Sangamo Announces UK Authorization of a Phase 1/2 Clinical Trial Evaluating the CAR-Treg Cell Therapy TX200 for Kidney Transp...
November 19 2019 - 07:00AM
Business Wire
The STEADFAST clinical study of TX200 is
the first-in-human trial to evaluate a CAR-Treg cell therapy
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, announced today that the United Kingdom’s (UK) Medicines
Healthcare Products Regulatory Agency (MHRA) has granted
authorization of the first-in-human clinical trial to evaluate a
Chimeric Antigen Receptor Regulatory T Cell (CAR-Treg) therapy.
TX200 is being studied for the prevention of immune-mediated
rejection following HLA-A2 mismatched kidney transplantation for
end-stage renal disease (ESRD). Sangamo expects to open the first
clinical sites for the STEADFAST study in 2020.
“Being the first company to test a CAR-Treg candidate in humans
is an important milestone for Sangamo and this exciting new
frontier of cellular therapy. We believe that the TX200 program
will be invaluable in expanding our understanding of the safety and
mechanism of action of CAR-Treg cells and their relevance in the
clinic,” said Adrian Woolfson, BM, BCh, PhD, Head of Research and
Development at Sangamo. “This innovative and personalized cellular
therapy approach for HLA-A2 mismatched kidney transplantation is
designed to help regulate the body’s immune system specifically and
locally to promote acceptance of an immunologically mismatched
donor organ. Beyond transplantation, we plan to explore the
potential of CAR-Tregs in a range of autoimmune and inflammatory
diseases.”
TX200 is an autologous (cells collected from the patient)
HLA-A2-targeted CAR-Treg cell therapy. The patient’s regulatory T
cells (Tregs), a type of white blood cell which plays a key role in
regulating the immune response and inflammation, are collected and
genetically engineered with a Chimeric Antigen Receptor (CAR)
designed to bind to HLA-A2. HLA-A2 is a protein belonging to the
human leukocyte antigen (HLA) system. Donor/recipient mismatch in
HLA molecules is a primary contributor to organ transplant
incompatibility and may ultimately lead to immune-mediated
rejection of the transplanted organ. The recipient’s immune system
can recognize this HLA-A2 mismatch and may attack the new kidney
carrying the HLA-A2 protein, potentially leading to graft
rejection.
TX200 HLA-A2 CAR-Treg cells are designed to accumulate and
localize within the new kidney where the HLA‑A2 protein is present,
thereby utilizing the ability of Tregs to suppress immune responses
against the transplanted kidney. This approach aims to help the
recipient’s immune system accept the new kidney and induce
immunological tolerance, potentially allowing tapering and eventual
elimination of accompanying standard-of-care immune suppressive
treatments.
Kidney transplantation is the treatment of choice for patients
with ESRD who must otherwise remain on long-term dialysis. To
prevent graft rejection, transplanted patients are treated with
lifelong immune suppressive therapy, which impacts the body’s
immune system broadly and is associated with multiple side effects,
including an increased risk of infectious complications, cancer,
and other drug-related toxicities.
TX200, which has been developed in collaboration with Professor
Megan Levings of the University of British Columbia1, entered
Sangamo’s pipeline in 2018 as part of the acquisition of TxCell.
Beyond kidney transplantation, Sangamo intends to use its zinc
finger nuclease (ZFN) gene editing technology to develop
next-generation autologous and allogeneic CAR-Treg cell therapies
for use in treating autoimmune and inflammatory diseases.
About the STEADFAST study
The STEADFAST study is a multicenter, open-label, single
ascending dose, dose-ranging Phase 1/2 study. Sangamo plans to run
this study in five different countries in Europe: United Kingdom,
France, Netherlands, Germany, and Belgium.
About Sangamo Therapeutics
Sangamo Therapeutics is committed to translating ground-breaking
science into genomic medicines with the potential to transform
patients’ lives using gene therapy, ex vivo gene-edited cell
therapy, and in vivo genome editing and gene regulation. For more
information about Sangamo, visit www.sangamo.com.
- JCI Insight. 2019;4(6):e123672.
https://doi.org/10.1172/jci.insight.123672.
Forward Looking Statements
This press release contains forward-looking statements regarding
Sangamo's current expectations. These forward-looking statements
include, without limitation, statements regarding the potential
benefits of cell therapy, including but not limited to Treg
therapy, the Company's ability to develop and commercialize product
candidates to address genetic diseases with the Company's
proprietary technologies and the timing of commencement or next
stages of such programs and the anticipated benefits therefrom.
These statements are not guarantees of future performance and are
subject to certain risks, uncertainties and assumptions that are
difficult to predict. Factors that could cause actual results to
differ include, but are not limited to, the outcomes of clinical
trials, the uncertain regulatory approval process, uncertainties
related to the execution of clinical trials and uncertainties that
research outcomes will support clinical programs. Actual results
may differ from those projected in forward-looking statements due
to risks and uncertainties that exist in Sangamo's operations and
business environments. These risks and uncertainties are described
more fully in Sangamo's Annual Report on Form 10-K for the year
ended December 31, 2018 as filed with the Securities and Exchange
Commission on March 1, 2019 and Sangamo's Quarterly Report on Form
10-Q for the quarter ended September 30, 2019 that it filed on
November 6, 2019. TX200 is an investigational product and has not
been determined to be safe or efficacious by regulatory
authorities. Forward-looking statements contained in this
announcement are made as of this date, and Sangamo undertakes no
duty to update such information except as required under applicable
law.
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version on businesswire.com: https://www.businesswire.com/news/home/20191119005346/en/
Investor Relations – Global McDavid
Stilwell 510-970-6000, x219 mstilwell@sangamo.com
Media Inquiries – Global Aron
Feingold 510-970-6000, x421 afeingold@sangamo.com
Investor Relations and Media Inquiries –
European Union & United Kingdom Caroline Courme 33 4 97
21 27 27 ccourme@sangamo.com
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