
CEO @scribetx | at the interface of molecular and genetic engineering | designing CRISPR to be safe enough for all
Joined April 2011
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๐ง๐ผ๐ฑ๐ฎ๐, ๐ฆ๐ฐ๐ฟ๐ถ๐ฏ๐ฒ ๐ง๐ต๐ฒ๐ฟ๐ฎ๐ฝ๐ฒ๐๐๐ถ๐ฐ๐ ๐ผ๐ณ๐ณ๐ถ๐ฐ๐ถ๐ฎ๐น๐น๐ ๐ฏ๐ฒ๐ฐ๐ฎ๐บ๐ฒ ๐ฎ ๐ฐ๐น๐ถ๐ป๐ถ๐ฐ๐ฎ๐น-๐๐๐ฎ๐ด๐ฒ ๐ฏ๐ถ๐ผ๐๐ฒ๐ฐ๐ต๐ป๐ผ๐น๐ผ๐ด๐ ๐ฐ๐ผ๐บ๐ฝ๐ฎ๐ป๐ ๐๐ถ๐๐ต ๐๐ต๐ฒ ๐ฎ๐ฑ๐๐ฎ๐ป๐ฐ๐ฒ๐บ๐ฒ๐ป๐ ๐ผ๐ณ ๐ฆ๐ง๐ซ-๐ญ๐ญ๐ฑ๐ฌ, a novel ๐ช๐ฏ ๐ท๐ช๐ท๐ฐ epigenetic CRISPR therapy designed to deliver ultra-durable lowering of โbad 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.
๐๐๐ ๐๐ต๐ฎ๐ ๐ฒ๐
๐ฐ๐ถ๐๐ฒ๐ ๐บ๐ฒ ๐บ๐ผ๐๐ ๐ถ๐ ๐๐ต๐ฎ๐ ๐๐ต๐ถ๐ ๐ฐ๐ผ๐๐น๐ฑ ๐บ๐ฒ๐ฎ๐ป ๐ณ๐ผ๐ฟ ๐ฝ๐ฎ๐๐ถ๐ฒ๐ป๐๐ ๐น๐ถ๐ธ๐ฒ ๐บ๐๐๐ฒ๐น๐ณ.
As someone at high risk of ASCVD, like roughly one-third of adults in the U.S., Iโve 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, thatโs 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.
๐ง๐ต๐ฒ ๐ณ๐๐๐๐ฟ๐ฒ ๐ผ๐ณ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ ๐๐ต๐ผ๐๐น๐ฑ ๐ฎ๐๐ฝ๐ถ๐ฟ๐ฒ ๐๐ผ ๐บ๐ผ๐ฟ๐ฒ ๐๐ต๐ฎ๐ป ๐ฐ๐ต๐ฎ๐ถ๐ป๐ถ๐ป๐ด ๐ฝ๐ฎ๐๐ถ๐ฒ๐ป๐๐ ๐๐ผ ๐น๐ถ๐ณ๐ฒ๐น๐ผ๐ป๐ด ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ฎ๐๐ถ๐ผ๐ป๐.
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 worldโs 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 ๐ฏ๐ฒ๐น๐ถ๐ฒ๐๐ฒ ๐ฎ ๐ป๐ฒ๐ ๐ฒ๐ฟ๐ฎ ๐ผ๐ณ ๐๐ต๐ฒ๐ฟ๐ฎ๐ฝ๐ฒ๐๐๐ถ๐ฐ๐ ๐ถ๐ ๐ผ๐ป ๐๐ต๐ฒ ๐ต๐ผ๐ฟ๐ถ๐๐ผ๐ป, ๐ผ๐ป๐ฒ ๐๐ต๐ฒ๐ฟ๐ฒ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ ๐ฐ๐ฎ๐ป ๐ฑ๐๐ฟ๐ฎ๐ฏ๐น๐ ๐ฟ๐ฒ๐๐ต๐ฎ๐ฝ๐ฒ ๐น๐ผ๐ป๐ด-๐๐ฒ๐ฟ๐บ ๐ต๐ฒ๐ฎ๐น๐๐ต ๐ฎ๐ป๐ฑ ๐ฎ๐น๐น๐ผ๐ ๐๐ ๐ฎ๐น๐น ๐๐ผ ๐น๐ถ๐๐ฒ ๐น๐ผ๐ป๐ด๐ฒ๐ฟ, ๐ต๐ฒ๐ฎ๐น๐๐ต๐ถ๐ฒ๐ฟ ๐น๐ถ๐๐ฒ๐ ๐๐ถ๐๐ต ๐ด๐ฟ๐ฒ๐ฎ๐๐ฒ๐ฟ ๐ณ๐ฟ๐ฒ๐ฒ๐ฑ๐ผ๐บ.
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 Australiaโs @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).
Details๐งต
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).
Thereโs 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 theyโre 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 AIโs capabilities itโs going to be at least 5-10 years until you could hope to reliably simulate human toxicology in silico (right now AI/ML canโt 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, donโt 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 โASIโ 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, weโd 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 donโt 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 approvedโฆbut it canโt 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.
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/arโฆ (Burger PM et al Atherosclerosis 2024)
We need to keep moving toward improving high lifetime-risk identification before the disease declares itself.
Benjamin L. Oakes retweeted
Next week, our CEO @BenjaminLOakes will speak at the @Biocom CEO Summit in Sonoma. Industry leaders can tune in for the โCEO 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.
Benjamin L. Oakes retweeted
Most clinicians & scientists donโt 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 Entrepreneurโs Journey: a candid talk on getting from idea to impact. professional.heart.org/en/meโฆ
Benjamin L. Oakes retweeted
Welcome to lesson #4 of ๐ฆ๐ฐ๐ฟ๐ถ๐ฏ๐ฒ ๐ฆ๐๐น๐น๐ฎ๐ฏ๐๐, our educational series exploring how genetic medicine can tackle some of healthโs most critical challenges.
Now that weโve covered CRISPRโs surprising and unassuming origin story, weโll boil down how the technology actually works โ and itโs much easier to understand than you might expect.
Leave any questions down in the comments!
๐ ๐๐ผ๐ ๐๐ฅ๐๐ฆ๐ฃ๐ฅ ๐ช๐ผ๐ฟ๐ธ๐: ๐ฃ๐ฎ๐ฟ๐ ๐ญ
It may seem complex, but the beauty of CRISPR is that itโs 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 ๐๐ฎ๐ ๐ฒ๐ป๐๐๐บ๐ฒ and the ๐ด๐๐ถ๐ฑ๐ฒ ๐ฅ๐ก๐ (โ๐ด๐ฅ๐ก๐โ).
CRISPRโs 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 cellโs own repair machinery can modify it. Thatโs 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!
๐ scribetx.substack.com/p/scriโฆ
Benjamin L. Oakes retweeted
Itโs #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 ๐
โค๏ธโ๐ฉน Every 40 seconds, someone in the US has a heart attack.
โค๏ธโ๐ฉน Americans have more than 1.5 million heart attacks and strokes each year.
โค๏ธโ๐ฉน About 1 in 5 heart attacks are silent.
โค๏ธโ๐ฉน 63 million US adults have high LDL cholesterol (LDL-C), known as โbad cholesterol.โ
โค๏ธโ๐ฉน 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:
๐ก People at lower risk of heart disease may gain the most from lowering LDL-C.
๐ก 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
Benjamin L. Oakes retweeted
Iโll 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๏ธโฃ 7th Genome Editing Therapeutics Summit hosted by the Cell & Gene Therapy Event Series, AKA #crispr2.0 (September 28-30, Boston)
2๏ธโฃ Tech Spotlight: AI and CRISPR Genome Editing Webinar hosted by @Tech_Networks (September 29, virtual)
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๏ธโฃ 7th Genome Editing Therapeutics Summit hosted by the Cell & Gene Therapy Event Series, AKA #crispr2.0 (September 28-30, Boston)
2๏ธโฃ Tech Spotlight: AI and CRISPR Genome Editing Webinar hosted by @Tech_Networks (September 29, virtual)
Benjamin L. Oakes retweeted
Welcome to lesson #3 of ๐ฆ๐ฐ๐ฟ๐ถ๐ฏ๐ฒ ๐ฆ๐๐น๐น๐ฎ๐ฏ๐๐, our educational series exploring how genetic medicine can tackle some of healthโs most critical challenges.
We previously covered the potential of genetic medicine to restore and improve everyone's health. In this post, weโll go back in time to understand where CRISPR โ one of the most powerful technologies for genetic medicine โ came from and how itโs 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.
๐ ๐๐ผ๐ ๐๐ฅ๐๐ฆ๐ฃ๐ฅ ๐๐ฎ๐ ๐ฑ๐ถ๐๐ฐ๐ผ๐๐ฒ๐ฟ๐ฒ๐ฑ
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:
โข The origin of CRISPR as a natural defense for bacteria to fend off viruses
โข How Jennifer Doudna (one of Scribeโs co-founders) and Emmanuelle Charpentier showed that CRISPR could be engineered and reprogrammed to target any piece of DNA, not just viral DNA
โข How CRISPR is being used today to improve human health across the globe
๐ scribetx.substack.com/p/scriโฆ
โ๏ธ Image credit: IGI Glossary Icon Collection by Christine Liu of Two Photon Art for the Innovative Genomics Institute
Benjamin L. Oakes retweeted
Hear directly from our CEO @BenjaminLOakes about Scribeโs 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-thโฆ
๐๐๐ ๐๐๐๐: @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-thโฆ
Thanks to @bradloncar and @BiotechTV for the opportunity to discuss our approach and pipeline!
๐๐๐ ๐๐๐๐: @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-thโฆ
Benjamin L. Oakes retweeted
๐๐๐ ๐๐๐๐: @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-thโฆ
Benjamin L. Oakes retweeted
๐ 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.
๐But without apoB-containing lipoproteins entering and being retained in the arterial wall, there is no atherosclerotic plaque.
๐LDL is not just another risk factor.
It is the main causal driver of atherosclerosis.
No cholesterol deposition. No plaque.
@society_eas
Could not agree more!
๐ 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.
๐But without apoB-containing lipoproteins entering and being retained in the arterial wall, there is no atherosclerotic plaque.
๐LDL is not just another risk factor.
It is the main causal driver of atherosclerosis.
No cholesterol deposition. No plaque.
@society_eas
Benjamin L. Oakes retweeted
๐งฌ๐ 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 ๐ฆ๐ฒ๐พ๐๐ฒ๐ป๐๐ถ๐ฎ๐น ๐ฃ๐ฟ๐ผ๐ผ๐ณ๐ฟ๐ฒ๐ฎ๐ฑ๐ถ๐ป๐ด. Itโs engineered into ELXR, the epigenetic silencing technology built on our CRISPR-CasX platform.
The results? ๐จ๐น๐๐ฟ๐ฎ-๐น๐ผ๐ป๐ด ๐ด๐ฒ๐ป๐ฒ ๐๐ถ๐น๐ฒ๐ป๐ฐ๐ถ๐ป๐ด ๐๐ถ๐๐ต๐ผ๐๐ ๐ฐ๐ต๐ฎ๐ป๐ด๐ถ๐ป๐ด ๐ฎ๐ป๐ ๐๐ก๐.
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 cellโs natural regulation
Learn more about this work led by Emeric J Charles, Christie C Sze, @BenjaminLOakes, Sarah Denny, @jdf_ev, and team below.
Benjamin L. Oakes retweeted
This week, donโt miss these upcoming talks by our co-founders:
1๏ธโฃ 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๏ธโฃ Panel discussion where VP of External Innovation @BStaahl will discuss โAdvances 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.
Benjamin L. Oakes retweeted
๐ง๐ต๐ฒ ๐๐๐๐๐ฟ๐ถ๐๐: ๐ง๐ฒ๐ฐ๐ต๐ป๐ผ๐น๐ผ๐ด๐ถ๐ฒ๐ ๐ง๐ต๐ฎ๐ ๐๐ผ๐๐น๐ฑ ๐ฅ๐ฒ๐ฑ๐ฒ๐ณ๐ถ๐ป๐ฒ ๐๐๐บ๐ฎ๐ป๐ถ๐๐ โ ๐ผ๐ฟ ๐ก๐ฒ๐๐ฒ๐ฟ ๐๐๐น๐น๐ ๐๐ฒ๐น๐ถ๐๐ฒ๐ฟ
๐๐ถ๐ด๐ช๐ฐ๐ฏ, ๐ข๐ฅ๐ท๐ข๐ฏ๐ค๐ฆ๐ฅ ๐ฏ๐ถ๐ค๐ญ๐ฆ๐ข๐ณ, ๐๐, ๐ฒ๐ถ๐ข๐ฏ๐ต๐ถ๐ฎ ๐ต๐ฆ๐ค๐ฉ๐ฏ๐ฐ๐ญ๐ฐ๐จ๐ช๐ฆ๐ด, ๐ฃ๐ช๐ฐ๐ฎ๐ฆ๐ฅ๐ช๐ค๐ช๐ฏ๐ฆ, ๐ข๐ฏ๐ฅ ๐จ๐ฆ๐ฏ๐ฆ๐ต๐ช๐ค ๐ฆ๐ฏ๐จ๐ช๐ฏ๐ฆ๐ฆ๐ณ๐ช๐ฏ๐จ
Next week, our co-founder and CEO @BenjaminLOakes will discuss todayโs 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.
๐
Wednesday, September 16, 2026
๐ 5:15-6:05 p.m. ET
๐ Atlanta
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 ๐
๐
Wednesday, September 9, 2026
๐ 8:00 a.m. ET
๐ Boston
Benjamin L. Oakes retweeted
Epigenetic silencing made the @nytimes today. The real-world example for how it could improve heart health: Scribeโs single shot designed to reduce LDL cholesterol.
Hereโs 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 โbadโ 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.
Benjamin L. Oakes retweeted
Welcome back to ๐ฆ๐ฐ๐ฟ๐ถ๐ฏ๐ฒ ๐ฆ๐๐น๐น๐ฎ๐ฏ๐๐, 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, weโll dive into the many ways that this technology can be harnessed to improve health worldwide.
๐ ๐๐ผ๐ ๐๐ฒ๐ป๐ฒ๐๐ถ๐ฐ ๐ ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ ๐๐ฎ๐ป ๐ฅ๐ฒ๐๐๐ผ๐ฟ๐ฒ ๐ฎ๐ป๐ฑ ๐๐บ๐ฝ๐ฟ๐ผ๐๐ฒ ๐๐ฒ๐ฎ๐น๐๐ต ๐ณ๐ผ๐ฟ ๐๐๐ฒ๐ฟ๐๐ผ๐ป๐ฒ
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:
โข Why genetic medicine has so far been largely limited to rare, terminal diseases
โข An introduction to Victoria Gray, the first patient to be treated with CRISPR genetic medicine for sickle cell disease
โข 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
โข Genetic medicine targeting the worldโs leading cause of death: heart disease
โข Examples of genetic switches that can raise or lower cardiovascular risk
๐ scribetx.substack.com/p/scriโฆ
๐จ๐ฝ ๐ป๐ฒ๐
๐: ๐ ๐ฑ๐ฒ๐ฒ๐ฝ๐ฒ๐ฟ ๐ฑ๐ถ๐๐ฒ ๐ถ๐ป๐๐ผ ๐๐ฅ๐๐ฆ๐ฃ๐ฅ-๐ฏ๐ฎ๐๐ฒ๐ฑ ๐ด๐ฒ๐ป๐ฒ๐๐ถ๐ฐ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ๐
In our next few posts, weโll explore:
๐งฌ How CRISPR technology works
๐งโ๐ฌ CRISPRโs surprising origin story!
๐ ๏ธ Engineering CRISPR into different medicines and tools
Let us know what other questions youโd like us to cover.




















