RICHMOND, Calif., Aug. 25, 2017 /PRNewswire/
-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO) and Pfizer Inc.
(NYSE: PFE) today announced that the first patient received
treatment in the Phase 1/2 clinical trial ("the Alta trial")
evaluating SB-525, an investigational gene therapy for patients
with Hemophilia A, a rare genetic blood disorder.
"We are excited to announce the first patient infusion in this
initial clinical trial of SB-525. Gene therapy holds promise as a
potential one-time treatment to control Hemophilia A and prevent
spontaneous bleeding," said Dr. Sandy
Macrae, Chief Executive Officer of Sangamo Therapeutics.
The Alta trial is an open-label, adaptive, dose-ranging clinical
study designed to assess the safety and tolerability of SB-525
investigational gene therapy in up to 20 adult subjects with severe
Hemophilia A. SB-525 is being developed as part of a global
collaboration between Sangamo and Pfizer for the development and
commercialization of gene therapy programs for Hemophilia A.
"We are excited to see progress in our partnership program for
SB-525 with Sangamo," said Greg
LaRosa, Chief Scientific Officer, Pfizer Rare Disease. "By
combining Sangamo's scientific and technical expertise across
multiple genomic platforms, with Pfizer's global reach, resources
and expertise, we are advancing the promise of gene therapy with
the hope of making a meaningful impact for people living with
Hemophilia A."
SB-525 comprises a recombinant adeno-associated virus (rAAV)
vector carrying a Factor VIII gene construct driven by a
proprietary, synthetic, liver-specific promoter. The U.S. Food and
Drug Administration has granted Orphan Drug and Fast Track
designations to SB-525, which also received Orphan Medicinal
Product designation from the European Medicines Agency. Hemophilia
A is a rare blood disorder caused by a genetic mutation resulting
in insufficient activity of Factor VIII, a blood clotting protein
the body uses to stop bleeding. There are approximately 16,000
patients in the U.S. and more than 150,000 worldwide with
Hemophilia A.
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 has open Phase 1/2 clinical trials in Hemophilia A and
Hemophilia B, and lysosomal storage disorders MPS I and MPS II.
Sangamo has an exclusive, global collaboration and license
agreement with Pfizer Inc. for gene therapy programs for Hemophilia
A, 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.
About Pfizer Inc.: Working together for a healthier
worldTM
At Pfizer, we apply science and our
global resources to bring therapies to people that extend and
significantly improve their lives. We strive to set the standard
for quality, safety and value in the discovery, development and
manufacture of health care products. Our global portfolio includes
medicines and vaccines as well as many of the world's best-known
consumer health care products. Every day, Pfizer colleagues work
across developed and emerging markets to advance wellness,
prevention, treatments and cures that challenge the most feared
diseases of our time. Consistent with our responsibility as one of
the world's premier innovative biopharmaceutical companies, we
collaborate with health care providers, governments and local
communities to support and expand access to reliable, affordable
health care around the world. For more than 150 years, we have
worked to make a difference for all who rely on us. We routinely
post information that may be important to investors on our website
at www.pfizer.com. In addition, to learn more, please visit us on
www.pfizer.com and follow us on Twitter at @Pfizer and
@Pfizer_News, LinkedIn, YouTube and like us on Facebook at
Facebook.com/Pfizer.
Forward Looking Statements
This press release
contains forward-looking statements, including, but not limited to,
statements related to the therapeutic potential of gene
therapy and SB-525, the number of patients to be
treated in the Alta trial and other statements that are not
historical facts. These forward-looking statements are based on
Sangamo's current plans, objectives, estimates, expectations and
intentions and inherently involve significant risks and
uncertainties. Actual results and the timing of events could differ
materially from those anticipated in such forward-looking
statements as a result of these risks and uncertainties, which
include, without limitation, risks and uncertainties associated
with: gene therapy product candidate development and the
uncertainty of clinical success, including
whether the Alta trial
will validate and support the tolerability and efficacy of SB-525;
Sangamo's substantial dependence on the clinical success of its
lead therapeutic programs, including SB-525; the initiation,
enrollment and completion of the stages of the Alta trial and its
other clinical trials; technological challenges; the lengthy and
uncertain regulatory approval process; Sangamo's ability to develop
commercially viable products; technological developments by its
competitors; and Sangamo's dependence on its collaboration with
Pfizer and its ability to maintain its collaboration with Pfizer. A
more detailed discussion of these and other risks and uncertainties
may be found under the caption "Risk Factors" and elsewhere in
Sangamo's SEC filings and reports, including Sangamo's Quarterly
Report on Form 10-Q for the quarter ended June 30, 2017 and future filings and reports by
Sangamo. Sangamo assumes no obligation to update the
forward-looking information contained in this press
release.
View original content with
multimedia:http://www.prnewswire.com/news-releases/sangamo-and-pfizer-announce-first-patient-receives-treatment-in-phase-12-clinical-trial-evaluating-sb-525-investigational-gene-therapy-for-hemophilia-a-300509560.html
SOURCE Sangamo Therapeutics, Inc.