Engineering the future of genetic medicine
Preclinical
We continue to custom engineer molecules that are designed for therapeutic use within the human body to enable highly effective and specific in vivo applications across various areas of unmet need. Our uniquely flexible “CRISPR by design” platform enables a new generation of purpose-built, highly functional CRISPR tools and medicines for addressing the fundamental root causes of genetic disease, starting with neurodegenerative disorders including Amyotrophic Lateral Sclerosis (ALS). Our engineering-first technology overcomes long-standing industry bottlenecks and the present challenges of current CRISPR technologies to open up new target disease areas that were previously challenging to target and deliver critical CRISPR-based therapies to patients who need it most.
Scribe is the only company engineering “CRISPR by design.” Our holistic approach to design and engineering, rather than “finding” makes it possible for us to continually expand our pipeline of genetic medicines with additional CRISPR molecules and technologies, and in fact we do not use any molecules based on the first generation CRISPR-Cas9 systems. Rather, we have built our first suite of technologies on CasX to enable greater editing activity, specificity, and deliverability. We are engineering custom gene editing and delivery technologies to deliver potentially curative therapeutics for diseases of greatest unmet need and establish a new standard of clinical care for millions of people with genetic disease. By creating custom molecules specifically designed for therapeutic use within the human body, Scribe is driving the shift in therapeutic development from slow drug discovery to scalable biological design to develop safe and effective genetic medicines for use in the human body.
In October 2020, we emerged from stealth to unveil a $400M+ collaboration with Biogen to develop and commercialize CRISPR-based therapies that address the underlying genetic causes of ALS. The unveiling of the first application of our technologies was accompanied by the announcements of our evergreen, proprietary “CRISPR by design” platform and initial $20M Series A financing led by Andreessen Horowitz. Our two teams are committed to expanding treatment for underserved genetic conditions. The terms of the collaboration include the additional option to pursue an additional neurological disease target of high unmet need through Scribe’s custom gene editing and delivery technologies. Scribe received $15 million upfront and is eligible for more than $400 million in potential development and commercial milestone payments between the two targets of interest, as well as tiered, high single digit to sub-teen royalties. In creating custom molecules specifically designed for therapeutic use within the human body, it is our first program driving the shift in therapeutic development from slow drug discovery to scalable biological design to develop safe and effective genetic medicines for use in the human body.
