Platform for engineering CRISPR-based medicines
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A platform for engineering CRISPR-based medicines by Scribe Therapeutics

Scribe Therapeutics established by 2 ex-members of CRISPR pioneer Jennifer Doudna’s UC Berkeley genetics lab (with Doudna herself) unveiled a platform made especially to assist develop and engineer new therapeutics based on CRISPR for resolving particular diseases, with permanent treatments in people.

Jennifer Doudna ( The Nobel Prize Winner in Chemistry 2020 ) is part of the leadership team behind Scribe Therapeutics. However, it is primarily led by Benjamin Oakes, CEO, and founder, and Brett T. Staahl, VP of the platform. Benjamin Oakes and Staahl worked together at Doudna’s laboratory, where Oakes was a student and Staahl was a Postdoctoral researcher. How gene editing, and CRISPR specifically, could be used to assist in the treatment of Huntington’s disease was Staahl’s interest in research. Oakes was set out to be a practicing medical doctor, but he turned back to his research to study the underlying reasons for disease.

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Oakes described that he set out on this trip to comprehend how they could, as well as how they can best solve those underlying issues of Huntington’s disease. This drove him to start a study in ZFN (Zinc-Finger Nuclease)- based genome editing – a precursor method to CRISPR that was time-consuming, far less particular, and far more work-intensive. The groundbreaking research article by Doudna on CRISPR was published in 2012, and Oakes instantly saw the potential, so he joined her laboratory.

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Meantime, Staahl was studying the treatments for disorders that particularly bring about neural degeneration – something that had not earlier been part of Doudna’s laboratory’s study before.

Doudna said that he had a significant period in the laboratory, establishing methods for neurons, and attempting to bring that technology to a point where it could be expanded as a genuine therapy for the neurodegenerative disease, with Huntington’s as a model. Both of them saw a very interesting opportunity to utilize the sort of technology development that Ben had been doing, which he was extremely crazy about proceeding, as well as to concentrate it on this challenge of neurodegenerative disease.

The outcomes are that Scribe has already raised 20 million dollars in a Series A funding round (with some amount of prior seed funding granted) led by Andreessen Horowitz. Since 2018, Scribe therapeutics has been at work on their solution. However, he continued to be quiet regarding their development until Oakes felt confident that what they’re presenting is an actual, feasible technology that can be made use of to generate therapies.

The firm is likewise revealing a current partnership with the drug manufacturer Biogen, Inc. to work together on CRISPR-based medications against neurological diseases, and especially Amyotrophic Lateral Sclerosis.

The deal is valued 15 million dollars in upfront commitments, with as high as 400 million dollars or more in milestone payouts to follow, and nobilities affixed to any shipping therapeutics that results.

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Oakes said it is a testimony to the maturity of the platform that they were capable to achieve this collaboration. However, Scribe therapeutics will likewise be continuing the expansion of its therapeutics internally. According to Oakes, it is a method in the service of dealing with the best number of possible disease treatments the startup can handle.

Oakes said he is in no hurry to get additional financing, while it’s already producing earnings, and he does believe that inevitably they will certainly look for extra investment in order to help make sure they can treat as many prospective conditions as feasible, as promptly and safely as feasible.

When it comes to the basic science behind Scribe, their benefit hinges on the job they have made to adapt a molecule called CRISPR-CasX, which is a little smaller sized than Cas9 as well as not originated from pathogen molecules. Both of these make it better-suitable to medicines. Scribe Therapeutics has spent the last year-and-a-half converting CasX into the foundation of a platform that operates better than any CRISPR protein that is present for delivery through via adeno-associated virus and engineering it for higher uniqueness.

Oakes added that they built Scribe particularly to do that, to construct a design core-focused specifically on making one of the most sophisticated the best restorative genome editing molecules.

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A platform for engineering CRISPR-based medicines by Scribe Therapeutics

Author: Sruthi S

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