World News – UA – Scribe Therapeutics emerges with $ 20 million Biogen pact to overcome CRISPR obstacles

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CRISPR evolved in bacteria as a form of ‘genetic vandalism’, as George Church puts it, to repel pathogens This means it works great in bacteria but runs into various obstacles when researchers try to implement it in humans Jennifer Doudna’s latest CRISPR company plans to overcome these obstacles with a brand new CRISPR platform that does not rely on molecules found in nature

Scribe Therapeutics Launches $ 20 Million Series A Funding to Develop Platform, Along with Partnership with Biogen to Apply Its Technology Biogen Provides Over $ 15 Million Upfront to Work on Treatments gene editing for amyotrophic lateral sclerosis (ALS), with potential to tackle another neurological disease target The Big Biotech is on the hook for an additional $ 400 million in development and in commercial milestones between two ALS targets

« There are types of ALS the underlying genetics of which we fully understand. This makes it an ideal target for us with genome editing technology We don’t have to wonder if we are altering these sub genetics. underlying, will this change the trajectory of the disease? ”said Benjamin Oakes, CEO of Scribe, Phré, who co-founded the company alongside Jennifer Doudna, Phré, Brett Staahl, Phré, and David Savage Phré

CRISPR molecules include a guide RNA that recognizes the target DNA sequence and an enzyme, usually Cas9, which cuts it Scientists, including Doudna’s team at the University of California at Berkeley, have identified more enzymes over the years, but this approach of discovering them in nature – rather than designing new, specially designed enzymes – limits the reach of CRISPR

Creating CRISPR molecules from scratch allows Scribe to create treatments that can safely and efficiently modify the genome, and that are small enough to adapt to adeno-associated viruses, or AAVs, which are one of the best ways to deliver CRISPR treatments to the body

“We sat down and wondered what characteristics we wanted from a therapeutic molecule,” said Oakes “What became clear was a single molecule that could edit the genome well with the least amount of possible proteins and which was very specific « 

The founders of Scribe were concerned about off-target effects, when a CRISPR process alters untargeted parts of the genome and causes harmful side effects.But they’re hoping their technology can go one step further and target sequences with even more precision, so that it can focus on diseases caused by mutations where a single nucleotide base – or letter – in the genome is altered

Some CRISPR-based treatments, like the sickle cell program CRISPR Therapeutics and Vertex, alter the genome of cells ex vivo, i.e. outside the body Scribe’s focus is in vivo editing, which corrects pathogenic mutations inside the body

Its first technology, dubbed X-Editing, is based on CRISPR-CasX, which was discovered in Doudna’s lab.The team members designed a CRISPR molecule that had the desired properties, then tested what would happen when they changed different parts of it

« We were testing thousands upon thousands of different modifications at a time We can then take the best changes, start stacking them on top of each other and run that exact question again We can select a molecule that is at the bottom. both very active and very specific ”, said Oakes

He compares Scribe’s work to the domestication of teosinte to corn: « Each of these cycles is like planting in a field and picking corn that’s just a little bigger and a little bigger »

It’s not a quick or easy process, and it’s « a lot less exciting » than discovering a new enzyme, Oakes added. But all this engineering will be added to CRISPR processing in the future, where the gains could Being Huge X-Editing is the first product of Scribe’s work, but it won’t be the last – it’s one of many upcoming technologies that will fit into a single, ever-evolving and expanding platform. , says the company

In addition to working on ALS treatments with Biogen, Scribe wants to develop his own pipeline, as well as partnerships with other biopharmaceutical companies He has identified several areas where the technology could be useful, including neurological diseases. , genetic forms of blindness, neuromuscular diseases and blood disorders

Partnering doesn’t just mean licensing all of its work, said Svetlana Lucas, Phré, Commercial Director of Scribe

“We would be interested in partnering to develop our pipeline together with Big Pharma because we are small and humble about what we can and cannot do,” she added. “We are extraordinary in the ‘engineering and technology discovery, but the big pharmaceutical companies have immense expertise in terms of clinical development, manufacturing and knowledge of the diseases we pursue »

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CRISPR, Jennifer Doudna, Biotechnology, Genetics, Biogen, Medicine, Genome Editing, University of California, Berkeley