MaxCyte, Inc. to Present Pre-Clinical Data for Targeted Gene-Correction in a Rare Disease at Keystone Symposia on Precision Genome Engineering
– MaxCyte’s Proprietary GT® System enabled a robust, scalable manufacturing process for ex vivo gene-corrected cell therapy that may serve as a potential treatment for chronic granulomatous disease (CGD) and other monogenic diseases
– Co-transfection of messenger RNA encoding for CRISPR/Cas9, guide RNA molecules, and modified single-strand oligonucleotide as donor template mediated correction of mutated CYBB gene in hematopoietic stem cells obtained from X-linked CGD patients
Gaithersburg, MD, January 4, 2017 – MaxCyte®, Inc., 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, announces data to be presented at the Keystone Symposia on Precision Genome Engineering summarizing in vitro and long-term preclinical toxicity and engraftment studies following targeted gene correction aimed at reversing mutations to wild-type sequence at clinically relevant levels in CD34+ hematopoietic stem cells (HSC) obtained from individuals with X-CGD. The oral and poster presentations will highlight use of MaxCyte’s proprietary, cGMP-compliant delivery platform in development of ex vivo gene-corrected cell therapies as a potential treatment for monogenic diseases. The Symposia is taking place January 8 to 12, 2017 in Breckenridge, Colorado.
The presentation entitled “Mutation Correction of X-linked Chronic Granulomatous Disease,” summarizes pre-clinical work of MaxCyte and collaborators at the National Institute of Allergy and Infectious Diseases (NIAID), and details how the CRISPR-Cas9 gene editing technique could efficiently revert mutations in the gene CYBB within CD34+HSC to clinically meaningful levels. Researchers conducted the study with genes from individuals with CGD, an x-linked trait marked by impaired phagocyte oxidase activity, recurrent infections, and auto-inflammation. The work will be presented as an oral presentation during the session entitled ‘Therapeutic Applications of Genome Engineering,’ which occurs January 12 from 5:00 to 6:45 p.m. MDT; and as a poster presentation during Poster Session 2 (Poster # 2019) on January 10 from 7:30 to 10:00 pm MDT. More information can be found at the Keystone Symposia website.
MaxCyte’s Proprietary GT® Flow Electroporation™ System enabled a robust, scalable, manufacturing process for this study. MaxCyte’s high-performance delivery platform is rapid, automated, cGMP-compliant and supported by a U.S. Food & Drug Administration master file. The platform is also a single-use, disposable, closed system process for cell-engineering and delivery.
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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™ 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.
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