Scribe Therapeutics

When I was a kid, my dad had his first heart attack. That moment defined how I see cardiovascular disease, not as a statistic but as a preview into my own future. It taught me that framing matters. Because the story we tell about ASCVD shapes how we fight it. At last year’s American Heart Association meeting, a study across 16 major U.S. centers found that fewer than half of ASCVD patients were adherent to their prescribed meds. Clearly, there’s a gap in desirable treatments, but, the problem runs deeper if half of those in need are not taking their readily available medication. As this year’s AHA nears, I’ve been thinking about how to explain the underlying cause of ASCVD more effectively. Here goes: Think of ASCVD like a car on a track. 𝗗𝗿𝗶𝘃𝗲 𝗹𝗼𝗻𝗴 𝗲𝗻𝗼𝘂𝗴𝗵, 𝗮𝗻𝗱 𝘆𝗼𝘂 𝗿𝗲𝗮𝗰𝗵 𝗮 𝗵𝗲𝗮𝗿𝘁 𝗮𝘁𝘁𝗮𝗰𝗸. Cholesterol in ApoB lipoproteins is the gas that powers the car. Other risk factors—stress, blood pressure, inflammation, diabetes, smoking, aging—are your foot pressing hard on the pedal. Here’s the key: without gas in the tank, the car can’t go far. Many of today’s strategies focus on the pedal: lifestyle modification, controlling blood pressure, reducing inflammation. These matter a ton, but we all still age, still experience stress, still live with imperfect control. You can’t avoid pressing the pedal entirely. 𝗪𝗵𝗮𝘁 𝗶𝗳 𝗶𝗻𝘀𝘁𝗲𝗮𝗱 𝘄𝗲 𝗱𝗿𝗮𝗶𝗻𝗲𝗱 𝘁𝗵𝗲 𝘁𝗮𝗻𝗸? What if we did so early enough to stop the car from ever finishing the race? With lifelong lowering of the ApoB-containing lipoproteins that fuel atherosclerosis, cardiovascular disease could become a destination that’s difficult to reach — not delayed, but potentially prevented altogether. Large scale genetics data supports this! This is the shift we’re working toward Scribe Therapeutics. Using engineered CRISPR systems built for safety, potency, and precision, we aim to durably silence the genes that drive ASCVD and eliminate the need for chronic therapy. If we can change our own biology early and durably, we don’t just slow the car. We change the road ahead. #𝗔𝗛𝗔 #𝗔𝗦𝗖𝗩𝗗 #𝗗𝗶𝘀𝗲𝗮𝘀𝗲𝗣𝗿𝗼𝗴𝗿𝗲𝘀𝘀𝗶𝗼𝗻 Special thanks to Scribe's ASCVD advisors Kausik Ray MD FMedSci and Stephen Nicholls for helping to drive this vision!

How can we transform the standard of care for heart disease? ❤️🩹 This weekend, our CEO Benjamin Oakes will discuss promising #CRISPR gene editing and epigenetic silencing strategies at the Cardiovascular Research Foundation’s TCT MedTech Innovation Forum. Learn more about our work engineering safe and potent genetic medicines for cardiometabolic disease at #TCT2025. We’re excited to share the promise of CRISPR at the world’s premier meeting in interventional cardiovascular medicine! 📅 Saturday, October 25, 2025 at 11am PT 💡 MedTech Main Arena Session 3: Keeping an Eye on the Rearview Mirror: What Is the Next Big Disruption in Healthcare? → Genomics and Molecular Therapeutics: Engineering the Next Therapeutic Frontier 📍 Moscone Center, SF

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

Missed our presentation on Scribe’s ELXR (epigenetic editor) technology at #ESGCT2025? Catch up with this article from BioWorld News highlighting how we engineered our ELXR platform for enhanced potency and its therapeutic application in durably targeting PCSK9. The ELXR platform is the technology that underpins STX-1150, our #CRISPR-based therapy designed to lower LDL-C and address cardiovascular disease effectively. Thanks to Mar de Miguel Bonet for interviewing our CEO Benjamin Oakes and showcasing our innovative ELXR engineering work! Read more here 👉 https://lnkd.in/d8TeCba6 Clarivate European Society of Gene and Cell Therapy #CRISPR #EpigeneticSilencing #EpigeneticEditing

Listen to Scribe’s co-founder, Jennifer Doudna, speak to the vision that Scribe was founded on: “I really hope that gene editing technology, like CRISPR, becomes a preventative [therapy]…CRISPR is capable of changing those genes in the right target tissues to have a preventative effect. I think that would be extraordinary. Amazingly, there already are companies, not only academics, but companies that already have that vision and are working on that for cardiovascular disease.” Jennifer Doudna, Scribe co-founder, Innovative Genomics Institute founder, and Nobel laureate, spoke yesterday on stage at the Masters of Scale Summit with Reid Hoffman and Siddhartha Mukherjee, highlighting the promise of #CRISPR to revolutionize the standard of care for cardiometabolic disease. At Scribe, we are pioneering custom-engineered CRISPR-based medicines to unlock this potential for cardiometabolic disease. Our current focus is on targeting the three core lipid drivers of ASCVD: LDL-C (“bad” cholesterol), Lp(a), and triglycerides. Our progress to date: → Our lead candidate, STX-1150, a novel CRISPR-CasX-based epigenome editor that silences PCSK9 without cutting DNA, substantially lowers LDL-C by >50% in non-human primates for at least 9 months → STX-1200, our second asset, is a CRISPR-CasX-based genome editor targeting LPA that has dramatically achieved >90% knockdown of Lp(a) in in vivo models of disease → Our third program, STX-1400, a CRISPR-CasX-based genome editor targeting APOC3, has effectively reduced triglyceride levels by >90% in vivo. We are proud to be advancing this next generation of CRISPR-based genetic medicines towards preventing disease. #MoSSummit

Congratulations to our CEO, Benjamin Oakes, on being named to BioSpace’s inaugural 40 Under 40 list of exemplary biotech leaders. His vision, innovation, and commitment to advancing next-generation #CRISPR-based therapeutics that improve patient outcomes are evident in Scribe’s validated gene and epigenetic editing technologies, strong pharma collaborations, and pipeline of genetic medicines aimed at solving leading killers like heart disease. Article below 🔽

Congratulations to our CEO, Benjamin Oakes, on being named to BioSpace’s inaugural 40 Under 40 list of exemplary biotech leaders. His vision, innovation, and commitment to advancing next-generation #CRISPR-based therapeutics that improve patient outcomes are evident in Scribe’s validated gene and epigenetic editing technologies, strong pharma collaborations, and pipeline of genetic medicines aimed at solving leading killers like heart disease. Article below 🔽

We’re headed to #ESGCT2025 to share our latest epigenome editing data. If you’ll be in Seville for the conference, check out our Senior Scientist Emeric Charles’ presentation on Thursday, October 9. 🧬 Engineering enhanced epigenetic editors: novel repressor domains drive potent and durable therapeutic gene silencing in non-human primates European Society of Gene and Cell Therapy #CRISPR #EpigeneticSilencing #EpigeneticEditing

Meet Scribe at #CGMesa25 in Phoenix 🌵 Our CFO David Parrot will be onsite at the Alliance for Regenerative Medicine Cell & Gene Meeting on the Mesa to present updates on our work engineering #CRISPR-based medicines for cardiometabolic disease. Swing by his presentation on Monday, October 6 at 1:45pm MST.

You might think that philosophy and biology are contradictory disciplines, humanities grating against a more traditional science. Not the case! In college, I discovered they could, and perhaps should, be essential complements. At Colby College, I pursued this somewhat unusual double major. And in the end, it was classic philosophy that instilled one of the most fundamental lessons of molecular biology: that we are at our best when we assume that we know much less than we expect. This Socratic approach is a bit distinct to how biology is often taught, as a fact-based, "to be memorized" discipline. Instead, I have found it's best to question every “known” assumption and realize that just because we've generally accepted one solution doesn't mean it's the only way, or even the right way. Ultimately this combination was supposed to lead to an M.D. I even took the MCATs. But from there, I spent time at a rural hospital in Maine and watched physicians focused on managing disease and patient decline, with few real treatments, even fewer preventive options and no cures. Unexpected. These were brilliant doctors doing necessary work, but they were stuck addressing downstream problems and not the source of disease. In this way, disease could only grow. Most often, they didn't even have the time to question what that source could be. Frustrating. This realization led me away from the practice of medicine and to genetic engineering. My goal became to create genetic medicines that could address problems at their molecular source. I started my career engineering the first generation of gene editing technologies known as Zinc Finger Nucleases at Princeton University, then moved to the labs of David Savage and Jennifer Doudna at University of California, Berkeley, where I sharpened my engineering skillsets even as I honed unique CRISPR technologies. Ultimately I was granted an opportunity to launch my own lab at the Innovative Genomics Institute to create CRISPR systems, potent and safe enough to use as preventative medicines. Philosophy taught me to always question the status quo. Now at Scribe Therapeutics, we're doing the same: 𝗪𝗲 𝗱𝗼𝗻'𝘁 𝗮𝗰𝗰𝗲𝗽𝘁 𝘁𝗵𝗮𝘁 𝗱𝗶𝘀𝗲𝗮𝘀𝗲 𝗶𝘀 𝗮𝗻 𝗶𝗻𝗲𝘃𝗶𝘁𝗮𝗯𝗶𝗹𝗶𝘁𝘆, 𝗮𝗻𝗱 𝘄𝗲 𝗱𝗼𝗻'𝘁 𝗮𝗰𝗰𝗲𝗽𝘁 𝘁𝗵𝗮𝘁 𝗻𝗮𝘁𝘂𝗿𝗲'𝘀 𝗳𝗶𝗿𝘀𝘁 𝗱𝗿𝗮𝗳𝘁 𝗼𝗳 𝗴𝗲𝗻𝗼𝗺𝗲-𝗲𝗱𝗶𝘁𝗶𝗻𝗴 𝘁𝗼𝗼𝗹𝘀 𝗮𝗿𝗲 𝗶𝗱𝗲𝗮𝗹 𝗳𝗼𝗿 𝗵𝘂𝗺𝗮𝗻 𝘁𝗵𝗲𝗿𝗮𝗽𝗲𝘂𝘁𝗶𝗰𝘀. We build, engineering our CRISPR systems with the goal of being safe enough to be preventative for all, first by determining the therapeutic needs and then creating new CRISPR technologies to address them. This is what drives me and our "CRISPR by Design" approach. (Photo: Fellows lab at the IGI)