Ocugen Granted FDA Orphan Drug Designation for OCU400 (AAV-hNR2E3) Gene Therapy for the Treatment of RHO Mutation-Associated ...
July 27 2020 - 07:30AM
Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on
discovering, developing, and commercializing transformative
therapies to cure blindness diseases, today announced the U.S. Food
and Drug Administration (FDA) granted the third Orphan Drug
Designation (ODD) for OCU400 in the treatment of RHO
mutation-associated retinal degeneration. The RHO mutation is part
of the Retinitis Pigmentosa (RP) group of rare, genetic disorders
that involve a breakdown and loss of cells in the retina and can
lead to visual impairment and blindness. This is one of the
larger mutations within the RP class, representing about 12% of RP
patients in the US.
A novel gene therapy product candidate, OCU400 has the potential
to be broadly effective in restoring retinal integrity and function
across a range of genetically diverse inherited retinal diseases.
It consists of a functional copy of a nuclear hormone receptor
(NHR) gene, NR2E3, delivered to target cells in the retina using an
adeno-associated viral vector. As a potent modifier gene,
expression of NR2E3 within the retina may help reset retinal
homeostasis and potentially offer longer benefit, stabilizing cells
and rescuing photoreceptor degeneration and vision loss.
Adding to ODDs for OCU400 for NR2E3 and CEP290
mutation-associated retinal degeneration, RHO gene
mutation-associated retinal degeneration further supports Ocugen’s
breakthrough modifier gene therapy platform’s potential to treat
multiple blindness diseases with a single product. RP is a
group of heterogenic inherited retinal diseases associated with
over 150 gene mutations, affecting over 1.5 million individuals
worldwide. In addition, ~40% of RP patients cannot be genetically
diagnosed, confounding the ability to develop personalized RP
therapies. Traditional gene therapy or gene editing
approaches may require more than 150 products to rescue these
patients from vision loss. OCU400, a single product
candidate, has potential to address broad-spectrum RP.
“OCU400, comprising the nuclear hormone receptor
gene NR2E3, has the potential to help modulate numerous
biological pathways that function in maintaining the health of the
retina. A recent preclinical study published in Nature Gene
Therapy demonstrated the potency of NR2E3 to elicit
broad-spectrum therapeutic benefits in early and intermediate
stages of RP in five unique mouse models,” said Dr. Mohamed Genead,
acting Chief Medical Officer of Ocugen and Chair of Ocugen’s Retina
Scientific Advisory Board. “We believe OCU400 has the potential to
address multiple genetic mutations associated with RP and,
therefore, help a broader pool of patients,” Dr. Genead
continued.
“Our third ODD for OCU400 from the FDA is an important step
towards developing a broad-spectrum treatment for RP and getting a
therapy faster to patients who are in desperate need of rescue,”
said Dr. Shankar Musunuri, Chairman, Chief Executive Officer and
Co-Founder of Ocugen. “Orphan designation for this indication
supports the goal of our Modifier Gene Therapy Platform to treat a
variety of inherited retinal diseases with a single gene therapy
product. There are currently no approved treatments which slow or
stop the progression of multiple forms of RP, which is why we’re
excited to have a platform that can potentially address multiple
mutations, including mutations in the Rhodopsin gene, with one
therapy.”
The FDA Office of Orphan Products Development grants orphan
designation for novel drugs or biologics that treat a rare disease
or condition affecting fewer than 200,000 patients in the U.S.
Orphan designation qualifies the sponsor of the drug for various
development incentives of the Orphan Drug Act, including a
seven-year period of U.S. marketing exclusivity, tax credits for
clinical research costs, clinical research trial design assistance,
the ability to apply for annual grant funding and waiver of
Prescription Drug User Fee Act filing fees.
About OCU400OCU400 (AAV-hNR2E3) 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 inherited retinal diseases. It
consists of a functional copy of a nuclear hormone receptor gene,
NR2E3, delivered to target cells in the retina using an
adeno-associated viral vector. As a potent modifier gene,
expression of NR2E3 within the retina may help reset retinal
homeostasis, potentially stabilizing cells and rescuing
photoreceptor degeneration and vision loss.
About Ocugen, Inc.Ocugen, Inc. is a
biopharmaceutical company focused on discovering, developing, and
commercializing transformative therapies to cure blindness
diseases. 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 https://ocugen.com/
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Corporate Contact:Ocugen,
Inc.Sanjay SubramanianChief Financial
OfficerIR@Ocugen.com
Media Contact:
LaVoieHealthScienceEmmie
Twomblyetwombly@lavoiehealthscience.com+1 857-389-6042
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