MaxCyte, Inc. Study Published in Science Translational Medicine
January 12 2017 - 2:00AM
RNS Non-Regulatory
TIDMMXCT
MaxCyte, Inc.
12 January 2017
MaxCyte, NIH NIAID Study Published in Science Translational
Medicine Demonstrates CRISPR-Cas9 X-Linked CGD Gene Repair
- MaxCyte's Proprietary GT(R) System fosters translational
development of ex vivo gene-corrected cell therapy as potential
'curative' treatment for monogenic diseases through robust,
scalable manufacture process
Gaithersburg, MD, January 12, 2017 - MaxCyte(R), Inc. (LSE:
MXCT), developer and supplier of cell engineering products and
technologies to biopharmaceutical firms engaged in cell therapy,
drug discovery and development, biomanufacturing, gene editing and
immuno-oncology, announced today pre-clinical results from a
collaborative study with investigators at the National Institutes
of Health's (NIH) National Institute of Allergy and Infectious
Diseases (NIAID). Results from the study demonstrated clinically
relevant levels of correction in the CYBB gene mutation in
long-term engrafted hematopoietic stem cells (HSC) and
differentiated neutrophils in cells obtained from individuals with
X-linked chronic granulomatous disease (X-CGD).
The study was published in Science Translational Medicine[i] and
entitled "CRISPR-Cas9 gene repair of hematopoietic stem cells from
patients with X-linked chronic granulomatous disease."
Using MaxCyte's patented Flow Electroporation(TM) Technology,
researchers demonstrated that transfecting three molecules, a
single-strand oligonucleotide correction template, along with
messenger RNA encoding for CRISPR-Cas9 gene editing complex and
selected guide RNA into HSC obtained from individuals with X-CGD,
resulted in correction of mutation in the CYBB gene. This
correction occurred at clinically relevant levels following
long-term engraftment in preclinical models.
"We are very encouraged by the preclinical data obtained in this
study, which showed the effectiveness of MaxCyte's proprietary,
high-performance technology to correct for genetic mutations,
especially in cases where traditional viral vector-based gene
insertion methods have not been effective," said Madhusudan V.
Peshwa, Ph.D., MaxCyte's Chief Scientific Officer. "This study also
demonstrates the commitment of MaxCyte to fostering development of
novel cell therapies and supporting our partners' use of our
delivery platform. MaxCyte invests early in translational
development of non-virally engineered and ex vivo gene-edited cell
therapy products."
CGD is an inherited genetic disorder that impairs the function
of the immune system and leads to ongoing and severe bacterial
infections. The disease affects approximately one in 250,000 people
worldwide, according to MedScape, and is currently only treatable
through high-risk treatments, such as allogeneic bone marrow
transplantation. It is caused by a mutation in the CYBB gene, that
enables immune cells to defend against microbes. The MaxCyte and
NIAID researchers were able to restore function of the immune cells
by repairing this mutation in CYBB.
About MaxCyte
MaxCyte is a developer and supplier of cell engineering products
and technologies to biopharmaceutical firms engaged in cell
therapy, drug discovery and development, biomanufacturing, gene
editing and immuno-oncology markets. The Company's patented Flow
Electroporation(TM) Technology enables its products to deliver
fast, reliable and scalable cell engineering to drive the research
and clinical development of a new generation of medicines.
MaxCyte's high performance platform allows transfection with any
molecule or multiple molecules and is compatible with nearly all
cell types, including hard-to-transfect human primary cells. It
also provides a high degree of consistency and minimal cell
disturbance, thereby facilitating rapid, large scale, clinical and
commercial grade cell engineering in a non-viral system and with
low toxicity concerns. The Company's cell engineering technology
platform is CE-marked and FDA-accredited, providing MaxCyte's
customers and partners with an established regulatory path.
Using the unique capabilities of its technology, MaxCyte is
developing CARMA, its proprietary platform in immuno-oncology, to
deliver a validated non-viral approach to CAR therapies across a
broad range of cancer indications, including solid tumors where
existing CAR-T approaches face significant challenges.
For more information, visit http://www.maxcyte.com/
MaxCyte +1 301 944 1660
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Officer
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Officer
Nominated Adviser and Broker
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Finance)
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Financial PR Adviser +44 (0)203 709 5700
Consilium Strategic Communications maxcyte@consilium-comms.com
Mary-Jane Elliott
Chris Welsh
Lindsey Neville
[i] De Ravin, et al. (2017). CRISPR-Cas9 gene repair of
hematopoietic stem cells from patients with X-linked chronic
granulomatous disease. Science Translational Medicine, Vol 9: Issue
372.
This information is provided by RNS
The company news service from the London Stock Exchange
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