Ocugen Announces OCU400—Modifier Gene Therapy—Phase 1/2 Data Presentation at Retinal Cell and Gene Therapy Innovation Summit 2024
April 29 2024 - 6:45AM
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 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
will present data from the OCU400 Phase 1/2 clinical trial at the
Retinal Cell and Gene Therapy Innovation Summit being held May 3,
2024 in Seattle, WA.
“I look forward to sharing more about my clinical experience
with this novel modifier gene therapy with my peers and industry
leadership during the summit,” said Dr. Bakall. “There remains a
significant unmet need for patients with retinitis pigmentosa (RP)
and I believe that this approach can offer a new therapeutic option
to address the disease itself.”
The Retinal Cell and Gene Therapy Innovation Summit 2024 is
jointly organized by the Foundation Fighting Blindness and the
Oregon Health and Science University Casey Eye Institute. The
Summit brings together representatives from the biotech and pharma
industries, along with members of the medical and research
communities, to discuss rapidly emerging ocular gene and cell
therapies and strategize how to move the most advanced retinal
disease therapies toward clinical utility.
Details on Dr. Bakall’s presentation are as follows:
Presentation Title: “Nuclear
Hormone Receptor-Based Gene Modifier Therapy: Safety and Efficacy
from Phase 1/2 Clinical Trials for Retinitis
Pigmentosa”Date: Friday, May 3,
2024Time: 10:50 – 11:05 a.m. (PT)
Location: Hyatt Regency Seattle
The OCU400 Phase 3 liMeliGhT clinical trial is currently
underway and on track to meet the Company’s 2026 BLA and
MAA approval targets. Between the U.S. and EU, nearly 300,000
people are affected by RP.
About OCU400OCU400 is the Company’s
gene-agnostic modifier gene therapy product based on NHR
gene, NR2E3. NR2E3 regulates diverse physiological
functions within the retina—such as photoreceptor development and
maintenance, metabolism, phototransduction, inflammation and cell
survival networks. Through its drive functionality, OCU400 resets
altered/affected cellular gene networks and establishes
homeostasis—a state of balance, which has the potential to improve
retinal health and function in patients with RP.
About RPRP is a group of rare, genetic
disorders that involve a breakdown and loss of cells in the retina,
leading to vision loss and blindness. Currently, RP is associated
with mutations in more than 100 genes. There are no approved
treatment options that slow or stop the progression of multiple
forms of RP. Proposed treatments for RP include gene replacement
therapy, retinal implant devices, retinal transplantation, stem
cells, vitamin therapy, and other pharmacological treatments.
Current gene replacement therapies are promising but are limited to
treating just a single mutation. In addition, while gene therapies
may provide a new functional gene, they do not necessarily
eliminate the underlying genetic defect, which may still cause
stress and toxic effects leading to retina degeneration. Therefore,
the development of gene-specific replacement therapy is highly
challenging, especially when multiple and unknown genes are
involved. Thus, novel therapeutic approaches targeting broader RP
disease in a gene-agnostic manner offer greater hope for
patients.
About Ocugen, Inc.Ocugen, Inc. is a
biotechnology company focused on discovering, developing, and
commercializing novel gene and cell therapies, and vaccines that
improve health and offer hope for patients across the globe. We are
making an impact on patient’s 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.
Cautionary Note on Forward-Looking
StatementsThis press release contains forward-looking
statements within the meaning of The Private Securities Litigation
Reform Act of 1995, including, but not limited to, statements
regarding qualitative assessments of available data, potential
benefits, expectations for ongoing clinical trials, anticipated
regulatory filings and anticipated development timelines, which are
subject to risks and uncertainties. We may, in some cases, use
terms such as “predicts,” “believes,” “potential,” “proposed,”
“continue,” “estimates,” “anticipates,” “expects,” “plans,”
“intends,” “may,” “could,” “might,” “will,” “should,” or other
words that convey uncertainty of future events or outcomes to
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, including, but not limited to, the risks
that preliminary, interim and top-line clinical trial results may
not be indicative of, and may differ from, final clinical data;
that unfavorable new clinical trial data may emerge in ongoing
clinical trials or through further analyses of existing clinical
trial data; that earlier non-clinical and clinical data and testing
of may not be predictive of the results or success of later
clinical trials; and that that clinical trial data are subject to
differing interpretations and assessments, including by regulatory
authorities. 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 HamiltonHead of
CommunicationsTiffany.Hamilton@ocugen.com
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