Scribe Therapeutics Unveils CRISPR Platform and Collaboration with Biogen

Rare Daily Staff

Scribe Therapeutics unveiled its platform for highly efficient and specific in vivo genome modification and said it will collaborate with Biogen to develop genetic medicines for neurodegeneration.

Since launching out of UC Berkeley’s Doudna Lab, Oakes Lab, and the Innovative Genomics Institute in late 2018 with $20 million in venture funding, it has developed a custom platform of gene editing and delivery technologies that it says will revolutionize genetic medicine to permanently treat the underlying cause of disease.

“Scribe’s technology and long-term, engineering approach can help establish CRISPR-based therapies as a new standard of clinical care and expand access to groundbreaking treatments that will impact millions of lives,” said Jennifer Doudna, co-inventor of CRISPR technology and co-founder of Scribe Therapeutics.

Scribe aims to move beyond the issues of safety, delivery, poor editing outcomes, and an uncertain intellectual property landscape that limit current CRISPR technology. With strong expertise and track record in CRISPR and protein engineering, the Scribe team is building and applying a suite of CRISPR technologies designed for therapeutic use into a single continually evolving and expanding genetic modification platform.

“Scribe’s approach is a philosophical and technical leap to address the fact that no existing approach can fully deliver the types of CRISPR-based therapies into the clinic that we believe are possible,” said Benjamin Oakes, CEO and co-founder of Scribe Therapeutics. “We designed Scribe to specifically deliver these therapeutic molecules and blueprinted out the ideal approach – the technology, the engineering philosophy, the team. We then built from scratch an entire system of novel delivery technologies and CRISPR enzymes specifically designed for therapeutic purposes. Ultimately, we are holistically redesigning what nature provides to bring new, best-in-class genetic medicines to people in need, as quickly as possible.”

Scribe engineers novel CRISPR molecules specifically for therapeutic attributes, allowing the company to control the exclusive rights to its custom platforms. The company’s first technology, called X-Editing (XE) molecules, are highly engineered CRISPR enzymes that provide combined aspects of greater efficacy, specificity and deliverability than currently available CRISPR genome editing technology. 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 therapeutics for neurodegenerative disorders.

The technology will be tested in a just announced research collaboration with Biogen to develop and commercialize CRISPR-based therapies that address the underlying genetic causes of Amyotrophic Lateral Sclerosis (ALS), a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord and eventually leads to the death of motor neurons and loss of the ability of the brain to initiate and control muscle movement. With muscle action progressively affected, patients in the later stages of the disease may become paralyzed and unable to breathe normally.

Under the terms of the deal, Scribe will work with Biogen to create therapeutics for genetically-driven ALS, with an option to pursue an additional neurological disease target with high, unmet need. Scribe will receive $15 million upfront and is eligible for more than $400 million in potential development and commercial milestone payments between the two targets of interest. Scribe is also eligible to receive tiered, high single digit to sub-teen royalties.

“The biopharma industry is looking for a new generation of purpose-built, highly functional CRISPR tools to enable their ability to develop future genetic medicines,” said Svetlana Lucas, chief business officer of Scribe. “We are building Scribe to be the partner of choice for the industry by creating a versatile set of technologies that allow us to address the fundamental root causes of disease, eliminating significant biology risk from the equation with a goal to dramatically improve the economics of drug development and affordability of these potentially curative therapeutics.”

Photo: Benjamin Oakes, CEO and co-founder of Scribe Therapeutics