RICHMOND, Calif., Jan. 8, 2015 /TRENDINGGLOBALNEWS/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO)
announced that the company has in-licensed nanoparticle technology to
enable systemic messenger RNA (mRNA) delivery of Sangamo's proprietary
zinc finger nucleases (ZFNs). mRNA delivery of ZFNs is being used in
the development of the next generation of Sangamo's ZFP Therapeutics®.
"We
continue to identify strategies to leverage and maximize the value of
our ZFN platform by enhancing and broadening the application of
Sangamo's ZFP technology into new therapeutic opportunities," stated Edward Lanphier,
Sangamo's president and CEO. "Nucleic acid delivery technologies have
significantly matured over the past several years and mRNA delivery is
coming of age. While we already employ mRNA delivery in our ex vivo genome editing programs, novel mRNA formulations potentially enable in vivo ZFN applications such as the knock-out of well-established repression targets in the liver."
"Specifically,
this strategy opens up an entirely new set of therapeutic
genome-editing targets," added Mr. Lanphier. "mRNA delivery could enable
progressive and permanent knock out of a gene at the DNA level versus
repressing its expression using continued and chronic administration of
antisense or RNAi therapeutics."
Because
RNA is rapidly turned over in cells and tissues, conventional RNA-based
drugs such as antisense and RNAi must be repeatedly administered to
maintain a therapeutic effect. In contrast, ZFNs need to be present
only transiently to enable permanent changes in the genome of cells, and
so are ideally suited for mRNA delivery.
This
delivery mode has several advantages. The potential to dose patients
multiple times provides the opportunity to "dose to effect" or to
administer the ZFNs in several treatments until a sufficient level of
permanent modification is obtained to achieve a life-long therapeutic
outcome.
This method potentially expands the application of ZFN in vivo genome-editing to numerous therapeutic "knock-out" targets such as PCSK9, which encodes a protein involved in cholesterol homeostasis. Knocking out the PCSK9 gene
is expected to lower levels of low-density lipoprotein cholesterol
(LDL-C), or "bad" cholesterol, which is considered a risk factor for
cardiovascular disease.
In
addition, the mRNA delivery method could be used in the development of
second generation ZFP Therapeutics to broaden the patient group that can
be treated using Sangamo's In Vivo Protein Replacement Platform (IVPRP)
which is being developed as a generally applicable strategy to provide a
permanent genetic cure for certain monogenic diseases.
Background on Sangamo's IVPRP Approach
The
IVPRP approach makes use of the albumin gene locus, a highly expressing
and liver-specific genomic "safe-harbor site", that can be edited with
zinc finger nucleases (ZFNs) to accept and express any therapeutic gene.
The platform enables the patient's liver to permanently produce
therapeutic levels of a corrective protein product such as factor VIII
or IX to treat hemophilia, or replacement enzymes to treat Lysosomal
Storage Disorders. With such a large capacity for protein production
(approximately 15g/day of albumin), which is in excess of the body's
requirements, 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 Sangamo
Sangamo BioSciences, Inc. is focused on Engineering Genetic CuresTM for
monogenic and infectious diseases by deploying its novel DNA-binding
protein technology platform in therapeutic gene regulation and genome
editing. The Company has ongoing Phase 2 clinical trials to evaluate the
safety and efficacy of a novel ZFP Therapeutic® for the
treatment of HIV/AIDS (SB-728-T) and NGF-AAV for Alzheimer's disease
(CERE-110). Sangamo's other therapeutic programs are focused on
monogenic and rare diseases. The company has formed a strategic
collaboration with Shire International GmbH to develop therapeutics for
hemophilia, Huntington's disease and other monogenic diseases, and with
Biogen Idec for hemoglobinopathies, such as sickle cell disease and
beta-thalassemia. It has also 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 mRNA technologies to expand
the therapeutic application of ZFNs, research and development of novel
ZFP TFs and ZFNs, their delivery, and therapeutic applications of
Sangamo's ZFP technology platform for the treatment of monogenic and
infectious diseases. Actual results may differ materially from these
forward-looking statements due to a number of factors, including
uncertainties relating to technological challenges, the initiation and
completion of stages of our clinical trials, whether the clinical trials
will validate and support the tolerability and efficacy of ZFNs and ZFP
TFs, 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.
source:http://www.prnewswire.com/
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