RICHMOND, Calif., March 1, 2017 /PRNewswire/ --
Sangamo Therapeutics, Inc. (NASDAQ: SGMO), the leader in
therapeutic genome editing, announced today that the U.S. Food and
Drug Administration (FDA) has granted orphan drug designation to
SB-913, a genome editing product candidate for the treatment of
Mucopolysaccharidosis Type II (MPS II), a rare lysosomal storage
disorder. Orphan drug designations are granted to drugs and
biologics intended to treat rare diseases with a patient population
less than 200,000 in the U.S. The designation provides incentives
to advance development and commercialization of rare disease drugs.
Sangamo has also submitted an application to the FDA for rare
pediatric disease designation for SB-913.
MPS II is caused by mutations in the gene encoding iduronate
2-sulfatase (IDS) enzyme. Using Sangamo's zinc finger nuclease
(ZFN) genome editing technology, SB-913 is designed as a single
treatment strategy intended to provide stable, continuous
production of the IDS enzyme for the lifetime of the patient.
In 2017, Sangamo is conducting a Phase 1/2 clinical trial
evaluating SB-913 as an in vivo genome editing treatment for
MPS II. Sangamo is also conducting Phase 1/2 studies this year
evaluating in vivo genome editing treatments SB-318 for MPS
I, another rare lysosomal storage disorder, and SB-FIX for
hemophilia B, a rare blood disease. Data from these studies and
from a clinical trial for a fourth lead program, SB-525, a gene
therapy approach for hemophilia A, are expected in late 2017 or
early 2018.
Sangamo's In Vivo Genome Editing
Approach
Sangamo's ZFN-mediated in vivo genome
editing approach makes use of the endogenous albumin gene locus, a
highly expressing and liver-specific site that can be edited with
ZFNs to accept and express therapeutic genes. The approach is
designed to enable the patient's liver to permanently produce
circulating therapeutic levels of a corrective protein. The ability
to permanently integrate the therapeutic gene in a highly specific,
targeted fashion significantly differentiates Sangamo's in
vivo genome editing approach from conventional AAV cDNA gene
therapy. Ultimately, the target population for these programs will
include pediatric patients, and it will be important in this
population to be able to produce stable levels of therapeutic
protein for the lifetime of the patient.
About Sangamo Therapeutics
Sangamo
Therapeutics, Inc. is focused on translating ground-breaking
science into genomic therapies that transform patients' lives using
the company's industry leading platform technologies in genome
editing, gene therapy, gene regulation and cell therapy. The
Company is advancing Phase 1/2 clinical programs in hemophilia A
and hemophilia B, and lysosomal storage disorders MPS I and MPS II.
Sangamo has a strategic collaboration with Bioverativ Inc. for
hemoglobinopathies, including beta thalassemia and sickle cell
disease, and with Shire International GmbH to develop therapeutics
for Huntington's disease. In addition, it has established strategic
partnerships with companies in non-therapeutic applications of its
technology, including Sigma-Aldrich Corporation and Dow
AgroSciences. For more information about Sangamo, visit the
Company's website at www.sangamo.com.
Forward Looking Statements
This press release may contain forward-looking statements based on
Sangamo's current expectations. These forward-looking statements
include, without limitation references relating to research and
development of therapeutic applications of Sangamo's gene therapy
and ZFP technology platforms, the potential of Sangamo's technology
to treat hemophilia and lysosomal storage disorders, the expected
timing of these clinical trials and the release of data from these
trials, the impact of Sangamo's clinical trials on the field of
genetic medicine and the benefit of orphan drug status. Actual
results may differ materially from these forward-looking statements
due to a number of factors, including uncertainties relating to
substantial dependence on the clinical success of lead therapeutic
programs, the initiation and completion of stages of our
clinical trials, whether the clinical trials will validate and
support the tolerability and efficacy of ZFNs, technological
challenges, Sangamo's ability to develop commercially viable
products and technological developments by our competitors. For a
more detailed discussion of these and other risks, please see
Sangamo's SEC filings, including the risk factors described in its
Annual Report on Form 10-K and its most recent Quarterly Report on
Form 10-Q. Sangamo Therapeutics, Inc. assumes no obligation to
update the forward-looking information contained in this press
release.
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/sangamo-therapeutics-receives-orphan-drug-designation-from-the-fda-for-sb-913-genome-editing-treatment-for-mps-ii-300415719.html
SOURCE Sangamo Therapeutics, Inc.