Jennifer Doudna’s New Gene Editing Company Launches With A $20 Million Round To Develop Genetic Medicines

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What if you had a tool to change the genetic instructions that cause disease?

That’s what San Francisco-based Scribe Therapeutics hopes to do with its next-generation platform for gene editing.

Today, the company announced a collaboration with Biogen to develop CRISPR-based genetic medicines for neurological diseases, including Amyotrophic Lateral Sclerosis (ALS).

CRISPR, you may remember, is a powerful tool used to control the genes (or genetic instructions) that are active in plants, animals, and even humans. With CRISPR gene editing, researchers can “silence” undesirable traits, and, potentially, add desirable traits. 

Over the past few years, CRISPR gene editing has been used to reduce the severity of genetic deafness and treat sickle-cell anemia in mice. Today, CRISPR is considered one of the most significant discoveries in the history of biology.

“To build a platform that would solve the challenges associated with in vivo genetic modification, we took an intentional design philosophy. We redesigned and engineered what nature provides us from scratch, testing thousands of evolved CRISPR enzymes to build the best platform and bring new, best-in-class genetic medicines to people in need,” said Benjamin Oakes, Chief Executive Officer, and co-founder of Scribe Therapeutics. “The resulting technology — X-Editing — is based on a philosophical and technical leap to provide greater editing activity, specificity, and deliverability compared to other currently available CRISPR genome editing tools and avoids issues associated with intellectual property uncertainty.”

According to Oakes, Scribe has applied the engineering principles of design, test, build to create a sophisticated gene editing platform. X-Editing molecules, the company’s first technology, are highly engineered CRISPR enzymes that allow researchers to edit living