Cell preparation instrument is expected to subvert gene therapy laboratory

The field of genetic testing, which greatly benefited people's livelihood, is in full swing in the context of the continuous overweight of policy dividends, and the innovation and research and development of cell frontier technology is also in full swing. Recently, "MIT Technology Review" launched "Top Ten Breakthrough Technologies in 2017", "Gene Therapy 2.0" is among them, and it is expected to usher in a new breakthrough this year. As soon as this news came out, discussions about portable cell preparation instruments quickly caught the attention of the industry.

Gene therapy refers to the introduction of exogenous normal genes into target cells to correct or compensate for diseases caused by genetic defects or abnormalities, and ultimately achieve therapeutic purposes, with a history of more than 30 years. Although the process is quite rough, with the optimization of vector viruses and delivery mechanisms, gene therapy has entered the 2.0 era, showing a good prospect in the treatment of cancer and rare diseases.

It is reported that UniQure's Glybera (treatment of familial lipoprotein lipase deficiency) and GSK's Strimvelis (treatment of severe combined immunodeficiency disease) two gene therapy products have been approved in Europe. In 2017, Spark Therapeutics' gene therapy Voretigene Neparvovec (the treatment of hereditary retinal disease) is expected to be the first FDA-approved product.

基因疗法有望迎来新突破 细胞制备仪将颠覆实验室

Portable instrumentation "gene therapy in a box"

How to achieve automated preparation of cells? This is the key to the Adair team's attempt to “popularize” gene therapy.

They used a cell processing device, Prodigy, sold by German instrument manufacturer Miltenyi, which automates the key steps in gene therapy – cell preparation and amplification. The cells are input from one end of the instrument, and after 30 hours of operation, they are output from the other end, which can be used for treatment, which greatly shortens the cell processing time.

Adair hopes that this mobile instrument will extend gene therapy to more countries and the planet, including Africa, where the AIDS epidemic is most severe.

Prodigy: Small in size, weighs about 150 pounds, and is equipped with a bright glass cover, neat dashboard and curved infusion hose. The patient's cells are transported in a hose, contacted with various chemicals, and finally fused with a viral vector carrying the target DNA.

The Prodigy is priced at about $150,000, and the cost of a reagent for a patient's cell preparation is about $12,000. Katharina Winnem ller, Marketing Manager, Germany, said: "This mobile device greatly simplifies the cell treatment process. Although this does not mean that every rural hospital can carry out related treatments, it can significantly reduce the cost of treatment for patients."

In addition to Miltenyi, other research institutes and companies are actively developing similar cell processing equipment. For example, General Electric has acquired Biosafe, a company specializing in cell processing, and Draper Laboratories of MIT is developing a micro. Fluid equipment for pre-preparation of CAR-T therapy.

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