MaxCyte, Inc. Presentation of Positive Preclinical Data
25 April 2017 - 4:01PM
RNS Non-Regulatory
TIDMMXCT
MaxCyte, Inc.
25 April 2017
MaxCyte, Inc.
("MaxCyte" or the "Company")
MaxCyte, Inc. to Present Positive Preclinical Data for Sickle
Cell Disease
Gaithersburg, Maryland - April 25, 2017: MaxCyte (LSE: MXCT,
MXCR) a US-based global company dedicated to driving the
acceleration of discovery, development, manufacturing and
commercialization of next-generation, cell-based medicines,
announced today it will present positive preclinical study results
at the American Society of Gene and Cell Therapy Annual Meeting
(ASGCT). The new in vitro data demonstrate the potential of
MaxCyte's cGMP-compliant proprietary delivery platform to enable
CRISPR gene editing in the treatment of sickle cell disease (SCD).
The ASGCT meeting will take place in Washington, DC from May 10-13,
2017.
Using MaxCyte's Proprietary GT(R) System, MaxCyte and its
collaborators at the National Heart, Lung and Blood Institute
(NHLBI) and National Institute of Allergy and Infectious Diseases
(NIAID) demonstrated successful CRISPR-induced corrections of the
mutation behind SCD in more than 30 percent of patient-derived B
cells. The objective of this pre-clinical effort is to develop
potential curative therapy based on "correcting" the faulty gene in
SCD while concurrently addressing DNA mutation sites in
non-corrected cells that further contribute to the disease in
people living with SCD. In contrast, competing approaches focus on
either external therapeutic gene addition using viral vectors (now
in early clinical trials) or on creation of hereditary persistence
of fetal hemoglobin (HPFH) mutations (preclinical research
underway). Neither of these approaches impact the level of faulty
HbS levels, which is the target of MaxCyte's approach and which are
at the core of SCD.
"These data represent an extension of our previously published
work achieving gene correction in X-linked chronic granulomatous
disease, demonstrating the ability of our cGMP-compliant delivery
platform to achieve robust, scalable, and a clinically relevant
levels of gene correction in sickle cell disease patient-derived
cells," said Doug Doerfler, Chief Executive Officer of MaxCyte, "We
are excited by these results, and look forward to evaluating
long-term data."
Data will be shared via a poster (Permanent Abstract Number:
642) at the ASGCT Annual Meeting with details as follows: Session:
"Clinical-Meaningful Level CRISPR-Oligomer-Mediated Correction of
Sickle Cell Disease (SCD) Using Non-viral, cGMP Compliance,
Scalable and Closed System." Timing: Friday, May 12, 2017 at 5:45 -
7:45 p.m. ET
About Sickle Cell Disease
According to the NHLBI, the term sickle cell disease (SCD)
describes a group of inherited red blood cell disorders. Patients
with SCD have abnormal hemoglobin, called hemoglobin S or sickle
hemoglobin, in their red blood cells. Hemoglobin is a protein in
red blood cells that carries oxygen throughout the body. Those who
have SCD inherit two abnormal hemoglobin genes, one from each
parent. In all forms of SCD, at least one of the two abnormal genes
cause a person's body to make hemoglobin S.
Sickle cell disease is a life-long illness. The severity of the
disease varies widely from person to person. In high-income
countries like the United States, according to NHLBI, the life
expectancy of a person with SCD is now about 40-60 years.
Currently, hematopoietic stem cell transplantation (HSCT) is the
only cure for SCD. Unfortunately, most people with SCD are either
too old for a transplant or don't have a relative who is a good
enough genetic match for them to act as a donor. A well-matched
donor is needed to have the best chance for a successful
transplant. While there are some treatments that can reduce
symptoms and prolong life, more medical options are needed.
About MaxCyte
MaxCyte (LSE: MXCT, MXCR) is a US-based global company dedicated
to driving the acceleration of the discovery, development,
manufacturing and commercialization of next-generation, cell-based
medicines. The Company provides its patented, high-performance cell
engineering platform to biopharmaceutical partners engaged in drug
discovery and development, biomanufacturing, and cell therapy,
including gene editing and immuno-oncology. With its robust
delivery platform, MaxCyte's team of scientific experts helps its
partners to unlock their product potential and solve problems. This
platform allows for the engineering of nearly all cell types,
including human primary cells, with any molecule, at any scale. 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 platform is
FDA-accredited, providing MaxCyte's customers and partners with an
established regulatory path to commercialize cell-based medicines.
MaxCyte is also developing CARMA, its proprietary, breakthrough
platform in immuno-oncology, to rapidly manufacture CAR therapies
for 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
Doug Doerfler, Chief Executive
Officer
Ron Holtz, Chief Financial
Officer
Nominated Adviser and Broker +44 (0) 20 7886 2500
Panmure Gordon (UK) Limited
Freddy Crossley (Corporate
Finance)
Duncan Monteith
Ryan McCarthy
Tom Salvesen (Corporate Broking)
+44 (0)20 3709 5700
Financial PR Adviser maxcyte@consilium-comms.com
Consilium Strategic Communications
Mary-Jane Elliott
Chris Welsh
Lindsey Neville
This information is provided by RNS
The company news service from the London Stock Exchange
END
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