Nightstar Expands Pipeline with Novel Gene Therapy for the Treatment of Stargardt Disease
08 November 2017 - 10:30PM
Fourth Gene Therapy Program Targets the Most
Common Inherited Juvenile Macular Dystrophy
Nightstar Therapeutics (NASDAQ:NITE), a clinical-stage gene therapy
company developing treatments for rare inherited retinal diseases,
today announced an expansion of its pipeline with an exclusive
global license for a gene therapy program for the treatment of
Stargardt disease from Oxford University Innovation.
The gene therapy program utilizes a novel technology developed
by the University of Oxford. Preclinical proof-of-concept studies
in the Abca4-/- murine model of Stargardt disease have demonstrated
the expression, localization and function of the ABCA4 protein.
Stargardt disease is the most common form of inherited juvenile
macular dystrophy with a prevalence of one in 10,000. This
autosomal recessive disease is linked to mutations in the ABCA4
gene that are inherited from both parents of an affected
individual. The progressive central vision loss associated with
Stargardt disease is caused by the degeneration of photoreceptor
cells in the central portion of the retina called the macula.
Stargardt disease typically develops during childhood and
adolescence, leading to blindness by the age of twenty. There are
currently no treatment options for this disease.
“The licensing of this novel gene therapy program exemplifies
our commitment to developing treatments for patients suffering from
inherited retinal diseases that would otherwise lead to blindness,”
said Dave Fellows, chief executive officer of Nightstar. “We plan
to leverage our clinical experience with choroideremia and X-linked
retinitis pigmentosa to accelerate the development of this exciting
program for the unmet medical need in Stargardt disease.”
Financial terms of the agreement were not disclosed.
About Nightstar
Nightstar is a leading clinical-stage gene therapy company
focused on developing and commercializing novel one-time treatments
for patients suffering from rare inherited retinal diseases that
would otherwise progress to blindness. Nightstar’s lead product
candidate, NSR-REP1, is being developed as a treatment for patients
with choroideremia, a rare, degenerative, genetic retinal disorder
that has no current treatments and affects approximately one in
every 50,000 people. Positive results from a Phase 1/2 trial of
NSR-REP1 were published in The Lancet in 2014 and in The New
England Journal of Medicine in 2016. Nightstar plans to commence a
Phase 3 registrational trial of NSR-REP1 for choroideremia at sites
in the United States, Europe and Canada in the first half of 2018.
Nightstar’s second product candidate, NSR-RPGR, is currently being
evaluated in a Phase 1/2 clinical trial for the treatment of
patients with X-linked retinitis pigmentosa, an inherited X-linked
recessive retinal disease that affects approximately one in every
40,000 people.
For more information about Nightstar or its clinical trials,
please visit www.nightstartx.com.
About Oxford University Innovation
Oxford University Innovation (OUI) supports
innovation activities across all divisions of the University of
Oxford, managing technology transfer and consulting activities, and
providing an innovation management service to clients around the
world. OUI provides access to technology from Oxford researchers
through intellectual property licensing, spinout company formation
and material sales, and to academic expertise through its
Consulting Services team. The New Venture Support & Funding
team of OUI supports investors or donors with an interest in
early-stage ventures, and manages the Oxford Angels Network. OUI’s
Startup Incubator supports members and ex-members of the University
who wish to start or grow entrepreneur-driven ventures that are not
University spinouts.
Forward-Looking Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995, including, but not limited to: statements about our plans to
develop and commercialize our product candidates, our planned
clinical trials for NSR-REP1 and NSR-RPGR, the clinical utility of
our product candidates, the prevalence of patient populations for
our targeted indications, and the utility of prior preclinical and
clinical data in determining future clinical results. These
forward-looking statements are based on management's current
expectations of future events and are subject to a number of
involve substantial known and unknown risks, uncertainties and
other factors that may cause our actual results, levels of
activity, performance or achievements to be materially different
from the information expressed or implied by these forward-looking
statements, including the risks and uncertainties set forth in the
"Risk Factors" section of our prospectus filed pursuant to Rule
424(b)(4) under the U.S. Securities Act of 1933, as amended, on
September 28, 2017, and subsequent reports that we file with the
U.S. Securities and Exchange Commission. We may not actually
achieve the plans, intentions or expectations disclosed in our
forward-looking statements, and you should not place undue reliance
on our forward-looking statements. Actual results or events could
differ materially from the plans, intentions and expectations
disclosed in the forward-looking statements we make. The
forward-looking statements in this press release represent our
views as of the date hereof. We anticipate that subsequent events
and developments will cause our views to change. However, while we
may elect to update these forward-looking statements at some point
in the future, we have no current intention of doing so except to
the extent required by applicable law. You should, therefore, not
rely on these forward-looking statements as representing our views
as of any date subsequent to the date of this press release.
Contact:Senthil Sundaram, Chief Financial
Officerinvestors@nightstartx.com
Alicia Davis, THRUST IR910-620-3302alicia@thrustir.com
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