was the co-inventor of a breakthrough in gene editing: the ability to target a single misspelling in DNA code. This invention, known as, is a further advancement of the Nobel Prize-winning Crispr technology and has the potential to cure thousands of rare diseases caused by point mutations. She then joinedto, in her words, “make the dream of having that technology become a reality for patients really materialize.
With clinical trials now underway at Beam for patients with sickle cell disease and a rare liver disease known as alpha-1 antitrypsin deficiency, Gaudelli hopes to embark on a new journey from the bench to the bedside. TodayVerve Therapeutics , which is targeting cardiovascular disease. “It's been a great way for us to bet on really talented people that we believe can start very interesting companies,” Rozen told. “We can take longer time horizons and take on more risky projects. Because it's kind of like an incubation with a supportive family around you that helps you through the different steps, different decisions.”, predicting “a new wave of applications of this powerful tool.
Last Thursday, surgeons transplanted a pig kidney into a living patient for the first time. The kidney was provided by eGenesis, a startup which has developed gene-edited pig organs to solve the problem of the severe shortage of organs available for donation. “We’ve shown that we actually have something that can help patients,” the company's CEO Mike Curtis told.
In addition to kidneys, the company also developed gene-edited hearts and livers for transplant. Curtis said he expects clinical trials for liver to start this year and kidneys next. He also hopes that, with more human data, gene-editing on organs can be refined to the point where patients don't need to be on a regimen of medication to prevent organ rejection.
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Source: ForbesTech - 🏆 318. / 59 Read more »
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