Ocugen, Inc. Announces First Patient Dosed in Phase 1/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410ST—Modifier Gene Therapy—for Stargardt Disease
November 10 2023 - 7:30AM
Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a
biotechnology company focused on discovering, developing, and
commercializing novel gene and cell therapies, biologics, and
vaccines, today announced that the first patient has been dosed in
its Phase 1/2 GARDian clinical trial for
OCU410ST (AAV5-hRORA)—a modifier gene therapy candidate being
developed for Stargardt disease, a rare genetically inherited
disease that directly affects the retina, often resulting in slow
progressive vision loss in children and adults.
“There is a significant unmet medical need for the approximate
35,000 patients in the U.S. living with Stargardt disease,” said
Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “It
is critical to our mission to develop innovative treatments for
inherited retinal diseases and this milestone is an important step
in bringing our novel modifier gene therapies to people who
desperately need them.”
This Phase 1/2 trial will assess the safety of unilateral
subretinal administration of OCU410ST in subjects with Stargardt
Disease and will be conducted in two phases. Phase 1 is a
multicenter, open-label, dose ranging study. Phase 2 is a
randomized, outcome accessor-blinded, dose-expansion study in which
adult and pediatric subjects will be randomized in a 1:1:1 ratio to
either one of two OCU410ST dose groups or to an untreated control
group.
OCU410ST utilizes an AAV delivery platform for the retinal
delivery of the RORA (RAR Related Orphan Receptor A) gene. It
represents Ocugen’s modifier gene therapy approach, which is based
on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links
to Stargardt disease such as lipofuscin formation, oxidative
stress, compliment formation, inflammation, and cell survival
networks.
“It is important and exciting to pursue novel therapies for
untreatable blinding diseases,” said Charles Wykoff, MD, PhD,
Director of Research, Retina Consultants of Texas. “Initiation of
this trial program investigating a new mechanism of action for the
treatment of Stargardt disease is inspiring and brings hope to
patients and families.”
About Stargardt Disease
Stargardt disease is a genetic eye disorder that causes retinal
degeneration and vision loss. Stargardt disease is the most common
form of inherited macular degeneration. The progressive vision loss
associated with Stargardt disease is caused by the degeneration of
photoreceptor cells in the central portion of the retina called the
macula.
Decreased central vision due to loss of photoreceptors in the
macula is the hallmark of Stargardt disease. Some peripheral vision
is usually preserved. Stargardt disease typically develops during
childhood or adolescence, but the age of onset and rate of
progression can vary. The retinal pigment epithelium (RPE), a layer
of cells supporting photoreceptors, is also affected in people with
Stargardt disease.
About Ocugen, Inc. Ocugen, Inc. is a
biotechnology company focused on discovering, developing, and
commercializing novel gene and cell therapies, biologics, and
vaccines that improve health and offer hope for patients across the
globe. We are making an impact on patients’ lives through
courageous innovation—forging new scientific paths that harness our
unique intellectual and human capital. Our breakthrough modifier
gene therapy platform has the potential to treat multiple retinal
diseases with a single product, and we are advancing research in
infectious diseases to support public health and orthopedic
diseases to address unmet medical needs. Discover more
at www.ocugen.com and follow us
on X and LinkedIn.
Forward-Looking Statements This press
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identify these forward-looking statements. Such statements are
subject to numerous important factors, risks, and uncertainties
that may cause actual events or results to differ materially from
our current expectations. These and other risks and uncertainties
are more fully described in our periodic filings with the
Securities and Exchange Commission (SEC), including the risk
factors described in the section entitled “Risk Factors” in the
quarterly and annual reports that we file with the SEC. Any
forward-looking statements that we make in this press release speak
only as of the date of this press release. Except as required by
law, we assume no obligation to update forward-looking statements
contained in this press release whether as a result of new
information, future events, or otherwise, after the date of this
press release.
Contact: Tiffany Hamilton Head of
Communications Tiffany.Hamilton@ocugen.com
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