Sleeping Beauty System Used to Co-express CAR with Membrane-Bound IL-15 to Enhance Persistence of CD19-Specific T Cells
November 15 2016 - 7:00AM
ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company
focused on new immunotherapies, today announced the publication of
data demonstrating enhanced persistence of genetically modified T
cells targeting leukemia through utilization of its non-viral
Sleeping Beauty (SB) system to co-express membrane-bound IL-15
(mbIL15) and a CD19-specific chimeric antigen receptor (CAR). The
article, titled “Tethered IL-15 augments antitumor activity and
promotes a stem-cell memory subset in tumor-specific T cells,” was
published in the Proceedings of the National Academy of Sciences
(PNAS) and is available online here.
Using the SB system, researchers generated
genetically modified T cells that preserved stem-cell memory (TSCM)
by co-expressing the CAR with a fusion variant of IL-15. These
engineered T cells were effective in treating established CD19+
leukemia in mice by facilitating the long-term persistence of TSCM
cells sustained by signaling through mbIL15. These findings provide
for a translational pipeline of immunotherapies with improved
potential by combining mbIL15 and T cells with diverse
specificities.
“The ability to generate CAR-T cells with
preserved stem-cell memory is a novel strategy for promoting
long-lived persistence and effectiveness of immunotherapies for the
treatment of patients with cancers. Producing this rare, but highly
desirable, T-cell subset has historically been a challenge,” said
Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM
and an author of the publication.
“We have demonstrated the ability to incorporate
membrane-bound IL-15 via the non-viral Sleeping Beauty platform,
thereby enhancing T-cell survival and raising our expectations for
corresponding therapeutic benefit. The fundamental role that IL-15
plays in T-cell activation and propagation makes it an attractive
candidate to incorporate into engineered immunotherapies, and we
are advancing CAR-modified T cells co-expressing mbIL15 to testing
in humans,” added Dr. Cooper.
The SB transposon-transposase is a unique
non-viral system for introducing genes into cells and is
exclusively licensed by Intrexon Corporation (NYSE:XON) through The
University of Texas MD Anderson Cancer Center and accessed as part
of ZIOPHARM's collaboration with Intrexon.
About ZIOPHARM Oncology,
Inc.:
ZIOPHARM Oncology is a Boston,
Massachusetts-based biotechnology company employing novel gene
expression, control and cell technologies to deliver safe,
effective and scalable cell- and viral-based therapies for the
treatment of cancer and graft-versus-host-disease. The Company's
immuno-oncology programs, in collaboration with Intrexon
Corporation (NYSE:XON) and the MD Anderson Cancer Center, include
chimeric antigen receptor T cell (CAR-T) and other adoptive
cell-based approaches that use non-viral gene transfer methods for
broad scalability. The Company is advancing programs in multiple
stages of development together with Intrexon Corporation's
RheoSwitch Therapeutic System® technology, a switch to turn on and
off, and precisely modulate, gene expression in order to improve
therapeutic index. The Company's pipeline includes a number of
cell-based therapeutics in both clinical and preclinical testing
which are focused on hematologic and solid tumor malignancies.
Forward-Looking Safe-Harbor
Statement:
This press release contains certain
forward-looking information about ZIOPHARM Oncology, Inc. that is
intended to be covered by the safe harbor for "forward-looking
statements" provided by the Private Securities Litigation Reform
Act of 1995, as amended. Forward-looking statements are statements
that are not historical facts, and in some cases can be identified
by terms such as "may," "will," "could," "expects," "plans,"
"anticipates," and "believes." These statements include, but are
not limited to, statements regarding the Company's plans and
expectations regarding its securities offerings, fundraising
activities and financial strategy, the progress, timing and results
of preclinical and clinical trials involving the Company's drug
candidates, and the progress of the Company's research and
development programs. All of such statements are subject to certain
risks and uncertainties, many of which are difficult to predict and
generally beyond the control of the Company, that could cause
actual results to differ materially from those expressed in, or
implied by, the forward-looking statements. These risks and
uncertainties include, but are not limited to: our ability to
finance our operations and business initiatives and obtain funding
for such activities, whether chimeric antigen receptor T cell (CAR
T) approaches, Ad-RTS-hIL-12, TCR and NK cell-based therapies, or
any of our other therapeutic candidates will advance further in the
pre-clinical or clinical trials process and whether and when, if at
all, they will receive final approval from the U.S. Food and Drug
Administration or equivalent foreign regulatory agencies and for
which indications; whether chimeric antigen receptor T cell (CAR T)
approaches, Ad-RTS-hIL-12, TCR and NK cell-based therapies, and our
other therapeutic products will be successfully marketed if
approved; the strength and enforceability of our intellectual
property rights; competition from other pharmaceutical and
biotechnology companies; and the other risk factors contained in
our periodic and interim SEC reports filed from time to time with
the Securities and Exchange Commission, including but not limited
to, our Annual Report on Form 10-K for the fiscal year ended
December 31, 2015, and our Quarterly Report for the quarter ended
June 30, 2016. Readers are cautioned not to place undue reliance on
these forward-looking statements that speak only as of the date
hereof, and we do not undertake any obligation to revise and
disseminate forward-looking statements to reflect events or
circumstances after the date hereof, or to reflect the occurrence
of or non-occurrence of any events.
Trademarks
RheoSwitch Therapeutic System® (RTS®)
technology is a registered trademark of Intrexon Corporation.
Contact:
Lori Ann Occhiogrosso
ZIOPHARM Oncology, Inc.
617-259-1987
locchiogrosso@ziopharm.com
David Pitts
Argot Partners
212-600-1902
david@argotpartners.com
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