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Expanding upon the CRISPR-Cas9 geneediting system, researchers at MIT have designed a new technique called PASTE geneediting that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE geneediting technique was recently published in Nature Biotechnology.
Novartis has shouldered its way into the in vivo geneediting category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4
Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt.
The French biotech said the cash injection – which follows a €45 million Series A in 2020 – will also help it bring forward the first development candidate in a collaboration signed with gene-editing specialist Intellia Therapeutics last year, currently in the early-discovery stage.
As per the company, dbDNA is suitable for rapid, scalable manufacture of GMP DNA and can incorporate genesequences of sizes ranging from 500bp to 20kb, which are typically unstable as pDNA. Rapid production will potentially enable researchers to overcome an industry bottleneck.
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