Benjamin Oakes’ Post

At ESGCT last week, our team presented a story that lifts the curtain just a bit on how we are building leading CRISPR-based medicines that aim to improve the health of millions at Scribe Therapeutics: To design a potentially best-in-class therapeutic for our epigenetic silencing program for preventing ASCVD, and offer patients with cardiovascular disease a potentially highly durable and safe solution for LDL-C lowering, we first start with the holistic creation of better CRISPR molecules.   In this work, our molecular engineering team created tens of thousands of potential transcriptional effectors and performed a comprehensive high-throughput screen to identify better solutions than currently available.   What we found: More than one thousand novel repressors that appear to silence transcription more strongly than standard issue molecules found in today's literature. When we tested one of our leading, newly identified repressor domains in vivo, we found that it can significantly outperform conventional repressor domains, yielding twofold improvements in potency. This matters because it will allow Scribe to deliver more effective epigenetic silencers at significantly lower doses -- a critical lever for safety.  This is the standard we hold ourselves to at Scribe: rigorous design and engineering that translate into safer, more effective medicines for patients with cardiovascular disease. We are not comfortable perpetuating the status quo. We want create a better future. This is just a sneak peak at how we are working to make CRISPR-based genetic medicines safe enough to transform how millions of us improve our health and lower our disease risk. European Society of Gene and Cell Therapy #CRISPR #EpigeneticEditing