constructs
adeno-associated
virus
lentivirus
continue
to
evolve
through
directed
evolution
rationale
design.
administration
techniques
expanded
beyond
pars
plana
vitrectomy
subretinal
injection
intravitreal
suprachoroidal
injection.The
success
of
treatment
IRD,
paired
the
promise
clinical
research
in
diseases
techniques,
has
raised
possibility
in-office
common
disorders
within
next
5
10
years.
European Journal of Ophthalmology,
Journal Year:
2024,
Volume and Issue:
34(6), P. 1631 - 1641
Published: March 18, 2024
Inherited
Retinal
Dystrophies
(IRD)
are
diverse
rare
diseases
that
affect
the
retina
and
lead
to
visual
impairment
or
blindness.
Research
in
this
field
is
ongoing,
with
over
60
EU
orphan
medicinal
products
designated
therapeutic
area
by
Committee
for
Orphan
Medicinal
Products
(COMP)
at
European
Medicines
Agency
(EMA).
Up
now,
COMP
has
used
traditional
disease
terms,
like
retinitis
pigmentosa,
designation
regardless
of
product's
mechanism
action.
The
reviewed
approach
IRDs
taking
into
account
all
previous
Designations
(OD)
experience
IRDs,
most
relevant
up
date
scientific
literature
input
from
patients
clinical
experts.
Following
review,
decided
there
should
be
three
options
available
concerning
condition:
i)
an
amended
set
OD
groups
therapies
might
a
broad
spectrum
conditions,
ii)
gene-specific
targeted
therapies,
iii)
occasional
term
do
not
fit
above
two
categories.
change
caters
different
scenarios
allow
optimum
future
applications
including
option
designation.
By
applying
new
approach,
increases
regulatory
clarity,
efficiency,
predictability
sponsors,
aligns
tools
latest
medical
developments
ensures
potentially
treatable
will
included
scope
OD.
Frontiers in Medicine,
Journal Year:
2022,
Volume and Issue:
9
Published: July 15, 2022
Successful
sequencing
of
the
human
genome
and
evolving
functional
knowledge
gene
products
has
taken
genomic
medicine
to
forefront,
soon
combining
broadly
with
traditional
diagnostics,
therapeutics,
prognostics
in
patients.
Recent
years
have
witnessed
an
extraordinary
leap
our
understanding
ocular
diseases
their
respective
genetic
underpinnings.
As
we
are
entering
age
medicine,
rapid
advances
sequencing,
delivery,
surgery,
computational
genomics
enable
ever-increasing
capacity
provide
a
precise
robust
diagnosis
development
targeted
treatment
strategies.
Inherited
retinal
major
source
blindness
around
world
where
large
number
causative
genes
been
identified,
paving
way
for
personalized
diagnostics
clinic.
Developments
genetics
transfer
techniques
also
led
first
FDA
approval
therapy
LCA,
childhood
blindness.
Many
such
focus
various
clinical
trials,
making
diagnoses
diseases,
underlying
studies
natural
history
important.
Here,
review
methodologies
identifying
new
variants
associated
disorders
complexities
them.
Thereafter
discuss
briefly,
application
technologies
diagnosis.
We
strategies,
challenges,
potential
inherited
acquired
diseases.
Additionally,
translational
aspects
therapy,
important
vector
types
considerations
trials
that
may
help
advance
therapeutics
ophthalmology.
Retinal
disease
research
precision
therapies;
therefore,
this
provides
general
current
status
Expert Opinion on Biological Therapy,
Journal Year:
2022,
Volume and Issue:
22(9), P. 1193 - 1208
Published: Sept. 2, 2022
Ocular
gene
therapy
represents
fertile
ground
for
rapid
innovation,
with
ever-expanding
therapeutic
strategies,
molecular
targets,
and
indications.Potential
indications
ocular
have
classically
focused
on
inherited
retinal
disease
(IRD)
but
more
recently
include
acquired
diseases,
such
as
neovascular
age-related
macular
degeneration,
geographic
atrophy,
diabetic
retinopathy.
strategies
proliferated
recently,
augmentation,
inactivation,
editing,
RNA
modulation,
gene-independent
augmentation.
Viral
vector
constructs
adeno-associated
virus
lentivirus
continue
to
evolve
through
directed
evolution
rationale
design.
administration
techniques
expanded
beyond
pars
plana
vitrectomy
subretinal
injection
intravitreal
suprachoroidal
injection.The
success
of
treatment
IRD,
paired
the
promise
clinical
research
in
diseases
techniques,
has
raised
possibility
in-office
common
disorders
within
next
5
10
years.
