Scribe Therapeutics CEO and co-founder Benjamin Oakes said: “Scribe has designed, engineered and tested thousands of evolved CRISPR enzymes to build an advanced platform for creating a breakthrough in vivo treatments. In addition, Scribe will secure tiered, high single-digit to sub-teen royalties from Biogen. The deal will also enable the companies to study an additional neurological disease target.Īs per terms of the agreement, Scribe will secure $15m upfront payment and is eligible to receive over $400m in potential development and commercial milestone payments between on the two targets of interest. X-Editing (XE), which is Scribe’s first technology, is said to offer enhanced editing activity and deliverability compared to other currently CRISPR genome editing tools.īoth companies will work together to develop therapeutics for genetically-driven AL. I’m the founder of SynBioBeta, and some of the companies that I write about are sponsors of the SynBioBeta 2020 Global Synthetic Biology Summit.Under the deal, Scribe Therapeutics and Biogen will develop and commercialise CRISPR-based therapies to treat amyotrophic lateral sclerosis (ALS). Thank you to Karl Schmieder for additional research and reporting in this article. Subscribe to my weekly synthetic biology newsletter. "We're proud to collaborate with Biogen and apply our uniquely customized approaches to develop new, safe, and effective medicines for neurodegenerative diseases." In others, we'd be interested in down-regulating or disrupting the toxic copy," continued Oakes. In some cases, we'd be trying to silence the broken function. We are focused on removing that toxic gene. "In one of the subsets of ALS, there is a mutated gene that is toxic. Scribe will receive a $15 million upfront payment and will be eligible for more than $400 million in potential development milestone payments between the two targets of interest. The two companies may choose to pursue an additional neurological disease target with high, unmet need. Scribe will work with Biogen to create therapeutics for genetically driven ALS. Although the cause of ALS is not understood, the biological underpinnings in certain subsets of the disease have been identified. In ALS, those motor neurons die off causing muscle tissues to waste away. Motor neurons run from the brain through the spinal cord to the muscles that control movement in the arms, legs, chest, throat, and mouth. ALS, known as Lou Gehrig's disease, was in the news in 2014, when the social media campaign the Ice Bucket Challenge, went viral, promoting awareness of the motor neuron disease and raising more than $200 million worldwide.ĪLS destroys the nerve cells or motor neurons that control voluntary muscle movement. The Biogen collaboration seeks to develop and commercialize CRISPR-based therapies to address an underlying genetic cause of Amyotrophic Lateral Sclerosis (ALS), a progressive, degenerative disease. Ice Bucket Challenge Meet Next-Gen Gene Editing The company is using its funding to build a continuously evolving, expanding genetic modification platform designed for therapeutic use. Scribe received an initial $20 million in funding from a number of individual and institutional investors, including Silicon Valley investors Andressen Horowitz. © 2015 Mark Joseph Hanson and the Regents of the University of California Berkeley, is one the co-founders of Scribe Therapeutics. Their expertise and track record in CRISPR gene editing and protein engineering is unmatched.ĭavid Savage, Associate Professor of Biochemistry, Biophysics, and Structural Biology at UC. In addition to Doudna's foundational work and leadership in CRISPR gene editing, the team has developed tens of thousands of tools to study and manipulate genomes. Scribe Therapeutics was co-founded in 2018 by Oakes, molecular engineers David Savage and Brett Stahl, and CRISPR co-inventor Jennifer Doudna. As Scribe’s engineering core continues to evolve and expand, the company will utilize XE to address diseases of significant unmet need, starting with a pipeline of neurodegenerative disorders. Scribe is taking the same engineering and design approach when it comes to building genetic medicines. X-Editing molecules, the company’s first technology, are highly engineered CRISPR enzymes that allow researchers to edit living cells with greater efficacy, specificity and deliverability than currently available CRISPR genome editing technology. According to Oakes, Scribe has applied the engineering principles of design, test, build to create a sophisticated gene editing platform.
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