CEO @scribetx | at the interface of molecular and genetic engineering | designing CRISPR to be safe enough for all

Joined April 2011
๐—ง๐—ผ๐—ฑ๐—ฎ๐˜†, ๐—ฆ๐—ฐ๐—ฟ๐—ถ๐—ฏ๐—ฒ ๐—ง๐—ต๐—ฒ๐—ฟ๐—ฎ๐—ฝ๐—ฒ๐˜‚๐˜๐—ถ๐—ฐ๐˜€ ๐—ผ๐—ณ๐—ณ๐—ถ๐—ฐ๐—ถ๐—ฎ๐—น๐—น๐˜† ๐—ฏ๐—ฒ๐—ฐ๐—ฎ๐—บ๐—ฒ ๐—ฎ ๐—ฐ๐—น๐—ถ๐—ป๐—ถ๐—ฐ๐—ฎ๐—น-๐˜€๐˜๐—ฎ๐—ด๐—ฒ ๐—ฏ๐—ถ๐—ผ๐˜๐—ฒ๐—ฐ๐—ต๐—ป๐—ผ๐—น๐—ผ๐—ด๐˜† ๐—ฐ๐—ผ๐—บ๐—ฝ๐—ฎ๐—ป๐˜† ๐˜„๐—ถ๐˜๐—ต ๐˜๐—ต๐—ฒ ๐—ฎ๐—ฑ๐˜ƒ๐—ฎ๐—ป๐—ฐ๐—ฒ๐—บ๐—ฒ๐—ป๐˜ ๐—ผ๐—ณ ๐—ฆ๐—ง๐—ซ-๐Ÿญ๐Ÿญ๐Ÿฑ๐Ÿฌ, a novel ๐˜ช๐˜ฏ ๐˜ท๐˜ช๐˜ท๐˜ฐ epigenetic CRISPR therapy designed to deliver ultra-durable lowering of โ€œbad cholesterol,โ€ from a single dose, all without permanently altering the genome. This program is built on years of intentional and iterative engineering focused on improving the safety, specificity, potency, and durability of CRISPR medicines. ๐—•๐˜‚๐˜ ๐˜„๐—ต๐—ฎ๐˜ ๐—ฒ๐˜…๐—ฐ๐—ถ๐˜๐—ฒ๐˜€ ๐—บ๐—ฒ ๐—บ๐—ผ๐˜€๐˜ ๐—ถ๐˜€ ๐˜„๐—ต๐—ฎ๐˜ ๐˜๐—ต๐—ถ๐˜€ ๐—ฐ๐—ผ๐˜‚๐—น๐—ฑ ๐—บ๐—ฒ๐—ฎ๐—ป ๐—ณ๐—ผ๐—ฟ ๐—ฝ๐—ฎ๐˜๐—ถ๐—ฒ๐—ป๐˜๐˜€ ๐—น๐—ถ๐—ธ๐—ฒ ๐—บ๐˜†๐˜€๐—ฒ๐—น๐—ณ. As someone at high risk of ASCVD, like roughly one-third of adults in the U.S., Iโ€™ve spent a lot of time thinking about the burden patients carry. For a chronic disease like ASCVD, prevention is far from easy. Success depends on maintaining near-perfect adherence to pills or injections for decades. In the real world, thatโ€™s incredibly difficult and simply not practical for most people. The fact that ASCVD remains the leading cause of death globally, despite plenty of therapeutic choices, makes that painfully clear. ๐—ง๐—ต๐—ฒ ๐—ณ๐˜‚๐˜๐˜‚๐—ฟ๐—ฒ ๐—ผ๐—ณ ๐—บ๐—ฒ๐—ฑ๐—ถ๐—ฐ๐—ถ๐—ป๐—ฒ ๐˜€๐—ต๐—ผ๐˜‚๐—น๐—ฑ ๐—ฎ๐˜€๐—ฝ๐—ถ๐—ฟ๐—ฒ ๐˜๐—ผ ๐—บ๐—ผ๐—ฟ๐—ฒ ๐˜๐—ต๐—ฎ๐—ป ๐—ฐ๐—ต๐—ฎ๐—ถ๐—ป๐—ถ๐—ป๐—ด ๐—ฝ๐—ฎ๐˜๐—ถ๐—ฒ๐—ป๐˜๐˜€ ๐˜๐—ผ ๐—น๐—ถ๐—ณ๐—ฒ๐—น๐—ผ๐—ป๐—ด ๐—บ๐—ฒ๐—ฑ๐—ถ๐—ฐ๐—ฎ๐˜๐—ถ๐—ผ๐—ป๐˜€. The vision behind STX-1150 is to provide year to decades of LDL-C lowering from a simple intervention, helping free patients from the constant burden of chronic treatment while more effectively reducing the risk of the worldโ€™s leading cause of death. The future is about empowering patients to take greater control of our own health destiny and preventing disease rather than waiting to treat it after catastrophe occurs. ๐—œ ๐—ฏ๐—ฒ๐—น๐—ถ๐—ฒ๐˜ƒ๐—ฒ ๐—ฎ ๐—ป๐—ฒ๐˜„ ๐—ฒ๐—ฟ๐—ฎ ๐—ผ๐—ณ ๐˜๐—ต๐—ฒ๐—ฟ๐—ฎ๐—ฝ๐—ฒ๐˜‚๐˜๐—ถ๐—ฐ๐˜€ ๐—ถ๐˜€ ๐—ผ๐—ป ๐˜๐—ต๐—ฒ ๐—ต๐—ผ๐—ฟ๐—ถ๐˜‡๐—ผ๐—ป, ๐—ผ๐—ป๐—ฒ ๐˜„๐—ต๐—ฒ๐—ฟ๐—ฒ ๐—บ๐—ฒ๐—ฑ๐—ถ๐—ฐ๐—ถ๐—ป๐—ฒ ๐—ฐ๐—ฎ๐—ป ๐—ฑ๐˜‚๐—ฟ๐—ฎ๐—ฏ๐—น๐˜† ๐—ฟ๐—ฒ๐˜€๐—ต๐—ฎ๐—ฝ๐—ฒ ๐—น๐—ผ๐—ป๐—ด-๐˜๐—ฒ๐—ฟ๐—บ ๐—ต๐—ฒ๐—ฎ๐—น๐˜๐—ต ๐—ฎ๐—ป๐—ฑ ๐—ฎ๐—น๐—น๐—ผ๐˜„ ๐˜‚๐˜€ ๐—ฎ๐—น๐—น ๐˜๐—ผ ๐—น๐—ถ๐˜ƒ๐—ฒ ๐—น๐—ผ๐—ป๐—ด๐—ฒ๐—ฟ, ๐—ต๐—ฒ๐—ฎ๐—น๐˜๐—ต๐—ถ๐—ฒ๐—ฟ ๐—น๐—ถ๐˜ƒ๐—ฒ๐˜€ ๐˜„๐—ถ๐˜๐—ต ๐—ด๐—ฟ๐—ฒ๐—ฎ๐˜๐—ฒ๐—ฟ ๐—ณ๐—ฟ๐—ฒ๐—ฒ๐—ฑ๐—ผ๐—บ. Extremely proud of the entire Scribe team for advancing this vision. Excited for what comes next.
Announcing our first clinical trial. Scribe has secured regulatory clearance from Australiaโ€™s @TGAgovau to initiate a first-in-human clinical study of STX-1150 for the treatment of hypercholesterolemia, a major driver of atherosclerotic cardiovascular disease (ASCVD). Details๐Ÿงต
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Largely agree with these thoughts, and have some perhaps discordant points to add. - Solving biological diseases in the next few years will benefit mostly from the usefulness of the language models, substantially more than molecular ML. - Proponents of the bitter lesson for biological models (not LLM) aren't wrong, just (possibly pathologically) too early. We don't have enough data, and the data we can collect quickly is probably not the right kind (we may get lucky with biology being easy, but it's usually not). - I think just scaling models and public (passively accumulating) data is a cost ineffective direction (it's like scaling dense NNs on the internet). Today's model outputs are as good as GPT2. A single bioinformatician with linear regression, sometimes even without any additional data, can beat these models in many applications (people rarely benchmark AI advances against a simple Potts model). The most promising approaches for molecular models are innovating heavily on architecture (e.g. Baker Lab, Alphafold, etc). This may automate/speed up with LLMs. - Few companies (hopefully Dyno included) are betting that we've triangulated enough pieces from multiple experimental channels to predict (with scale) how things translate in humans. Having worked on translating in vitro things to primates, I'm very cautious with the idea that current models, trained on current public data, can do anything more than manipulating already working solutions, either explicitly or through implicit remixing of what has worked. It may still work, but the underlying bets offer no specific reason why there is any epistemic risk advantage other than being "made with AI". - Short term, there is more promise in combining molecular models with LLMs rather than scaling them. The two sources of lift in AI discovery elsewhere has been (1) Breaking Silos: AI synthesizes a bunch of knowledge/expertise that is rarely present in the same room (2) Data mining : AI pushes through volumes of data, with an intelligent eye, that humans or simple algorithms simply can't scale or adapt to. I'm curious what will come out of multimodal training of LLMs and biological data, which seems to be something the big players are pursuing. I think latent reasoning in the language (or better yet, + code) domain will reveal a lot more biology than scaling on nucleotides or proteins alone. - My view is that the next opportunity of combining lab and AI will be massive-scale hypothesis testing with LLMs. Currently a multiplexed assay generally tries to test a handful of ideas because the humans are bandwidth limited in designing properly powered experiments. LLM's can scale this very well. I have no doubt lots of good news will come on the impact of AI in biology in the next five years. I can't tell what paradigm will win, but my prediction is that it will not be the same paradigm imported from the LLM land (which will get a ton of funding anyways, because it's easy to pattern match for tech VCs).
Thereโ€™s a really pronounced divide between people who are on the AI side of things and people who are on the biology side of things. Even among people working in AI, the divide persists based on where theyโ€™re focused. The AI side, adherents of scaling laws and the bitter lesson, believe that AI getting better means AI being more capable of novel discoveries in medicine (that go beyond discovering new drugs in well understood targets or speeding up dry lab work etc). The biology people think the idea of a bitter lesson in biology is kind of ridiculous and that regardless of AIโ€™s capabilities itโ€™s going to be at least 5-10 years until you could hope to reliably simulate human toxicology in silico (right now AI/ML canโ€™t even reliably simulate in vivo studies like binding affinities or functional assays). Even the most AI pilled of them, who will list all the areas that AI can reduce the time spent on various areas, donโ€™t think the concept of AI discovering novel targets or generating new understanding of causal biology is realistic in the near term. Most, in fact, will readily characterize the assumption that even an โ€œASIโ€ level model could find cures hidden in our current understanding (without expanding that understanding with significantly more biological data) as patently false. This was a big part of why we wanted to experience it ourselves by using AI for a (very) small portion of what it can do in biology. It was both mind blowing and revealed some poignant insights into how different the problem of making AI better at something like coding is to the problems of biology. Even if it unlocked the ability to generate new molecules at a stunning pace, its simulation (in silico) of binding only had 49% accuracy. And if we wanted to improve that, weโ€™d need to feed it back the data from the lab - data that took about 30x as long to get as it took to generate the molecules. And these were tests in a test tube (turn of phrase), imagine how long it would take if we wanted to improve the model with data that required recruiting humans and monitoring their reactions. Human biology is very complex, and adding a drug - especially a novel drug and doubly so for a novel drug acting on a novel mechanism - means mapping interactions that have nth-order-effects that we donโ€™t have the data to predict. Virtual cells have made advancements enabled by AI, but the perturbations equate to a small fraction of the possible. This is before you even get into curing disease, especially uniquely human diseases. Brain function involves cellular identities that are unique to humans, meaning animal studies are of little use. How long does it take to understand what the side effects of chronic administration of a drug in humans are? AI can speed up analyzing the data, or designing the trial or even getting it approvedโ€ฆbut it canโ€™t make a 6 month trial complete in 3 months. I think its important to point out that significant advancements can be made in AI that contribute to biology, while also being realistic about how much it speeds up something as complex as curing a specific cancer or diseases of the brain.
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Benjamin L. Oakes retweeted
A prevention conundrum: - LDL-C lowering is prioritized after an ASCVD event because near-term absolute risk is high. - Yet relative benefit is greater before a first event and at younger ages. sciencedirect.com/science/arโ€ฆ (Burger PM et al Atherosclerosis 2024) We need to keep moving toward improving high lifetime-risk identification before the disease declares itself.
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Next week, our CEO @BenjaminLOakes will speak at the @Biocom CEO Summit in Sonoma. Industry leaders can tune in for the โ€œCEO Perspectives: Pharma BD&Lโ€ session โ€“ a real-world tutorial for biotech CEOs and executive teams โ€“ on Friday, October 23. This #BiocomCEOSummit panel will walk through how business development deals actually move through biotech and pharmaceutical organizations, breaking down the practical mechanics, workflows, and decision points that shape whether a deal gets done or stalls along the way.
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Benjamin L. Oakes retweeted
Most clinicians & scientists donโ€™t set out to start a company. They set out to solve a problem. At #ScientificSession26, our first-ever Innovation Forum with @AHAScience opens with The Entrepreneurโ€™s Journey: a candid talk on getting from idea to impact. professional.heart.org/en/meโ€ฆ
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Benjamin L. Oakes retweeted
Welcome to lesson #4 of ๐—ฆ๐—ฐ๐—ฟ๐—ถ๐—ฏ๐—ฒ ๐—ฆ๐˜†๐—น๐—น๐—ฎ๐—ฏ๐˜‚๐˜€, our educational series exploring how genetic medicine can tackle some of healthโ€™s most critical challenges. Now that weโ€™ve covered CRISPRโ€™s surprising and unassuming origin story, weโ€™ll boil down how the technology actually works โ€“ and itโ€™s much easier to understand than you might expect. Leave any questions down in the comments! ๐Ÿ“– ๐—›๐—ผ๐˜„ ๐—–๐—ฅ๐—œ๐—ฆ๐—ฃ๐—ฅ ๐—ช๐—ผ๐—ฟ๐—ธ๐˜€: ๐—ฃ๐—ฎ๐—ฟ๐˜ ๐Ÿญ It may seem complex, but the beauty of CRISPR is that itโ€™s a remarkably elegant tool. That simplicity is why scientists around the world keep finding new uses for it beyond its original purpose as an immune defense system. In our last Syllabus lesson, we learned that CRISPR harnesses biological functions from bacteria that allow them to fight viruses. The entire CRISPR system has two main components: the ๐—–๐—ฎ๐˜€ ๐—ฒ๐—ป๐˜‡๐˜†๐—บ๐—ฒ and the ๐—ด๐˜‚๐—ถ๐—ฑ๐—ฒ ๐—ฅ๐—ก๐—” (โ€œ๐—ด๐—ฅ๐—ก๐—”โ€). CRISPRโ€™s revolutionary function is its ability to recognize, attach to and edit pieces of our DNA that predispose us to disease. In order to do this, it needs to identify the problematic sequence of DNA, and then cut it so the cellโ€™s own repair machinery can modify it. Thatโ€™s where the gRNA and Cas come in. The gRNA finds the target DNA, and the Cas enzyme cuts it. Read all about it in our full post on Substack, and stay tuned for a deeper dive in part 2! ๐Ÿ”— scribetx.substack.com/p/scriโ€ฆ
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Benjamin L. Oakes retweeted
Itโ€™s #WorldHeartDay and #CholesterolEducationMonth. Take action against cardiovascular disease for yourself and your loved ones by knowing the numbers that make it the leading cause of death ๐Ÿ‘‡ โค๏ธโ€๐Ÿฉน Every 40 seconds, someone in the US has a heart attack. โค๏ธโ€๐Ÿฉน Americans have more than 1.5 million heart attacks and strokes each year. โค๏ธโ€๐Ÿฉน About 1 in 5 heart attacks are silent. โค๏ธโ€๐Ÿฉน 63 million US adults have high LDL cholesterol (LDL-C), known as โ€œbad cholesterol.โ€ โค๏ธโ€๐Ÿฉน LDL-C is one of the largest drivers of heart disease, but only 20% of US patients with high LDL-C get treated. To protect your #HeartHealth, get your LDL-C checked and treated early. Recent studies show: ๐Ÿ’ก People at lower risk of heart disease may gain the most from lowering LDL-C. ๐Ÿ’ก The benefits build over time: Keeping LDL-C lower for longer could mean fewer procedures down the line. Sources: @American_Heart, @CDCgov, European Journal of Preventive Cardiology, American Journal of Preventive Cardiology Learn about our work at Scribe to develop CRISPR-based genetic medicines that are safe and potent enough to address high LDL-C โ€“ among other major risk factors of cardiometabolic disease โ€“ to improve heart health for all: scribetx.com
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Benjamin L. Oakes retweeted
Iโ€™ll be presenting in Scribe technology platform in two venues this week. Thanks to everyone who tunes in!
This week, tune into two events where our VP of Research Sarah Denny will break down the science and engineering approach behind our CRISPR-based genetic medicines. 1๏ธโƒฃ 7th Genome Editing Therapeutics Summit hosted by the Cell & Gene Therapy Event Series, AKA #crispr2.0 (September 28-30, Boston) 2๏ธโƒฃ Tech Spotlight: AI and CRISPR Genome Editing Webinar hosted by @Tech_Networks (September 29, virtual)
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Benjamin L. Oakes retweeted
This week, tune into two events where our VP of Research Sarah Denny will break down the science and engineering approach behind our CRISPR-based genetic medicines. 1๏ธโƒฃ 7th Genome Editing Therapeutics Summit hosted by the Cell & Gene Therapy Event Series, AKA #crispr2.0 (September 28-30, Boston) 2๏ธโƒฃ Tech Spotlight: AI and CRISPR Genome Editing Webinar hosted by @Tech_Networks (September 29, virtual)
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Benjamin L. Oakes retweeted
Welcome to lesson #3 of ๐—ฆ๐—ฐ๐—ฟ๐—ถ๐—ฏ๐—ฒ ๐—ฆ๐˜†๐—น๐—น๐—ฎ๐—ฏ๐˜‚๐˜€, our educational series exploring how genetic medicine can tackle some of healthโ€™s most critical challenges. We previously covered the potential of genetic medicine to restore and improve everyone's health. In this post, weโ€™ll go back in time to understand where CRISPR โ€“ one of the most powerful technologies for genetic medicine โ€“ came from and how itโ€™s being used to develop breakthrough therapies today. Have a question or a topic we should cover in a future post? Drop it in the comments. ๐Ÿ“– ๐—›๐—ผ๐˜„ ๐—–๐—ฅ๐—œ๐—ฆ๐—ฃ๐—ฅ ๐˜„๐—ฎ๐˜€ ๐—ฑ๐—ถ๐˜€๐—ฐ๐—ผ๐˜ƒ๐—ฒ๐—ฟ๐—ฒ๐—ฑ Decades before CRISPR was harnessed into a gene editing technology, scientists understood that DNA holds the natural blueprint for disease prevention. The problem was turning that knowledge into safe, reliable treatments. They lacked a tool precise enough to make efficient, accurate edits to the human genome. The discovery of CRISPR closed that gap, and it all started with bacteria. For the full post, head over to our Substack where we cover: โ€ข The origin of CRISPR as a natural defense for bacteria to fend off viruses โ€ข How Jennifer Doudna (one of Scribeโ€™s co-founders) and Emmanuelle Charpentier showed that CRISPR could be engineered and reprogrammed to target any piece of DNA, not just viral DNA โ€ข How CRISPR is being used today to improve human health across the globe ๐Ÿ”— scribetx.substack.com/p/scriโ€ฆ โœ๏ธ Image credit: IGI Glossary Icon Collection by Christine Liu of Two Photon Art for the Innovative Genomics Institute
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Benjamin L. Oakes retweeted
Hear directly from our CEO @BenjaminLOakes about Scribeโ€™s lead asset STX-1150, a clinical-stage epigenetic silencing therapy purpose-built to repress PCSK9, lower LDL cholesterol, and offer durable cardiovascular risk reduction to patients in need. More on our cardiometabolic pipeline, engineering approach compared to first-generation CRISPR technologies, and more in his conversation with @bradloncar. Full video is up on @BiotechTV: biotechtv.com/post/scribe-thโ€ฆ
๐‡๐‚๐– ๐Ÿ๐ŸŽ๐Ÿ๐Ÿ”: @scribetx which represents the next generation of CRISPR, had its IPO in July - the first clinical program targets PCSK9. With Co-Founder & CEO @BenjaminLOakes. $SCTX Full video: biotechtv.com/post/scribe-thโ€ฆ
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Thanks to @bradloncar and @BiotechTV for the opportunity to discuss our approach and pipeline!
๐‡๐‚๐– ๐Ÿ๐ŸŽ๐Ÿ๐Ÿ”: @scribetx which represents the next generation of CRISPR, had its IPO in July - the first clinical program targets PCSK9. With Co-Founder & CEO @BenjaminLOakes. $SCTX Full video: biotechtv.com/post/scribe-thโ€ฆ
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Benjamin L. Oakes retweeted
๐‡๐‚๐– ๐Ÿ๐ŸŽ๐Ÿ๐Ÿ”: @scribetx which represents the next generation of CRISPR, had its IPO in July - the first clinical program targets PCSK9. With Co-Founder & CEO @BenjaminLOakes. $SCTX Full video: biotechtv.com/post/scribe-thโ€ฆ
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Benjamin L. Oakes retweeted
๐Ÿ‘‰ Atherosclerosis is complex. Its root cause is not. โ†’ Inflammation modifies the response. โ†’ Hypertension accelerates the damage. โ†’ Diabetes amplifies the risk. โ†’ Smoking fuels the process. ๐Ÿ‘†But without apoB-containing lipoproteins entering and being retained in the arterial wall, there is no atherosclerotic plaque. ๐Ÿ‘‰LDL is not just another risk factor. It is the main causal driver of atherosclerosis. No cholesterol deposition. No plaque. @society_eas
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Could not agree more!
๐Ÿ‘‰ Atherosclerosis is complex. Its root cause is not. โ†’ Inflammation modifies the response. โ†’ Hypertension accelerates the damage. โ†’ Diabetes amplifies the risk. โ†’ Smoking fuels the process. ๐Ÿ‘†But without apoB-containing lipoproteins entering and being retained in the arterial wall, there is no atherosclerotic plaque. ๐Ÿ‘‰LDL is not just another risk factor. It is the main causal driver of atherosclerosis. No cholesterol deposition. No plaque. @society_eas
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Benjamin L. Oakes retweeted
๐Ÿงฌ๐Ÿ” We built two-factor authentication into gene silencing. Read about it in our new blog and paper โ€“ links in the comments. We call this approach ๐—ฆ๐—ฒ๐—พ๐˜‚๐—ฒ๐—ป๐˜๐—ถ๐—ฎ๐—น ๐—ฃ๐—ฟ๐—ผ๐—ผ๐—ณ๐—ฟ๐—ฒ๐—ฎ๐—ฑ๐—ถ๐—ป๐—ด. Itโ€™s engineered into ELXR, the epigenetic silencing technology built on our CRISPR-CasX platform. The results? ๐—จ๐—น๐˜๐—ฟ๐—ฎ-๐—น๐—ผ๐—ป๐—ด ๐—ด๐—ฒ๐—ป๐—ฒ ๐˜€๐—ถ๐—น๐—ฒ๐—ป๐—ฐ๐—ถ๐—ป๐—ด ๐˜„๐—ถ๐˜๐—ต๐—ผ๐˜‚๐˜ ๐—ฐ๐—ต๐—ฎ๐—ป๐—ด๐—ถ๐—ป๐—ด ๐—ฎ๐—ป๐˜† ๐——๐—ก๐—”. โ†’ More than 10-fold greater specificity โ†’ More than 4-fold greater silencing activity across every target tested โ†’ No toxicity in stress tests, rescuing growth defects observed with Cas9-based silencers โ†’ Mechanism mirrors the cellโ€™s natural regulation Learn more about this work led by Emeric J Charles, Christie C Sze, @BenjaminLOakes, Sarah Denny, @jdf_ev, and team below.
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Benjamin L. Oakes retweeted
This week, donโ€™t miss these upcoming talks by our co-founders: 1๏ธโƒฃ Fireside chat with CEO @BenjaminLOakes at the @HCWCO 28th Annual Global Investment Conference. This will take place Tuesday, September 15 at 2:30 p.m. ET in New York. #HCWConference 2๏ธโƒฃ Panel discussion where VP of External Innovation @BStaahl will discuss โ€œAdvances in Gene Editingโ€ at the Cell & Gene Therapy Symposium. The session begins Thursday, September 17 at 11:00 a.m. PT in La Jolla and is hosted by the @CALifeSciences, @CIRMnews, and Gene Therapy Initiative at @UCSanDiego.
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Benjamin L. Oakes retweeted
๐—ง๐—ต๐—ฒ ๐—™๐˜‚๐˜๐˜‚๐—ฟ๐—ถ๐˜€๐˜: ๐—ง๐—ฒ๐—ฐ๐—ต๐—ป๐—ผ๐—น๐—ผ๐—ด๐—ถ๐—ฒ๐˜€ ๐—ง๐—ต๐—ฎ๐˜ ๐—–๐—ผ๐˜‚๐—น๐—ฑ ๐—ฅ๐—ฒ๐—ฑ๐—ฒ๐—ณ๐—ถ๐—ป๐—ฒ ๐—›๐˜‚๐—บ๐—ฎ๐—ป๐—ถ๐˜๐˜† โ€” ๐—ผ๐—ฟ ๐—ก๐—ฒ๐˜ƒ๐—ฒ๐—ฟ ๐—™๐˜‚๐—น๐—น๐˜† ๐——๐—ฒ๐—น๐—ถ๐˜ƒ๐—ฒ๐—ฟ ๐˜๐˜ถ๐˜ด๐˜ช๐˜ฐ๐˜ฏ, ๐˜ข๐˜ฅ๐˜ท๐˜ข๐˜ฏ๐˜ค๐˜ฆ๐˜ฅ ๐˜ฏ๐˜ถ๐˜ค๐˜ญ๐˜ฆ๐˜ข๐˜ณ, ๐˜ˆ๐˜, ๐˜ฒ๐˜ถ๐˜ข๐˜ฏ๐˜ต๐˜ถ๐˜ฎ ๐˜ต๐˜ฆ๐˜ค๐˜ฉ๐˜ฏ๐˜ฐ๐˜ญ๐˜ฐ๐˜จ๐˜ช๐˜ฆ๐˜ด, ๐˜ฃ๐˜ช๐˜ฐ๐˜ฎ๐˜ฆ๐˜ฅ๐˜ช๐˜ค๐˜ช๐˜ฏ๐˜ฆ, ๐˜ข๐˜ฏ๐˜ฅ ๐˜จ๐˜ฆ๐˜ฏ๐˜ฆ๐˜ต๐˜ช๐˜ค ๐˜ฆ๐˜ฏ๐˜จ๐˜ช๐˜ฏ๐˜ฆ๐˜ฆ๐˜ณ๐˜ช๐˜ฏ๐˜จ Next week, our co-founder and CEO @BenjaminLOakes will discuss todayโ€™s most ambitious and promising technologies โ€“ including gene editing and silencing โ€“ at #IPAVision 2026, the Institute for Portfolio Alternativesโ€™ premier event. Panel details below. ๐Ÿ“… Wednesday, September 16, 2026 ๐Ÿ•— 5:15-6:05 p.m. ET ๐Ÿ“ Atlanta
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Benjamin L. Oakes retweeted
This morning at the @WellsFargo Healthcare Conference, our co-founder and CEO @BenjaminLOakes will participate in a fireside chat. More information below ๐Ÿ‘‡ ๐Ÿ“… Wednesday, September 9, 2026 ๐Ÿ•— 8:00 a.m. ET ๐Ÿ“ Boston
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Benjamin L. Oakes retweeted
Epigenetic silencing made the @nytimes today. The real-world example for how it could improve heart health: Scribeโ€™s single shot designed to reduce LDL cholesterol. Hereโ€™s how it works. While gene editing rewrites the DNA you were born with, our silencing approach changes how a gene behaves and leaves the code intact. Instead of a daily statin, a single dose of our epigenetic silencing therapy STX-1150 represses the PCSK9 gene in the liver that destroys the receptors your body uses to clear LDL-C, keeping the levels of this โ€˜badโ€™ cholesterol lowered for years and drastically lowering the risk of heart disease. STX-1150 is in clinical trials now. For more details, read the Times story and check out our website in the links below.
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Benjamin L. Oakes retweeted
Welcome back to ๐—ฆ๐—ฐ๐—ฟ๐—ถ๐—ฏ๐—ฒ ๐—ฆ๐˜†๐—น๐—น๐—ฎ๐—ฏ๐˜‚๐˜€, where we explore how genetic medicine can tackle some of the most critical challenges in human health. Last time, we defined genetic medicine and discussed how it differs from traditional forms of treatment. In this lesson, weโ€™ll dive into the many ways that this technology can be harnessed to improve health worldwide. ๐Ÿ“– ๐—›๐—ผ๐˜„ ๐—š๐—ฒ๐—ป๐—ฒ๐˜๐—ถ๐—ฐ ๐— ๐—ฒ๐—ฑ๐—ถ๐—ฐ๐—ถ๐—ป๐—ฒ ๐—–๐—ฎ๐—ป ๐—ฅ๐—ฒ๐˜€๐˜๐—ผ๐—ฟ๐—ฒ ๐—ฎ๐—ป๐—ฑ ๐—œ๐—บ๐—ฝ๐—ฟ๐—ผ๐˜ƒ๐—ฒ ๐—›๐—ฒ๐—ฎ๐—น๐˜๐—ต ๐—ณ๐—ผ๐—ฟ ๐—˜๐˜ƒ๐—ฒ๐—ฟ๐˜†๐—ผ๐—ป๐—ฒ The goal of genetic medicine is to directly remedy the underlying biology of harmful disease to restore, improve, and extend healthy quality of life. Instead of managing symptoms and requiring daily medication like traditional drugs may, genetic medicine can prevent and even potentially cure disease before it strikes. The Human Genome Project, launched in 1990, laid the foundation of genetic medicine and gave us a roadmap to understand how our DNA can drive or prevent disease. In fact, some people are born with natural, lifelong protection from specific diseases written into their DNA. That's nature's blueprint, which genetic medicine aims to read and deliver to everyone. For the full post, head over to our Substack where we cover: โ€ข Why genetic medicine has so far been largely limited to rare, terminal diseases โ€ข An introduction to Victoria Gray, the first patient to be treated with CRISPR genetic medicine for sickle cell disease โ€ข Ongoing advances in CRISPR technology to support treatments for a much wider range of people and that are not only effective enough but also safe enough to address common diseases โ€ข Genetic medicine targeting the worldโ€™s leading cause of death: heart disease โ€ข Examples of genetic switches that can raise or lower cardiovascular risk ๐Ÿ”— scribetx.substack.com/p/scriโ€ฆ ๐—จ๐—ฝ ๐—ป๐—ฒ๐˜…๐˜: ๐—” ๐—ฑ๐—ฒ๐—ฒ๐—ฝ๐—ฒ๐—ฟ ๐—ฑ๐—ถ๐˜ƒ๐—ฒ ๐—ถ๐—ป๐˜๐—ผ ๐—–๐—ฅ๐—œ๐—ฆ๐—ฃ๐—ฅ-๐—ฏ๐—ฎ๐˜€๐—ฒ๐—ฑ ๐—ด๐—ฒ๐—ป๐—ฒ๐˜๐—ถ๐—ฐ ๐—บ๐—ฒ๐—ฑ๐—ถ๐—ฐ๐—ถ๐—ป๐—ฒ๐˜€ In our next few posts, weโ€™ll explore: ๐Ÿงฌ How CRISPR technology works ๐Ÿง‘โ€๐Ÿ”ฌ CRISPRโ€™s surprising origin story! ๐Ÿ› ๏ธ Engineering CRISPR into different medicines and tools Let us know what other questions youโ€™d like us to cover.
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