MIT team improving gene therapies wins Sloan health care prize | MIT News

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The MIT workforce Kano Therapeutics gained the 2021 Sloan Healthcare Improvements Prize Thursday with its novel method for producing single-stranded DNA (ssDNA). The know-how might make gene therapies safer, extra customized, and cheaper.

Most gene therapies make the most of double-stranded DNA, which may miss its goal within the physique and produce undesirable uncomfortable side effects. Single-stranded DNA has the potential to extra exactly ship genetic materials to cells, however manufacturing ssDNA is at the moment costly and rigid.

Kano makes use of a novel method to make ssDNA manufacturing extra sensible for purposes like engineering immune cells to struggle illness and altering genomes with CRISPR.

“Sustainable biomanufacturing of lengthy single-stranded DNA can open doorways to gene and cell therapies we at the moment solely dream of,” says Kano workforce member Floris Engelhardt, who’s at the moment a postdoc at MIT. “Not solely do we expect lengthy ssDNA can enhance the protection and efficacy of cell and gene therapies — whereas unlocking new indications — it might additionally have the ability to improve the effectivity and pace of producing, shortening the time it takes to get therapies to sufferers.”

The workforce’s know-how is predicated on the analysis of Engelhardt, whose workforce additionally contains John Vroom, a first-year MBA candidate at MIT’s Sloan Faculty of Administration.

Kano’s manufacturing course of makes use of two variations of a kind of DNA molecule referred to as a plasmid to program a replicating virus referred to as a bacteriophage. One plasmid holds the code to get into the bacteriophage, and the opposite carries the specified genetic code to be harvested.

“Cell and gene remedy at the moment is just a little like assembling furnishings earlier than Ikea got here alongside,” Vroom says. “The items don’t at all times match collectively, meeting isn’t profitable, and it may be extremely irritating for the builders. Kano goals to be just a little just like the Ikea of cell and gene remedy.”

Kano, which earned $20,000 as a part of the prize, was one in all eight finalists to pitch on the competitors. Every workforce had 5 minutes to present their displays, which had been judged based mostly on classes together with the answer’s impression, novelty, market alternative, feasibility, and traction. The annual pitch competitors is a student-led occasion at MIT Sloan and is a part of the Sloan Healthcare and BioInnovations Convention that was held Feb. 25 and 26.

The $4,000 second-place prize went to Blue Ocean Therapeutics, an organization working to establish biomarkers that would assist deal with a kind of epilepsy often known as Focal Cortical Dysplasia sort II (FCD II). Utilizing advances in gene sequencing and machine studying to establish diseased cells, the corporate is hoping to enhance the precision of present remedies in addition to develop remedies of its personal.

In third place was Hive Well being, a startup utilizing knowledge science to offer a simpler, reasonably priced medical insurance choice to Filipino staff. Via a partnership with the Filipino authorities, the corporate is utilizing well being care knowledge to broaden entry to protection within the nation whereas sustaining profitability.

Past the prize cash, every of the eight finalists acquired suggestions from each the judges and different trade specialists who had been made out there to the groups by way of “workplace hours” within the weeks main as much as the ultimate pitch.

“The groups already gained whatever the final result,” says Zen Chu, a professor within the MIT Sloan Faculty of Administration who helped evaluate purposes. “This means of pitching and honing their concepts and getting suggestions from a broad array of traders and patrons, whether or not they’re physicians or insurance coverage firms or others, actually informs and improves their outcomes.”

Irena King, the founding father of Surgicure Applied sciences, which is creating a tool to safe medical tubes in affected person’s mouths once they’re intubated, says the competitors helped her workforce to assume by means of enterprise processes and to community, including that she’s been keen to attach with extra feminine founders and advisors.

“It’s at all times nice to get extra suggestions on how we are able to strengthen our enterprise mannequin,” King says. “Any connections we are able to make with the trade and traders are tremendous useful. Additionally, the extra credibility we get from going by means of a screening and competitors helps rather a lot.”

Different finalists included Cupid Well being, which helps heart-failure sufferers handle medicines whereas offering medical insights to suppliers; International New child Options, which is working to enhance neonatal well being in low-resource settings; Demos, which has developed a brand new drug-injection method to broaden entry to organic medicine; and Glyphic Biotechnologies, which is decoding the proteins within the human physique at single-molecule decision to enhance diagnostics and therapeutics.

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