RICHMOND, Calif., Sept. 6, 2016 /PRNewswire/ -- Sangamo
BioSciences, 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-FIX, the company's
zinc finger nuclease (ZFN)-mediated genome editing product
candidate for the treatment of hemophilia B. Sangamo expects to
initiate a Phase 1/2 clinical study (SB-FIX-1501) in adult subjects
with the disease in 2016.
"We are pleased with the FDA's decision to grant orphan drug
designation to SB-FIX for hemophilia B," said Geoff Nichol, M.B., Ch.B., Sangamo's executive
vice president of research and development. "By enabling targeted
integration of a therapeutic factor 9 gene, our ZFN-mediated genome
editing approach may offer hemophilia B patients a therapeutic
option that has potential advantages over conventional gene therapy
approaches. We will enroll adult hemophilia patients into our first
clinical trial, however, our goal is to move into pediatric
patients, a population we believe could particularly benefit from a
treatment that has the potential to provide lifelong expression of
therapeutic levels of Factor IX protein."
"Our mission is to translate our ground-breaking science into
genetic therapies that transform patients' lives," said
Sandy Macrae, M.B., Ch.B., Ph.D.,
Sangamo's president and chief executive officer. "This is another
positive step forward for the first therapeutic in vivo
genome editing application cleared for clinical evaluation in
humans. I am pleased with the progress that we have made to advance
this program and we look forward to initiating a Phase 1/2 clinical
trial by the end of 2016."
The FDA grants orphan drug designation to investigational drugs
and biologics that are intended for the treatment of rare diseases
that affect fewer than 200,000 people in the U.S. Orphan drug
status is intended to facilitate drug development for rare diseases
and provides several benefits to drug developers, including
assistance with clinical study design and drug development, tax
credits for qualified clinical trials costs, exemptions from
certain FDA application fees, and seven years of market exclusivity
upon regulatory product approval.
Sangamo's In Vivo Genome Editing
Approach
SB-FIX is designed as a single treatment strategy
intended to provide stable, continuous production of Factor IX
clotting protein (FIX) for the lifetime of the patient. Sangamo's
ZFN-mediated in vivo genome editing approach makes use of
the albumin gene locus, a highly expressing and liver-specific
genomic "safe-harbor 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 product. 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
approaches, which are non-integrating, and may "wash out" of the
liver as cells divide and turn over. 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. With such a large capacity for protein production
(approximately 15g/day of albumin), targeting and co-opting only a
very small percentage of the albumin gene's capacity is sufficient
to produce the needed replacement protein at therapeutically
relevant levels with no significant effect on albumin
production.
About Hemophilia B
Hemophilia, a rare bleeding
disorder in which the blood does not clot normally, is caused by
mutations in genes that encode factors which help the blood clot
and stop bleeding when blood vessels are injured. Hemophilia B is
caused by a defect in the gene encoding clotting Factor IX protein
and individuals with this mutation experience bleeding episodes
after injuries and spontaneous bleeding episodes that often lead to
joint disease such as arthritis. According to the National
Hemophilia Foundation and the World Federation of Hemophilia,
hemophilia B occurs in about one in every 50,000 male births with
approximately 4,000 males currently affected in the U.S. The
current standard treatment for individuals with hemophilia is
protein replacement of the defective clotting factor with regular
infusion of recombinant clotting factors or plasma concentrates.
These therapies are expensive and sometimes stimulate the body to
produce antibodies that inhibit the benefits of treatment. The most
severe forms of hemophilia B require the need for ongoing,
preventive infusions.
About Sangamo
Sangamo BioSciences, Inc. is focused on
Engineering Genetic Cures® for monogenic and infectious
diseases by deploying its novel zinc finger DNA-binding protein
technology, in therapeutic genome editing and gene regulation, and
AAV-based gene therapy platforms. The Company's proprietary
ZFN-mediated in vivo genome editing approach is focused on
monogenic diseases, including hemophilia and lysosomal storage
disorders. Based on its in vivo genome editing approach,
Sangamo is initiating a Phase 1/2 clinical trial for hemophilia B,
the first in vivo genome editing application cleared by the
FDA. In addition, Sangamo has a Phase 2 clinical program to
evaluate the safety and efficacy of novel ZFP
Therapeutics® for the treatment of HIV/AIDS (SB-728).
The Company has also formed a strategic collaboration with Biogen
Inc. for hemoglobinopathies, such as sickle cell disease and
beta-thalassemia, and with Shire International GmbH to develop
therapeutics for Huntington's disease. It has established strategic
partnerships with companies in non-therapeutic applications of its
technology, including Dow AgroSciences and Sigma-Aldrich
Corporation. For more information about Sangamo, visit the
Company's website at www.sangamo.com.
ZFP Therapeutic® is a registered trademark of
Sangamo BioSciences, Inc.
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 novel ZFP TFs and ZFNs and therapeutic
applications of Sangamo's ZFP technology platform, the potential of
Sangamo's ZFP technology to treat hemophilia B, and lysosomal
storage disorders, the expected timing of trial enrollment for
SB-FIX-1501, the impact of the SB-FIX clinical trial on the field
of genetic medicine, the benefit of orphan drug status, and the
safety and efficacy of the approach of using ZFN-mediated genome
editing. Actual results may differ materially from these
forward-looking statements due to a number of factors, including
uncertainties relating to the initiation and completion of stages
of our clinical trials, whether the clinical trials will validate
and support the safety, tolerability and efficacy of ZFNs and ZFP
TFs, 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 public filings with the Securities and
Exchange Commission, including the risk factors described in its
Annual Report on Form 10-K and its most recent Quarterly Report on
Form 10-Q. Sangamo assumes no obligation to update the
forward-looking information contained in this press
release.
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