Nature Gene Therapy Publishes Preclinical Data of Ocugen’s OCU400 (NR2E3-AAV) Genetic Modifier to treat Retinitis Pigmentos...
March 03 2020 - 7:30AM
Ocugen, Inc. (NASDAQ: OCGN), a clinical-stage company focused on
discovering, developing and commercializing transformative
therapies to treat rare and underserved ophthalmic diseases,
announced today the publication in Nature Gene Therapy of
preclinical data of nuclear hormone receptor gene NR2E3 as a
genetic modifier and therapeutic agent to treat multiple retinal
degenerative diseases. OCU400 (NR2E3-AAV) has received two
orphan drug designations targeting two distinct inherited retinal
diseases (IRDs): NR2E3 mutation-associated retinal diseases and
CEP290 mutation-associated retinal diseases.
The publication details efficacy results in five
unique mouse models of retinitis pigmentosa (RP) that underwent
administration of NR2E3-AAV by subretinal injection. The five
RP models tested were rd1 (PDE6β associated RP), Rho-/- and
RhoP23H (both Rhodopsin associated RP), rd16 (Leber Congenital
Amaurosis) and rd7 (Enhanced S-cone Syndrome). The study
demonstrates the potency of a novel modifier gene therapy to elicit
broad-spectrum therapeutic benefits in early and intermediate
stages of RP.
Please refer to Nature Gene Therapy’s online
publication for additional results from this study.
“This represents an important milestone for the
development of this therapy. I am impressed by the protection that
was elicited in multiple animal models of degeneration caused by
different mutations. A treatment for inherited retinal
degenerations that is mutation independent would have wide reaching
implications,” said Mark Pennesi, M.D., Ph.D., Associate Professor
of Ophthalmology at the Oregon Health & Science University
(OHSU) School of Medicine and Division Chief of the Ophthalmic
Genetics Service at the OHSU Casey Eye Institute.
“Dr. Haider and her vision research team have
successfully demonstrated proof of principle in their elegant study
by rescuing 5 animal models of RP by resetting homeostasis.
This is the foundation work for the development of the first broad
spectrum therapy for inherited retinal degeneration diseases and is
a game changer for rescue even after disease onset,” said Cheryl
Mae Craft, Ph.D., Professor of Ophthalmology, USC Keck School of
Medicine at USC Roski Eye Institute Los Angeles, CA.
“One of the biggest advantages of our modifier gene
therapy platform is that it has the potential to eliminate the need
for individual gene replacement and gene editing strategies and may
revolutionize gene therapy treatments for eye diseases.
Inherited retinal degenerations such as RP affect over 1.5 million
people worldwide. Over 150 gene mutations have been
associated with RP and this number represents only 60% of the RP
population. The remaining 40% of RP patients cannot be
genetically diagnosed, making it difficult to develop individual
treatments. Our modifier gene therapy has potential to
eliminate the need for developing more than 150 individual products
and provide one treatment option for all RP patients,” said Rasappa
Arumugham, Ph.D., Ocugen’s Chief Scientific Officer. “We are
completing preclinical studies for OCU400 and anticipate commencing
a Phase 1/2a clinical trial in patients in 2021.”
About Retinitis
PigmentosaRetinitis Pigmentosa (RP) is a group of rare,
genetic disorders that involve a breakdown and loss of cells in the
retina. According to the National Eye Institute, it is
estimated that RP affects approximately 1 in 4,000 people, both in
the U.S. and worldwide. RP symptoms often begin in childhood and
progress over time. Children often have difficulty getting
around in the dark and as their symptoms progress, lose their
peripheral (side) vision and eventually experience vision loss and
blindness. Because there are many gene mutations that cause
this disorder, its progression can differ greatly from person to
person.
About OCU400 OCU400 is a novel
gene therapy product candidate with the potential to be broadly
effective in restoring retinal integrity and function across a
range of genetically diverse IRDs. OCU400 is the first program that
Ocugen is advancing based on its breakthrough modifier gene therapy
platform developed by Dr. Neena Haider, PhD, Associate Professor of
Ophthalmology at Harvard Medical School and Associate Scientist at
the Schepens Eye Research Institute of Massachusetts Eye and
Ear. Ocugen obtained an exclusive worldwide license from SERI
to develop and commercialize ophthalmology products based on the
platform. Consisting of a functional copy of the nuclear
hormone receptor gene NR2E3, OCU400 is delivered to target cells in
the retina using an adeno-associated viral (AAV) vector. As a
potent modifier gene, expression of NR2E3 within the retina may
help reset retinal homeostasis, stabilizing cells and potentially
rescuing photoreceptors from degeneration.
About Ocugen, Inc. Ocugen, Inc. is
a clinical-stage biopharmaceutical company focused on discovering,
developing and commercializing transformative therapies to treat
the whole eye. Our Phase 3 small molecule drug candidate for ocular
graft-versus-host disease, if approved, will be the first and only
treatment for this orphan disease. Our breakthrough modifier
gene therapy platform has the potential to treat multiple retinal
diseases with one drug – “one to many”. And our novel
biologic product candidate aims to offer better therapy to patients
with underserved diseases such as wet age-related macular
degeneration, diabetic macular edema and diabetic retinopathy. For
more information, please visit www.ocugen.com.
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Ocugen, Inc.
Kelly Beck
kelly.beck@ocugen.com
+1 484-328-4698
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