Ocugen, Inc. Announces Dosing Completion of Subjects with Stargardt in Cohort 1 of Phase 1/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410ST
February 22 2024 - 4:05PM
Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a
biotechnology company focused on discovering, developing, and
commercializing novel gene and cell therapies and vaccines, today
announced that dosing is complete in the first cohort of its Phase
1/2 GARDian clinical trial for OCU410ST (AAV5-hRORA)—a
modifier gene therapy candidate being developed for Stargardt
disease. Stargardt disease is an orphan blindness disease that
affects approximately 35,000 people in the United States alone.
“This is an important clinical milestone for our first-in-class,
potential one-time therapy for the treatment of Stargardt disease,”
said Dr. Arun Upadhyay, Chief Scientific Officer and Head of
R&D at Ocugen. “Although Stargardt is one of the most common
inherited retinal diseases, there remains no treatment option to
address this condition. OCU410ST provides hope to these patients
who may eventually lose their vision.”
Up to 10 leading retinal surgery centers across the United
States are participating in the GARDian clinical trial. In the
first cohort, 3 subjects received 200L single subretinal
administration of the low dose (3.75x1010 vg/mL) of OCU410ST.
“I am very pleased to participate in this study and to offer a
novel modifier gene therapy option to my patients,” said Benjamin
Bakall, MD, PhD, Director of Clinical Research at Associated Retina
Consultants and Clinical Assistant Professor at the University of
Arizona, College of Medicine—Phoenix. “Until now, we have not had
any effective treatment that can prevent the vision loss
in patients with Stargardt disease. Now, I believe that
this approach can offer a new therapeutic option to address the
disease itself.”
The GARDian clinical 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 consisting of three
dose levels [low dose (3.75×10E10 vg/mL), medium dose (7.5×10E10
vg/mL), and high dose (2.25×10E11 vg/mL)]. 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. The Company
will continue to provide clinical updates.
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 OCU410ST
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.
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.
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Contact:Tiffany HamiltonHead of
CommunicationsTiffany.Hamilton@ocugen.com
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