BRISBANE, Calif., Feb. 20, 2019 /PRNewswire/ --
Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine
company, announced today that the U.S. Food and Drug Administration
(FDA) has accepted the Investigational New Drug (IND) application
for ST-920, a gene therapy candidate being evaluated for the
treatment of adults with Fabry disease. Current standard of care
for this rare, progressive condition involves regular lifelong
infusions of enzyme replacement therapy (ERT).
"The FDA's acceptance of the IND to evaluate ST-920 in Fabry
disease enables the initiation of our third clinical development
program focused on rare metabolic diseases, and our sixth active
clinical program," said Edward
Conner, M.D., Chief Medical Officer at Sangamo. "We are
eager to commence trials of ST-920 to evaluate our hypothesis that
the one-time administration of a gene therapy can fundamentally
change the clinical course for patients with Fabry
disease."
Fabry disease is an inherited metabolic disease caused by
mutations in the GLA gene, which result in a deficiency of
the enzyme alpha-galactosidase A (α-Gal A). In the absence of
functional enzyme, fatty compounds called ganglioside
globotriaosylceramides (Gb3) accumulate and can cause serious harm
to the skin, kidneys, heart, and the nervous system.
ST-920 comprises an AAV vector carrying a GLA gene
construct driven by a proprietary liver-specific promoter. ST-920
gene therapy is designed to enable a patient's liver to produce a
long-lasting and continuous supply of the α-Gal A enzyme.
The active IND enables Sangamo to initiate a Phase 1/2 clinical
trial designed to assess the safety, tolerability and efficacy of
ST-920 in adults with Fabry disease. Sangamo expects to open
several clinical sites later this year.
About Sangamo Therapeutics
Sangamo Therapeutics, Inc. is focused on translating
ground-breaking science into genomic medicines with the potential
to transform patients' lives using the Company's platform
technologies in genome editing, gene therapy, gene regulation and
cell therapy. For more information about Sangamo, visit
www.sangamo.com.
Forward-Looking Statements
This press release contains forward-looking statements
regarding Sangamo's current expectations. These forward-looking
statements include, without limitation, Sangamo's plans to initiate
the Phase 1/2 clinical trial for ST-920 in 2019; the potential for
these trials to evaluate our hypothesis that the one-time
administration of a gene therapy can fundamentally change the
clinical course for these patients; the design of ST-920 gene
therapy to enable a patient's liver to produce a long-lasting and
continuous supply of the α-Gal A enzyme; and Sangamo's expectations
to open several clinical sites later this year. These statements
are not guarantees of future performance and are subject to certain
risks, uncertainties and assumptions that are difficult to predict.
Factors that could cause actual results to differ include, but are
not limited to, the dependence on the success of clinical trials of
programs, the lengthy and uncertain regulatory approval process,
uncertainties related to the initiation, enrollment and completion
of clinical trials, Sangamo's reliance on partners and other
third-parties to meet their clinical and manufacturing obligations,
and the ability to maintain strategic partnerships. Further, there
can be no assurance that the necessary regulatory approvals will be
obtained or that Sangamo and its partners will be able to develop
commercially viable product candidates. Actual results may differ
from those projected in forward-looking statements due to risks and
uncertainties that exist in Sangamo's operations and business
environments. These risks and uncertainties are described more
fully in Sangamo's Quarterly Report on Form 10-Q for the quarter
ended September 30, 2018 as filed
with the Securities and Exchange Commission. Forward-looking
statements contained in this announcement are made as of this date,
and Sangamo undertakes no duty to update such information except as
required under applicable law.
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SOURCE Sangamo Therapeutics, Inc.