MeiraGTx Expands ALS Gene Therapy Program in Collaboration with Weill Cornell Medicine and LSU

October 11, 2016 - als

NEW YORK–(BUSINESS WIRE)–MeiraGTx, a New York and UK formed gene therapy company, announces an
enlargement of a gene therapy module to yield amyotrophic lateral
sclerosis (Lou Gehrig’s Disease, ALS).

MeiraGTx, together with Prof. Gregory Petsko, D.Phil. (The Arthur J.
Mahon Professor of Neuroscience in a Feil Family Brain and Mind
Research Institute during Weill Cornell Medicine), and Prof. Ronald Klein,
Ph.D. (Associate Professor Department of Pharmacology, Toxicology and
Neuroscience, LSU Health Shreveport), are building a gene therapy to
aim TDP-43 toxicity in ALS patients. The mobile protein TDP-43 is
misregulated in a immeasurable infancy of all ALS patients and is rising as
a probable Achilles’ heel in a diagnosis of this deadly
neurodegenerative disease.

Research conducted in a Petsko laboratory (in partnership with
coworkers during Brandeis University, Wright State University and the
University of Michigan) suggested that one of a cell’s possess quality
control mechanisms, called a nonsense-mediated spoil (NMD) pathway,
can assuage TDP-43 mediated toxicity in cell-based illness models of
ALS. Increased countenance of UPF1, a master regulator of NMD,
significantly protects mammalian engine neurons from TDP-43 mediated
toxicity. “UPF1 has shown earnest formula in animal models of ALS
involving TDP-43 dysfunction and provides a motive for developing
gene-based therapies for ALS, and we are vehement about relocating this
brazen with a association that has a systematic insights of MeiraGTx”
pronounced Dr. Petsko. MeiraGTx recently entered into a investigate collaboration
with Professor Klein of Louisiana State University (LSU) to conduct
pre-clinical animal studies directed during demonstrating a efficiency of a
UPF1-based therapy in animal models of TDP-43 prompted ALS pioneered in
his laboratory.

MeiraGTx and a educational collaborators are defining a purpose of RNA
metabolism in a pathology of ALS, with a idea of developing
effective gene therapies. “We are really vehement about a work with Drs.
Petsko and Klein, leaders in a margin of neurodegeneration, to develop
a gene therapy targeting TDP-43 dysfunction in ALS,” pronounced Alexandria
Forbes, Ph.D., President and CEO of MeiraGTx. “We trust that this may
lead to not usually an critical new healing approach, though further
review of a genetic causes of ALS will yield discernment for the
growth of additional gene therapies to yield this and other
harmful neurodegenerative diseases.”

About MeiraGTx

MeiraGTx is committed to a growth of novel gene therapies to
renovate a lives of patients pang from acquired and inherited
disorders. The association is building treatments for visible diseases,
including singular hereditary blindness and age-related macular degeneration
(AMD). MeiraGTx is also substantiating treatments for xerostomia, a
revisit and debilitating side outcome of deviation diagnosis used in
conduct and neck cancers, as good as certain neurodegenerative diseases. In
addition, MeiraGTx is building novel gene law platforms that
guarantee to renovate a approach gene therapy can be practical and emanate new
paradigms for biologic therapeutics.

About Weill Cornell Medicine

Weill Cornell Medicine is committed to value in studious care,
systematic find and a preparation of destiny physicians in New York
City and around a world. The doctors and scientists of Weill Cornell
Medicine — expertise from Weill Cornell Medical College, Weill Cornell
Graduate School of Medical Sciences, and Weill Cornell Physician
Organization — are intent in world-class clinical caring and cutting-edge
investigate that bond patients to a latest diagnosis innovations and
impediment strategies. Located in a heart of a Upper East Side’s
systematic corridor, Weill Cornell Medicine’s absolute network of
collaborators extends to a primogenitor university Cornell University; to
Qatar, where an general campus offers a U.S. medical degree; and
to programs in Tanzania, Haiti, Brazil, Austria and Turkey. Weill
Cornell Medicine expertise yield extensive studious caring at
NewYork-Presbyterian/Weill Cornell Medical Center,
NewYork-Presbyterian/Lower Manhattan Hospital and
NewYork-Presbyterian/Queens. Weill Cornell Medicine is also affiliated
with Houston Methodist. For some-more information, revisit
Weill Cornell Medicine’s Office of Biopharma Alliances and Research
Collaborations’ goal is to proactively generate, structure and
negotiate translational investigate alliances with attention in sequence to
allege earnest investigate projects that have blurb potential.

About LSU

LSU Health Shreveport core goal is to heal, to learn and to discover.
The Health Sciences Center located in Northwest Louisiana includes a
School of Medicine, School of Allied Health and School of Graduate
Studies. It provides caring to patients via a state of Louisiana
by a 530 expertise physicians and other medical providers, and
educates over 1,400 students, residents and fellows. Designated Centers
of Excellence embody a Feist Weiller Cancer Center (FWCC), a Center
for Cardiovascular Diseases and Science, and a Center for Arthritis
and Rheumatology. Research is a core partial of our institution’s mission
and a LSUHSC-S investigate space includes approximately 200,000 sq. ft.
of laboratories and offices, as good as comforts including shared
apparatus in a departments and a designated Research Core
Facility. Faculty, staff and students during LSU Health Shreveport are
actively intent in investigate in a accumulation of biomedical areas, with
concentrations in cancer, cardiovascular sciences, virology and

This press recover contains forward-looking statements. These
forward-looking statements are formed on management’s expectations and
are theme to certain factors, risks and uncertainties that might cause
tangible results, outcome of events, timing and opening to differ
materially from those voiced or pragmatic by such statements. The
information contained in this press recover is believed to be stream as
of a date of strange issue. MeiraGTx specifically disclaims any
requirement or endeavour to recover publicly any updates or revisions
to any forward-looking statements contained herein to simulate any change
in a expectations with courtesy thereto or any change in events,
conditions or resources on that any such statements are based.

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