Pipeline
Portfolio by Design
Genetic medicines designed to be safe and specific forcardiometabolic disease (CMD)
We’re building a portfolio of genetic medicines for cardiometabolic disease (CMD) using our gene editing and epigenetic silencing platforms. We prioritize genetic targets with clinically-validated biology and demonstrated patient benefit from on-target modification, starting with targeting PCSK9 to address cardiovascular disease associated with LDL-C.
Why treat cardiometabolic disease (CMD)?
Despite availability of existing medications, CVD remains the leading cause of death globally.
It doesn’t have to be this way. For common conditions like CMD, genetic medicines have the potential to be once-in-a-lifetime therapies that fundamentally lower people’s major risks.
We’re addressing the greatest unmet need in healthcare by deliberately engineering therapeutics to become the standard of care for CMD.
93%
People in the U.S. living with suboptimal cardiometabolic health, leading to an increased risk for CVD, diabetes, liver failure, neurodegeneration, and cancer
500M
Projected new CMD cases over the next decade, costing the US healthcare system greater than $27B annually
Why target LDL-C?
Low-density lipoprotein cholesterol (LDL-C) is a key driver of dyslipidemia and cardiovascular risk. Elevated LDL-C levels in the bloodstream can promote the formation of plaques in arterial walls, restricting blood flow and increasing the risk of heart attacks and strokes. Lowering LDL-C effectively reduces the risk of major cardiovascular events.
Cardiovascular risk is cumulative, thus the unmet need for effective LDL-C-lowering treatments that start earlier in disease progression is even greater. Today’s standard of care is insufficient - the chronic care model is plagued with high treatment burden, safety concerns, and lack of early intervention, leading to suboptimal outcomes.
Our LDL-C lowering programs are starting with targeting PCSK9 to address cardiovascular disease associated with LDL-C. Our candidates are designed to be used earlier, with persistent durability, replicating the low lifetime risk profile of individuals with favorable PCSK9 genetics.
18M
Lives lost to CVD annually, close to 1 in 5 deaths overall
40s
A heart attack occurs every 40 seconds in America
What could the future look like for CMD patients?
Today
Typical patient journey over time
Tomorrow
With genetic medicine over time
Today
Typical patient journey over time
Tomorrow
With genetic medicine over time
Partnerships
Our CRISPR-based platforms have the potential to create life-changing genetic medicines across a broad spectrum of disease. We’re leveraging collaborations with world-leading pharmaceutical companies to expand our patient impact.
In preclinical studies, we’ve observed gene editing at or near saturating levels across several major organ systems and tissue types, including the central nervous system, muscle, liver, and eye. We’ve similarly demonstrated high levels of editing in ex vivo applications.
Given these promising results, we are committed to broadening and accelerating the potential impact of our platforms into other therapeutic areas via strategic collaborations with leading partners.
Partnered Programs
We are pleased to expand our work with Scribe, an illustration of our shared commitment to advance best-in-class genome editing therapies for patients in need.
Christian Mueller
Sanofi, Global Head of Genomic Medicine Unit