RICHMOND, Calif., Nov. 15, 2017 /PRNewswire/
-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO) today announced
treatment of the first patient in the Phase 1/2 clinical trial
("the CHAMPIONS study") evaluating SB-913, an investigational in
vivo genome editing therapy for people with
mucopolysaccharidosis type II (MPS II), also known as Hunter
syndrome.
"For the first time, a patient has received a therapy intended
to precisely edit the DNA of cells directly inside the body. We are
at the start of a new frontier of genomic medicine," said Dr.
Sandy Macrae, CEO of Sangamo
Therapeutics.
Sangamo aims to treat MPS II by using genome editing to insert a
corrective gene into a precise location in the DNA of liver cells
with the goal of enabling a patient's liver to produce a lifelong
and stable supply of an enzyme he or she currently lacks.
Without that enzyme, called iduronate-2-sulfatase (IDS), people
with MPS II suffer debilitating buildup of toxic carbohydrates in
cells throughout their body. Approximately one in 100,000 to one in
170,000 people are born with MPS II. Many people with MPS II
receive weekly infusions of enzyme replacement therapy (ERT), the
current standard-of-care treatment. Within a day of receiving ERT,
however, IDS quickly returns to near undetectable levels in the
blood.
"Even with regular infusions of ERT, which has markedly improved
functional health outcomes, patients endure progressive damage to
heart, bones and lungs. Many patients with MPS II die of airway
obstruction, upper respiratory infection or heart failure before
they reach the age of 20," said Paul
Harmatz, M.D., a pediatric gastroenterologist and a
principal investigator for the CHAMPIONS study at the UCSF Benioff
Children's Hospital Oakland, where the first subject in the study
was treated.
The CHAMPIONS study, which is also screening subjects at
hospitals specializing in the care of patients with MPS II,
including hospitals in Chapel Hill, Chicago, Minneapolis and Philadelphia, is an open-label clinical study
designed to assess the safety, tolerability and preliminary
efficacy of the SB-913 investigational genome editing therapy in up
to nine adult males with MPS II.
SB-913 makes use of Sangamo's zinc finger nuclease (ZFN) genome
editing technology to insert a corrective gene into a precise
location in the DNA of liver cells. To restrict editing to liver
cells, the ZFNs and the corrective gene are delivered in a single
intravenous infusion using AAV vectors that target the liver. The
ZFNs enter the cells as inactive DNA instructions in a format
designed only for liver cells to unlock. Once "unlocked", the ZFNs
then identify, bind to and cut the DNA in a specific location
within the albumin gene. Using the cells' natural DNA repair
processes, liver cells can then insert the corrective gene for IDS
at that precise location.
The ability to permanently and precisely integrate the
therapeutic IDS gene into the DNA differentiates Sangamo's in
vivo genome editing approach from conventional AAV cDNA gene
therapy and from lenti- or retroviral-based gene therapies that
insert genes randomly into the genome.
Two additional clinical trials are underway in the United States to evaluate Sangamo's in
vivo genome editing therapeutics for hemophilia B and MPS I,
which is also known as Hurler or Hurler-Scheie syndrome. All three
trials use ZFNs designed to edit liver cells at the same location
in the albumin gene, but differ in delivering the corrective gene
relevant to the respective disease.
"As a physician, I feel a real sense of responsibility toward
the patients who are participating in these three clinical trials,"
said Ed Conner, M.D., chief medical
officer of Sangamo. "We have been working closely with the FDA and
the NIH Recombinant DNA Advisory Committee to make sure that we are
thoroughly and prudently developing this new class of
medicines."
All three of Sangamo's in vivo genome editing product
candidates have received Fast Track and Orphan Drug designations
from the U.S. Food and Drug Administration (FDA). Additionally,
SB-318 for MPS I and SB-913 for MPS II have received Rare Pediatric
Disease designations from the FDA.
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. For more information about Sangamo,
visit 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, including MPS
I and MPS II, and the impact of Sangamo's clinical trials on the
field of genetic medicine. 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 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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SOURCE Sangamo Therapeutics, Inc.