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
Fantastic to kick off AHA26 with a panel conversation at the innovation forum on the future of cardiovascular care with the fantastic @manesh_patelMD, @skathire and Lauren Cheung!
Please come say hi - come to the abstract session, late breaking science, and the Connor lecture. Look forward to engaging with all of our colleagues. @AHAScience
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
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
๐๐ผ๐ฒ๐ ๐บ๐ฎ๐ธ๐ถ๐ป๐ด ๐ฎ ๐๐ฅ๐๐ฆ๐ฃ๐ฅ ๐ฏ๐ฎ๐๐ฒ ๐ฒ๐ฑ๐ถ๐๐ผ๐ฟ ๐บ๐ผ๐ฟ๐ฒ ๐ฎ๐ฐ๐๐ถ๐๐ฒ ๐ฎ๐น๐๐ผ ๐ฟ๐ฒ๐พ๐๐ถ๐ฟ๐ฒ ๐ถ๐ ๐๐ผ ๐ฏ๐ฒ ๐น๐ฒ๐๐ ๐๐ฎ๐ณ๐ฒ?
Last week, we launched โ๐ข๐ณ๐ณ๐ถ๐ฐ๐ฒ ๐๐ผ๐๐ฟ๐,โ ๐ฎ ๐ป๐ฒ๐ ๐ค&๐ ๐๐ฒ๐ฟ๐ถ๐ฒ๐ where we sit down with scientific leaders to dig into their latest findings. In the first installment, I spoke with Scribe co-founder David Savage about a new study from his lab, co-led by Maria Lukarska and Luke Oltrogge, examining this question and outlining the activity <> specificity spectrum adenine base editors.
In short, the deaminase component of a base editor can and does act independently of the CRISPR component. When it does, it behaves less like a targeted editor and more like an untargeted mutagen.ย
Now, this is not entirely new, and many observations of CRISPR-independent DNA and RNA editing, as well as Cas-dependent off-targets and bystander effects have been simmering beneath the surface across previous reports for years.ย
What makes this newest work so compelling is its breadth. ๐๐ ๐พ๐๐ฎ๐ป๐๐ถ๐๐ฎ๐๐ถ๐๐ฒ๐น๐ ๐ฒ๐๐ฎ๐น๐๐ฎ๐๐ถ๐ป๐ด ๐ผ๐๐ฒ๐ฟ ๐๐ฒ๐ป ๐๐ต๐ผ๐๐๐ฎ๐ป๐ฑ ๐๐๐ ๐๐ฎ๐ฟ๐ถ๐ฎ๐ป๐๐ ๐ฎ๐ฐ๐ฟ๐ผ๐๐ ๐ฎ๐น๐น ๐๐ต๐ฒ๐๐ฒ ๐ผ๐ณ๐ณ-๐๐ฎ๐ฟ๐ด๐ฒ๐ ๐ฎ๐ฐ๐๐ถ๐๐ถ๐๐ถ๐ฒ๐, ๐๐ต๐ฒ ๐ฎ๐๐๐ต๐ผ๐ฟ๐ ๐๐๐ฟ๐ป ๐๐ฐ๐ฎ๐๐๐ฒ๐ฟ๐ฒ๐ฑ ๐๐ฎ๐ฟ๐ป๐ถ๐ป๐ด ๐๐ถ๐ด๐ป๐ ๐ถ๐ป๐๐ผ ๐ฎ ๐ฑ๐ฒ๐๐ฎ๐ถ๐น๐ฒ๐ฑ ๐บ๐ฎ๐ฝ ๐ฐ๐ผ๐น๐น๐ฎ๐ฝ๐๐ถ๐ป๐ด ๐ถ๐ป๐๐ฟ๐ถ๐ป๐๐ถ๐ฐ ๐ฑ๐ฒ๐ฎ๐บ๐ถ๐ป๐ฎ๐๐ฒ ๐ฎ๐ฐ๐๐ถ๐๐ถ๐๐ ๐ฎ๐ป๐ฑ ๐ผ๐ณ๐ณ-๐๐ฎ๐ฟ๐ด๐ฒ๐ ๐ฒ๐ณ๐ณ๐ฒ๐ฐ๐๐ ๐ผ๐ป๐๐ผ ๐ฎ ๐๐ถ๐ป๐ด๐น๐ฒ ๐ฝ๐ฟ๐ฒ๐ฑ๐ถ๐ฐ๐๐ถ๐๐ฒ ๐ฎ๐
๐ถ๐. This is all despite the specificity the CRISPR molecule was assumed to bring.
As Dr. Savage lays out, the counterintuitive clinical takeaway is that, unlike with normal allosteric proofreading CRISPR systems, ๐๐ถ๐๐ต ๐ฏ๐ฎ๐๐ฒ ๐ฒ๐ฑ๐ถ๐๐ผ๐ฟ๐ ๐๐ฒ ๐๐ต๐ผ๐๐น๐ฑ ๐ฎ๐ถ๐บ ๐๐ผ ๐๐๐ฒ ๐๐ต๐ฒ ๐บ๐ผ๐น๐ฒ๐ฐ๐๐น๐ฒ๐ ๐๐ถ๐๐ต ๐๐ต๐ฒ ๐น๐ผ๐๐ฒ๐๐ ๐ฝ๐ผ๐๐๐ถ๐ฏ๐น๐ฒ ๐ฎ๐ฐ๐๐ถ๐๐ถ๐๐, while also working hard to minimize both the dose and exposure time.ย
โ๐๐๐ ๐๐ค๐จ๐ ๐ข๐๐ ๐๐จ ๐ฉ๐๐ ๐ฅ๐ค๐๐จ๐ค๐ฃ.โ
๐ฅ๐ฒ๐ฎ๐ฑ ๐๐ต๐ฒ ๐ณ๐๐น๐น ๐ถ๐ป๐๐ฒ๐ฟ๐๐ถ๐ฒ๐ below on Scribe Therapeuticsโ๐ ๐ฆ๐๐ฏ
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Full Q&A: scribetx.substack.com/p/q-anโฆ
Preprint: biorxiv.org/content/10.64898โฆ
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Kicking off the Scribe Syllabus with a fundamental question: "What is genetic medicine?" Our post below provides a formal definition. ๐๐๐ ๐ ๐๐ฎ๐ป๐ ๐๐ผ ๐ผ๐ณ๐ณ๐ฒ๐ฟ ๐ฎ ๐บ๐ผ๐ฟ๐ฒ ๐ต๐๐บ๐ฎ๐ป ๐๐ฎ๐ ๐๐ผ ๐๐ต๐ถ๐ป๐ธ ๐ฎ๐ฏ๐ผ๐๐ ๐ถ๐:
For decades, medicine has largely offered us two paths. First, pharmacological interventions that may keep us healthier but require lifelong adherence, monitoring, and continuous access. Second, surgical interventions that can, in rare instances, correct an underlying problem.
Genetic medicine has the potential to bridge these worlds, to change the underlying biology of disease with an intervention that provides enduring benefit.
If ๐๐ผ๐ฑ๐ฎ๐โ๐ ๐ฐ๐ต๐ฟ๐ผ๐ป๐ถ๐ฐ ๐๐ฟ๐ฒ๐ฎ๐๐บ๐ฒ๐ป๐ ๐ฝ๐ฎ๐ฟ๐ฎ๐ฑ๐ถ๐ด๐บ ๐ถ๐ ๐ฎ ๐ฆ๐ถ๐๐๐ฝ๐ต๐ฒ๐ฎ๐ป ๐๐๐ฟ๐๐ด๐ด๐น๐ฒ, requiring us to push the boulder of health uphill every day through pills, injections, monitoring, lifestyle changes, and appointments, genetic medicine offers a fundamentally different possibility: to shake the ground, reset the angle of the hill, and let the boulder roll forward more easily by modifying our biological processes toward health.
๐๐ฒ๐ป๐ฒ๐๐ถ๐ฐ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ ๐ฐ๐ฎ๐ป ๐ฏ๐ฒ ๐ฑ๐ฒ๐๐ถ๐ด๐ป๐ฒ๐ฑ ๐๐ผ ๐ด๐ถ๐๐ฒ ๐ฝ๐ฒ๐ผ๐ฝ๐น๐ฒ ๐ด๐ฟ๐ฒ๐ฎ๐๐ฒ๐ฟ ๐ฎ๐ด๐ฒ๐ป๐ฐ๐ ๐ผ๐๐ฒ๐ฟ ๐๐ต๐ฒ๐ถ๐ฟ ๐ต๐ฒ๐ฎ๐น๐๐ต, allowing them to alter the biological trajectory they inherited rather than manage its consequences for the rest of their lives.
Most importantly, genetic medicine could move healthcare from reacting to disease toward preventing it before it begins.
๐ฆ๐ผ, โ๐ช๐ต๐ฎ๐ ๐ถ๐ ๐ด๐ฒ๐ป๐ฒ๐๐ถ๐ฐ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ?โ ๐๐ฒ๐ป๐ฒ๐๐ถ๐ฐ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ ๐ถ๐ ๐ฎ ๐ณ๐๐๐๐ฟ๐ฒ ๐ถ๐ป ๐๐ต๐ถ๐ฐ๐ต ๐๐ฒ ๐ฐ๐ฎ๐ป ๐๐ถ๐๐ถ๐ ๐ผ๐๐ฟ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ฎ๐น ๐ฝ๐ฟ๐ผ๐๐ถ๐ฑ๐ฒ๐ฟ ๐ฎ๐ป๐ฑ ๐น๐ฒ๐ฎ๐๐ฒ ๐๐ฒ๐ ๐ผ๐ป ๐ฎ ๐ณ๐๐ป๐ฑ๐ฎ๐บ๐ฒ๐ป๐๐ฎ๐น๐น๐ ๐ต๐ฒ๐ฎ๐น๐๐ต๐ถ๐ฒ๐ฟ ๐ฝ๐ฎ๐๐ต, ๐๐ป๐๐ฒ๐๐ต๐ฒ๐ฟ๐ฒ๐ฑ ๐ณ๐ฟ๐ผ๐บ ๐๐ต๐ฒ ๐น๐ถ๐ณ๐ฒ๐น๐ผ๐ป๐ด ๐๐ฟ๐ฒ๐ฎ๐๐บ๐ฒ๐ป๐ ๐ฎ๐ฝ๐ฝ๐ฟ๐ผ๐ฎ๐ฐ๐ต ๐๐ต๐ฎ๐ ๐ถ๐ ๐ฐ๐ผ๐บ๐บ๐ผ๐ป๐ฝ๐น๐ฎ๐ฐ๐ฒ ๐๐ผ๐ฑ๐ฎ๐.
We are excited to kick off ๐ฆ๐ฐ๐ฟ๐ถ๐ฏ๐ฒ ๐ฆ๐๐น๐น๐ฎ๐ฏ๐๐, our new educational series exploring how genetic medicine can tackle some of the most critical challenges in human health.
Join us as we dive into how epigenetic silencing and gene editing technologies work, why they matter, and how they could change the future of human health.
๐ ๐ช๐ต๐ฎ๐ ๐๐ ๐๐ฒ๐ป๐ฒ๐๐ถ๐ฐ ๐ ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ?
Can medicine prevent disease at its source, for lifeโnot just temporarily manage its symptoms? Instead of a lifetime of pills, infusions, and risk monitoring, what if we could stop disease before it happens?
Many traditional drugs are designed to treat symptoms and be taken chronically for as long as the underlying disease persists, which in many cases can be for life. This makes staying adherent to treatment a challenge in the long term, with day-to-day burdens such as regular side effects, regular doctor visits, costs that can add up, needle self-injections that are uncomfortable, and just getting in the way of life generally.
Genetic medicine takes a different approach. It is designed to act on the biological instructions that contribute to disease, potentially preventing disease before it even starts and providing lifelong health benefits.
Deoxyribonucleic acid (๐๐ก๐) is the instruction manual for our bodies. Itโs found inside the majority of our cells, with individual sections called genes telling the body what proteins to produce and when to help it function.
Sometimes, faulty instructions can cause the body to produce a harmful protein, or make too much or little of a critical protein. These changes can contribute to disease.
Genetic medicine can intervene when our biological instructions go awry. Depending on the disease, it may correct a genetic instruction, silence an unwanted gene, or restore a function the body needs. This gives us a way to overcome the effects of harmful genes in a way weโve never been able to before.
๐๐ผ๐ ๐ด๐ฒ๐ป๐ฒ๐๐ถ๐ฐ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ ๐ต๐ฎ๐ ๐ฒ๐๐ผ๐น๐๐ฒ๐ฑ ๐ผ๐๐ฒ๐ฟ ๐๐ถ๐บ๐ฒ
Early approaches focused on adding a working copy of a gene when the original was missing or not functioning properly.
Scientists then developed medicines that act on ribonucleic acid, or ๐ฅ๐ก๐โthe temporary messages cells used to turn instructions from DNA into proteins.
In 2012, Scribe co-founder and Nobel Prize winner Dr. Jennifer Doudna transformed the field of genetic medicine by showing that a bacterial immune system called CRISPR-Cas9 could be programmed to target and cut a specific location in human DNA.
Her co-discovery of ๐๐ฅ๐๐ฆ๐ฃ๐ฅโshort for Clustered Regularly Interspaced Short Palindromic Repeatsโlaunched a new era of CRISPR-based genetic medicines. More to come on how CRISPR works in our Syllabus series.
Today, genetic medicine technologies are constantly improving. First, theyโre being engineered to edit DNA more effectively and safely, and second to control the activity of specific genes without changing the underlying DNA sequence.
These advances have moved us closer to having many purpose-built genetic medicine technologies to safely and effectively address disease at its source.
The goal of genetic medicine is to move beyond repeatedly managing the effects of disease and instead, remedy its underlying biology. This will lead to long-lasting benefits, including disease prevention and improvements in human healthspan.
๐จ๐ฝ ๐ป๐ฒ๐
๐: ๐ช๐ต๐ฎ๐ ๐ฑ๐ถ๐๐ฒ๐ฎ๐๐ฒ๐ ๐ฐ๐ฎ๐ป ๐ด๐ฒ๐ป๐ฒ๐๐ถ๐ฐ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ ๐ฎ๐ฑ๐ฑ๐ฟ๐ฒ๐๐?
Stay tuned for our next few posts, where weโll explore:
๐ How applications of genetic medicine and CRISPR could help restore and improve everyoneโs health
๐งฌ How CRISPR works and its surprising origin story
๐งโ๐ฌ Different ways that scientists have adapted and refined CRISPR for research and drug development
Let us know what other questions youโd like us to cover.
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๐๐บ๐ฎ๐ด๐ถ๐ป๐ฒ ๐ฎ ๐ณ๐๐๐๐ฟ๐ฒ ๐ถ๐ป ๐๐ต๐ถ๐ฐ๐ต ๐ผ๐๐ฟ ๐ต๐ฒ๐ฎ๐น๐๐ต ๐ถ๐ ๐ป๐ผ ๐น๐ผ๐ป๐ด๐ฒ๐ฟ ๐ฑ๐ฒ๐ณ๐ถ๐ป๐ฒ๐ฑ ๐ฏ๐ ๐๐ต๐ฒ ๐ด๐ฒ๐ป๐ฒ๐๐ถ๐ฐ๐ ๐๐ฒ ๐ถ๐ป๐ต๐ฒ๐ฟ๐ถ๐ ๐ฏ๐๐ ๐ฒ๐บ๐ฝ๐ผ๐๐ฒ๐ฟ๐ฒ๐ฑ ๐ฏ๐ ๐๐ต๐ฒ ๐ฏ๐ถ๐ผ๐น๐ผ๐ด๐ ๐๐ฒ ๐๐ป๐ฑ๐ฒ๐ฟ๐๐๐ฎ๐ป๐ฑ. A future in which genetic medicine could safely raise the baseline level of health for millions, a future in which cardioprotective genetics are accessible to everyone.
Over fifteen years ago I began a journey focused on dramatically improving genetic medicine approaches, and nine years ago we founded Scribe to formally launch this mission: ๐ฟ๐ฒ๐ฑ๐ฒ๐ณ๐ถ๐ป๐ถ๐ป๐ด ๐๐ต๐ฒ ๐ฝ๐ฟ๐ฒ๐๐ฒ๐ป๐๐ถ๐ผ๐ป ๐ผ๐ณ ๐ฐ๐ผ๐บ๐บ๐ผ๐ป ๐ฑ๐ถ๐๐ฒ๐ฎ๐๐ฒ.
One week ago, Scribe reached an important milestone on the path toward realizing our vision: becoming a publicly traded company. We are thrilled to begin this next chapter, which will accelerate our efforts to rewrite what's possible in the treatment and prevention of ASCVD.
I want to express a huge amount of gratitude to those that have supported Scribe and believed in our long term mission: our board, investors, partners, collaborators, and most importantly our families. It was truly a delight to have so many of you there to celebrate with us at the @NasdaqExchange.
๐ ๐ผ๐๐ ๐ผ๐ณ ๐ฎ๐น๐น, ๐ ๐๐ฎ๐ป๐ ๐๐ผ ๐๐ต๐ฎ๐ป๐ธ ๐๐ต๐ฒ ๐ฆ๐ฐ๐ฟ๐ถ๐ฏ๐ฒ ๐ง๐ฟ๐ถ๐ฏ๐ฒ. ๐ฌ๐ผ๐๐ฟ ๐ฐ๐ฟ๐ฒ๐ฎ๐๐ถ๐๐ถ๐๐, ๐ด๐ฟ๐ถ๐, ๐ฑ๐ถ๐๐ฐ๐ถ๐ฝ๐น๐ถ๐ป๐ฒ, ๐ฝ๐ฒ๐ฟ๐๐ฒ๐๐ฒ๐ฟ๐ฎ๐ป๐ฐ๐ฒ, ๐ฎ๐ป๐ฑ ๐ฏ๐ฒ๐น๐ถ๐ฒ๐ณ ๐ฎ๐ฟ๐ฒ ๐๐ต๐ฎ๐ ๐ต๐ฎ๐ ๐บ๐ฎ๐ฑ๐ฒ ๐๐ต๐ถ๐ ๐บ๐ผ๐บ๐ฒ๐ป๐ ๐ฝ๐ผ๐๐๐ถ๐ฏ๐น๐ฒ. Onwards! $SCTX
.@Scribetx is engineering purpose-built CRISPR medicines to broaden access to cardioprotective genetics for us all. Look back on $SCTXโs milestone celebration becoming #NasdaqListed and delivering a new era of scalable, transformative, and preventative genetic medicines.
CRISPR genetic medicines are often described as more โconvenientโ than chronic treatments like pills or injections. ๐ ๐๐ต๐ถ๐ป๐ธ ๐๐ต๐ถ๐ ๐บ๐ถ๐๐๐ฒ๐ ๐๐ต๐ฒ ๐ฝ๐ผ๐ถ๐ป๐ ๐ฒ๐ป๐๐ถ๐ฟ๐ฒ๐น๐.
๐๐ ๐ถ๐ ๐น๐ถ๐ธ๐ฒ ๐๐ฎ๐๐ถ๐ป๐ด ๐ณ๐น๐๐ถ๐ป๐ด ๐ฎ๐ฐ๐ฟ๐ผ๐๐ ๐๐ต๐ฒ ๐ฐ๐ผ๐๐ป๐๐ฟ๐ ๐ถ๐ ๐บ๐ผ๐ฟ๐ฒ ๐ฐ๐ผ๐ป๐๐ฒ๐ป๐ถ๐ฒ๐ป๐ ๐๐ต๐ฎ๐ป ๐๐ฎ๐น๐ธ๐ถ๐ป๐ด. ๐ง๐ฒ๐ฐ๐ต๐ป๐ถ๐ฐ๐ฎ๐น๐น๐ ๐๐ฟ๐๐ฒ, ๐ฏ๐๐ ๐๐ถ๐น๐ฑ๐น๐ ๐ถ๐ป๐ฐ๐ผ๐บ๐ฝ๐น๐ฒ๐๐ฒ. One is not just an easier version of the other. Flying makes the journey possible for everyone, while only a select few could ever hope to complete a cross-country trek.
We see this same dynamic play out again and again in cardiovascular disease. The current standard of care asks patients to commit to chronic therapy for decades: daily pills, repeat injections, and continued persistence fails for most. ๐๐ป ๐ฟ๐ฒ๐ฐ๐ฒ๐ป๐ ๐๐๐๐ฑ๐ถ๐ฒ๐, ๐บ๐ผ๐ฟ๐ฒ ๐๐ต๐ฎ๐ป ๐ณ๐ฌ% ๐ผ๐ณ ๐ฝ๐ฎ๐๐ถ๐ฒ๐ป๐๐ ๐๐ฒ๐ฟ๐ฒ ๐ป๐ผ ๐น๐ผ๐ป๐ด๐ฒ๐ฟ ๐๐ฎ๐ธ๐ถ๐ป๐ด ๐๐ต๐ฒ๐ถ๐ฟ ๐๐ต๐ฒ๐ฟ๐ฎ๐ฝ๐ถ๐ฒ๐ ๐ฎ๐ณ๐๐ฒ๐ฟ ๐ผ๐ป๐น๐ ๐ฎ ๐ณ๐ฒ๐ ๐๐ฒ๐ฎ๐ฟ๐.
Against that backdrop, describing durable genetic medicines as simply more โconvenientโ does no one justice. The current chronic medication paradigm, our โsick-careโ framework, simply does not work well enough for silent lifelong diseases such as CVD.
๐ช๐ฒ ๐บ๐๐๐ ๐ฏ๐๐ถ๐น๐ฑ ๐๐ผ๐บ๐ฒ๐๐ต๐ถ๐ป๐ด ๐ฏ๐ฒ๐๐๐ฒ๐ฟ. The goal is not just fewer doses. The goal is a model of prevention that is practical for all and matched to how lifelong risk actually works.
๐ช๐ฒ ๐๐ต๐ผ๐๐น๐ฑ ๐ป๐ผ๐ ๐๐ฒ๐๐๐น๐ฒ ๐ณ๐ผ๐ฟ ๐บ๐ฎ๐ธ๐ถ๐ป๐ด ๐๐ต๐ฒ ๐๐ฎ๐น๐ธ ๐ฒ๐ฎ๐๐ถ๐ฒ๐ฟ. ๐ช๐ฒ ๐บ๐๐๐ ๐ฏ๐๐ถ๐น๐ฑ ๐๐ต๐ฒ ๐ฎ๐ถ๐ฟ๐ฝ๐น๐ฎ๐ป๐ฒ.
I am incredibly proud that the California Institute for Regenerative Medicine is helping us make that vision a reality. Scribe recently announced that we have been awarded more than $25 million from CIRM to help advance STX-1200 for Lp(a) and STX-1400 for severely elevated triglycerides toward the clinic.
This funding helps us move these programs from possibility to patients.
๐๐ผ๐ป๐ด๐ฟ๐ฎ๐๐๐น๐ฎ๐๐ถ๐ผ๐ป๐ ๐๐ผ ๐๐ต๐ฒ ๐ฆ๐ฐ๐ฟ๐ถ๐ฏ๐ฒ ๐๐ฒ๐ฎ๐บ, ๐ฎ๐ป๐ฑ ๐๐ต๐ฎ๐ป๐ธ ๐๐ผ๐ ๐๐ผ ๐๐๐ฅ๐ ๐ณ๐ผ๐ฟ ๐๐๐ฝ๐ฝ๐ผ๐ฟ๐๐ถ๐ป๐ด ๐ผ๐๐ฟ ๐บ๐ถ๐๐๐ถ๐ผ๐ป ๐๐ผ ๐ฏ๐ฟ๐ถ๐ป๐ด ๐ฑ๐๐ฟ๐ฎ๐ฏ๐น๐ฒ ๐ด๐ฒ๐ป๐ฒ๐๐ถ๐ฐ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ๐ ๐๐ผ ๐ฝ๐ฎ๐๐ถ๐ฒ๐ป๐๐ ๐๐ถ๐๐ต ๐ฐ๐ฎ๐ฟ๐ฑ๐ถ๐ผ๐บ๐ฒ๐๐ฎ๐ฏ๐ผ๐น๐ถ๐ฐ ๐ฑ๐ถ๐๐ฒ๐ฎ๐๐ฒ.
Weโre excited to share that @CIRMnews has awarded Scribe more than $25 million through its Preclinical Development Program to accelerate the advancement of two preclinical programs targeting cardiovascular and metabolic disease.
Press release in thread ๐ฃ
Thanks to @mrdannysullivan for the conversation highlighting the critical question: can genetic medicine help us better treat and prevent ASCVD?
Lipid levels are genetically driven for many people, and while todayโs treatments work, they rely on lifelong, burdensome adherence.
I believe, next-gen CRISPR and epigenetic technologies may finally offer durable protection that gives patients real agency and ownership over their health.
Appreciate the chance to talk about how weโre working to make that future real @scribetx .
Scribe Therapeutics CEO on how CRISPR technologies are ready to move beyond rare diseases and take on the worldโs biggest killers.
longevity.technology/news/caโฆ
#longevity #CRISPR #geneticmedicine #CVD #heartdisease
We've spent billions on treatments that patients can't stick to. ๐๐ป๐ฑ ๐๐๐ถ๐น๐น, ๐ฒ๐๐ฒ๐ฟ๐ ๐ฏ๐ฐ ๐๐ฒ๐ฐ๐ผ๐ป๐ฑ๐, ๐๐ผ๐บ๐ฒ๐ผ๐ป๐ฒ ๐ฑ๐ถ๐ฒ๐ ๐ณ๐ฟ๐ผ๐บ ๐ฐ๐ฎ๐ฟ๐ฑ๐ถ๐ผ๐๐ฎ๐๐ฐ๐๐น๐ฎ๐ฟ ๐ฑ๐ถ๐๐ฒ๐ฎ๐๐ฒ. That's more than cancer, accidents, and diabetes combined. Weโve been solving the wrong problem entirely.
What if we could prevent it from happening in the first place?
The industry is making tons of progress on the ๐๐ต๐ฟ๐ฒ๐ฒ ๐ธ๐ฒ๐ ๐น๐ถ๐ฝ๐ถ๐ฑ ๐ฑ๐ฟ๐ถ๐๐ฒ๐ฟ๐ ๐ผ๐ณ ๐ฎ๐๐ต๐ฒ๐ฟ๐ผ๐๐ฐ๐น๐ฒ๐ฟ๐ผ๐๐ถ๐: ๐๐๐-๐, ๐๐ฝ(๐ฎ), ๐ฎ๐ป๐ฑ ๐๐ฟ๐ถ๐ด๐น๐๐ฐ๐ฒ๐ฟ๐ถ๐ฑ๐ฒ-๐ฟ๐ถ๐ฐ๐ต ๐น๐ถ๐ฝ๐ผ๐ฝ๐ฟ๐ผ๐๐ฒ๐ถ๐ป๐ (๐ง๐ฅ๐๐). PCSK9 inhibitors for LDL-C lowering are now, finally, frontline therapies. Lp(a) CVOTs are hotly awaited, and triglyceride-lowering approaches have just been approved with promising results in FCS and SHGT.
But here's the catch.
For these approaches to workโthey require patients to take their medications regularly. ๐๐ผ๐ฟ ๐ฑ๐ฒ๐ฐ๐ฎ๐ฑ๐ฒ๐.
And in the real world? Thatโs not always possible, or even probable.
This adherence problem won't be solved by more drugs that still need to be taken for a lifetime.
We donโt need better or more versions of treatments that still face that problem.
๐ช๐ฒ ๐ป๐ฒ๐ฒ๐ฑ ๐๐ผ ๐๐ต๐ถ๐ป๐ธ ๐ฑ๐ถ๐ณ๐ณ๐ฒ๐ฟ๐ฒ๐ป๐๐น๐.
That's why at Scribe Therapeutics, we've been ๐ฏ๐๐ถ๐น๐ฑ๐ถ๐ป๐ด ๐ฎ ๐ฐ๐ผ๐บ๐ฝ๐ฟ๐ฒ๐ต๐ฒ๐ป๐๐ถ๐๐ฒ ๐ฝ๐ถ๐ฝ๐ฒ๐น๐ถ๐ป๐ฒ ๐๐ฎ๐ฟ๐ด๐ฒ๐๐ถ๐ป๐ด ๐ฎ๐น๐น ๐๐ต๐ฟ๐ฒ๐ฒ ๐ฐ๐ฎ๐๐๐ฎ๐น ๐ฑ๐ฟ๐ถ๐๐ฒ๐ฟ๐ ๐ฎ๐ ๐๐ต๐ฒ ๐ต๐ฒ๐ฎ๐ฟ๐ ๐ผ๐ณ ๐๐ฆ๐๐ฉ๐: LDL-C, LP(a), & TRL.
Engineered to be safe, durable, and transformative.
๐๐'๐ ๐ฎ๐ฏ๐ผ๐๐ ๐บ๐ฎ๐ธ๐ถ๐ป๐ด ๐๐ฅ๐๐ฆ๐ฃ๐ฅ ๐ด๐ฒ๐ป๐ฒ๐๐ถ๐ฐ ๐บ๐ฒ๐ฑ๐ถ๐ฐ๐ถ๐ป๐ฒ ๐๐ฎ๐ณ๐ฒ ๐ฒ๐ป๐ผ๐๐ด๐ต ๐๐ผ ๐ฝ๐ฟ๐ฒ๐๐ฒ๐ป๐ ๐ฐ๐ฎ๐ฟ๐ฑ๐ถ๐ผ๐๐ฎ๐๐ฐ๐๐น๐ฎ๐ฟ ๐ฑ๐ถ๐๐ฒ๐ฎ๐๐ฒ.
Last week at the European Society of Cardiology Congress the Scribe team proudly presented new data on STX-1200. Our XE-based in vivo CRISPR therapy designed to safely and durably lower Lp(a). This data, with >90% lowering and no observable off-targets, is representative of what our engineered CRISPR approaches can achieve when applied to one of cardiovascular medicine's toughest targets.
One treatment instead of decades upon decades of constant medication.
That's what intentional and precise molecular engineering can deliver for the millions of us at risk of cardiovascular disease.
Big thanks to @blalpert & @mackenziettnni for sitting down with us to hear more about why the future of cardiometabolic disease ๐ซ is looking bright๐ก
At Scribe, our aim is to build medicines that are safe, effective, and durable enough to be both highly preventative and broadly accessible for people at risk of ASCVD.
Our goal is to liberate patients and high-risk individuals, like myself, from the burden of daily pills or lifelong injections. Based on our data, this future is real. It is not decades away. It is within reach.
If you're interested in learning more, check out our recent data shared at ASGCT and EAS. More to come soon, so stay tuned.
In this feature, @barronsonline highlights that โgenetic-medicine approaches to PCSK9 are exploring nonpermanent, but long-lasting fixes ... Scribe Therapeutics has reported intriguing results in monkeys from โepigeneticโ edits of the molecules that turn genes on and off.โ
Over the past few weeks, we've had the privilege of sharing our groundbreaking results, highlighting the cutting-edge advancements of our engineered CRISPR technologies across multiple areas.
Most notably, we've demonstrated that the durability of our epigenetic approach to reduce LDL-C will set a new standard for therapeutic durability in ASCVD without any permanent genetic sequence modification. The future of medicine is bright. ๐ก
Learn more below:
This month, we presented positive data on our #CRISPR technologies at the 2025 @EASCongress and @ASGCTherapy Annual Meeting.
For more details, read our thread and press release below ๐
At Scribe, the hits just keep on coming. ๐ฌ๐๐ซ
Check it out: Tomorrow at the 93rd European Atherosclerosis Society (EAS) Congress, our CSO Dr. Aarif Khakoo will present Scribe's breakthrough preclinical data from our lead epigenetic silencing program, STX-1150. ๐ข
Built on our ELXR platform, we show that STX-1150 can deliver >50% reduction in LDL-C levels in non-human primates, with durability that far exceeds todayโs RNAi standard of care โ all without permanently editing the genome. ๐งฌโ
Be sure to attend the session for more details!
We believe this is the first and only epigenetic silencing system engineered to be safe and potent enough for therapeutic use in vivo across large patient populations. Itโs programmable, precise, and designed for lasting impact in cardiometabolic disease. โค๏ธโ๐ฅ
Catch the data in the Late Breaker Session with more in our press release below. ๐๐
Join us at #EASCongress2025 where our CSO Aarif Khakoo will deliver a late-breaking presentation with data on our #CRISPR epigenetic silencing technology ELXR for LDL-C lowering.
To learn about our advances developing ELXR for cardiometabolic disease at @EASCongress, read on ๐
Today I am proud to share important progress from @scribetx in collaboration with @EliLillyandCo in advancing CRISPR-based therapies and delivery systems for neurological diseases.
๐ ๏ธ Reaching this milestone, and our upcoming presentation at #ASGCT2025, underscores the strength of Scribeโs CRISPR engineering engines. Drug development is never easy, but throughout this work our team has demonstrated the ability to consistently solve complex problems with molecular engineering. This achievement marks an important step toward delivering safer and more effective in vivo genetic medicines for neurodegenerative diseases, a goal we are determined to achieve.
๐งฌ At the @ASGCTherapy meeting, we'll unveil initial data on our novel self-inactivating AAV technology, developed through this collaboration. Full details are available in our press release below.
***
P.S. If this announcement sounds familiar, it should. Earlier this year, we reached a separate milestone in another in vivo genetic medicine program with @sanofi. While the broader biotech markets remain turbulent, Scribeโs momentum is building, the science has never been stronger, and we are just getting started. Stay tuned. ๐
Weโre pleased to announce the achievement of a success milestone in our collaboration with Prevail Therapeutics, a part of @EliLillyandCoโs Gene Therapy Division, to develop in vivo #CRISPR-based therapeutics for neurological and neuromuscular disorders.
Highlights below ๐งต
As I mentioned a few days ago, the Senate just released a report on U.S. biotech competitiveness. It makes one thing very clear:
๐งฌ Biotech is strategic infrastructure. The global healthcare market is estimated at $21Tโthree times the size of the global energy market. Let that sink in.
This is fundamentally important to the future of America and while the U.S. still holds a lead in biotech, we have only a short window to act before others catch up or pull ahead.
Hereโs a snapshot of the senate recommendations ๐งพ (in brief):
๐๏ธ Create a unified national biotech strategy
๐ต Expand NIH, NSF, and DoD funding for biotech
๐งช Modernize FDA/USDA/EPA to enable faster, risk-based regulation
๐งฑ Invest in domestic biomanufacturing and resilient supply chains
๐ Train a specialized biotech workforce
๐ฐ๏ธ Integrate biotech into the national defense strategy
All very sensible ideas ๐ง โbut perhaps a bit vague. Read the report for more detail.
Over the next few days/weeks, as time allows, Iโll share a few of my own ideas.
In brief: Iโm focused on expanding the American lead in biotech by doubling investment across research areas, streamlining regulation for speed, and 10x-ing U.S. engagement outside of major biotech hubs. ๐งต๐
๐จ Can we stop the offshoring of U.S. biotech?
A Senate report dropped today with a clear warning: U.S. biotech is at risk of falling behind as Chinaโs state-backed sector rapidly accelerates. While thereโs a lot of analysis in the report I see one major trend it misses:
๐ U.S. healthcare dollars are increasingly subsidizing Chinese biotech innovation.๐
Confusing? Letโs break that down:
In recent years teams like Merck, Novo, and Roche have spent billions of $ licensing Chinese drug assets. Take Merck for example, in the past ~8 months, it made 4 deals with Chinese biotechs totaling:
~$1.6B upfront
~$8.5B in milestones and royalties
๐ฆ All flowing into China๐ธ
Now, only ~7% of Merckโs profits come from China while over 50% come from the U.S. healthcare system ๐บ๐ธ. So yes โ U.S. healthcare spending is now directly funding Chinese R&D.
We pay the highest drug prices in the world ๐ฐ. That system is meant and required to fund global medical innovation ๐. But now? A significant portion of that subsidy is fueling Chinaโs R&D ecosystem โ not reinvesting in U.S. or EU innovation. And itโs not just Merck.
In 2024, over one-third of all in-licensed drug assets are coming from Chinese companies โ many of them me-too/fast-follower drugs, based on American research, patents, and innovation just executed cheaper and faster, with significantly fewer regulatory hurdles and lower labor/operating costs ๐ญ.
Last year alone saw:
$6B+ in upfronts to Chinese firms
Tens to hundreds of billions in potential milestones
Thatโs capital that used to go toward supporting domestic or allied innovation. Now even U.S. VCs are backing Chinese knockoffs and Iโve heard the logic directly: โWhy fund U.S. biotech when I can get a Chinese copycat asset for half the cost?โ
These deals may look like short-term wins โ but, at best, theyโre Pyrrhic ๐ฅ victories. We risk gutting the very innovation engine โ๏ธ that powers not just the U.S. biotech industry, but global progress in medicine as a whole ๐
The report is right to sound the alarm๐จ. Chinaโs biotech sector is fast, state-backed, and cost-effective. This looks a lot like semiconductors in the 1980s and 90s. Weโre at risk of watching the offshoring of biotech in real time ๐ข
American biotech is under threat. Time to act like it.
I'll cover some potential solutions in the report as well as my take on them in the coming days but here is the @biotech_gov report as a start: biotech.senate.gov/final-repโฆ
๐ฅ Fund, Baby, Fund!
A recent report shows NIH delivers an approximate 250% ROI per dollar. Yet in real, inflation-adjusted terms, NIH funding has been flat or declining for 25 years. That doesnโt preserve our lead โ it erodes it, bad. The first solution must be: Fund, Baby, Fund.
๐น NIH
Slowly grow and double NIH funding over the next 5โ10 years. Yes, rein in and flatten university overhead across the board, but reinvest those savings directly into new research via larger grants. Build additional structures to support longer-term ideas, push the boundaries of โnormal science,โ and allow more flexibility in grant design. Focus on funding incredible people, not just projects. (Much of the research that led to Scribe was supported by such a grant.) If our biotech edge is one of the few strategic assets the U.S. has left, we need to double down and surge ahead.
๐น NSF / DOE / USDA
Apply the same doubling principle here, but with a focus on basic biological researchโnot just medical. Fund work that pushes biologists to think like both engineers and explorers. CRISPR came from decades of curiosity-driven research into bacterial immune systems. Imagine what AI could do with a U.S. biobank designed to sequence examples of every genome and transcriptome on Earth.
๐ฅ Large Healthcare Systems
Encourage the VA and Medicare to become platforms for cutting-edge therapeutics and preventive medicine: such as using CRISPR to prevent common diseases. Preventive innovation will save health, lives and money, ๐ธ which can then be reinvested into future biotech breakthroughs. Letโs replace ever growing healthcare services๐ฉบ with smart, scalable science ๐.
๐ 10x Small Business Innovation
Iโve personally declined U.S. SBIR grants because they were too slow and too small to matter. I support @AppleHelix 's SBIR proposal, here it is again, with a few tweaks:
Letโs 10x the program from ~$200M/year to $2B/year.
For Bio startups specifically, jump Phase 1's up to $1-2M to provide the actual startup capital needed to perform experiments as a new spinout. This is often first money in the door. (do you know how expensive plastics are!?)
Next, a more stringent Phase 2 award needs to be $10M+ to support the CMC and quality work required to meet FDA standards especially in a newer modality (which we want to encourage to stay in the lead!).
Last, make submissions quarterly, with reviews and awards within 6 months. Good ideas can't wait for cycles. With this, SBIRs/STTRs could become rocket fuel for deeptech rather than the bureaucratic side quest they are today.
๐ Cut Out the Financial Parasites
Too many systems siphon off public research dollars, from excessive university bureaucracy to overpriced consumables. But the worst offenders? Academic publishers. These organizations operate under a model so broken it's, quite frankly, impressive: First, the government funds the research, pays the salaries of peer reviewers, pays to publish the paper, and then it has to pay again for access to the final publication. All government-funded research should be made immediately and permanently accessible across all journals.
Next week, Iโll dive into the rest of my ideas such as a draft model for national bio hubs to 10x engagement across the U.S., reforming regulation, and unlocking talent. Stay tuned.๐
๐จ Can we stop the offshoring of U.S. biotech?
A Senate report dropped today with a clear warning: U.S. biotech is at risk of falling behind as Chinaโs state-backed sector rapidly accelerates. While thereโs a lot of analysis in the report I see one major trend it misses:
๐ U.S. healthcare dollars are increasingly subsidizing Chinese biotech innovation.๐
Confusing? Letโs break that down:
In recent years teams like Merck, Novo, and Roche have spent billions of $ licensing Chinese drug assets. Take Merck for example, in the past ~8 months, it made 4 deals with Chinese biotechs totaling:
~$1.6B upfront
~$8.5B in milestones and royalties
๐ฆ All flowing into China๐ธ
Now, only ~7% of Merckโs profits come from China while over 50% come from the U.S. healthcare system ๐บ๐ธ. So yes โ U.S. healthcare spending is now directly funding Chinese R&D.
We pay the highest drug prices in the world ๐ฐ. That system is meant and required to fund global medical innovation ๐. But now? A significant portion of that subsidy is fueling Chinaโs R&D ecosystem โ not reinvesting in U.S. or EU innovation. And itโs not just Merck.
In 2024, over one-third of all in-licensed drug assets are coming from Chinese companies โ many of them me-too/fast-follower drugs, based on American research, patents, and innovation just executed cheaper and faster, with significantly fewer regulatory hurdles and lower labor/operating costs ๐ญ.
Last year alone saw:
$6B+ in upfronts to Chinese firms
Tens to hundreds of billions in potential milestones
Thatโs capital that used to go toward supporting domestic or allied innovation. Now even U.S. VCs are backing Chinese knockoffs and Iโve heard the logic directly: โWhy fund U.S. biotech when I can get a Chinese copycat asset for half the cost?โ
These deals may look like short-term wins โ but, at best, theyโre Pyrrhic ๐ฅ victories. We risk gutting the very innovation engine โ๏ธ that powers not just the U.S. biotech industry, but global progress in medicine as a whole ๐
The report is right to sound the alarm๐จ. Chinaโs biotech sector is fast, state-backed, and cost-effective. This looks a lot like semiconductors in the 1980s and 90s. Weโre at risk of watching the offshoring of biotech in real time ๐ข
American biotech is under threat. Time to act like it.
I'll cover some potential solutions in the report as well as my take on them in the coming days but here is the @biotech_gov report as a start: biotech.senate.gov/final-repโฆ
If you know me, youโve probably heard this before โ but itโs worth repeating
The future of medicine is bright๐ก. And itโs going to be radically different from today. It will be:
๐งฌ Preventive
Weโll use genetic insights to erase major disease risks before they happen.
Imagine taking the DNA of your healthiest 100-year-old relative โ the one who never had heart disease or cancer โ and making it yours. Thatโs the promise of genomic medicine: everyone gets to win the genetic lottery.
โก Liberating
Treatments wonโt be daily pills or ongoing injections.
Youโll walk into a clinic, get a highly durable treatment, and walk out fundamentally healthier. Transformative care with no upkeep required.
๐ Universal
Safe enough to be standard. Broad enough to prevent disease across the board. Weโre talking about stopping ASCVD, stroke, diabetes, kidney disease โ all as part of routine care.
This future is real. Itโs not even decades away, itโs within reach and building this future is what weโre doing at Scribe.
So yeah, Iโm likely to keep saying it:
Get excited. ๐ Spread the word. Support genetic medicine and help shape what comes next. The future of health is already on its way.
This #WorldHealthDay, join us in building a future of a healthier life for all.
At Scribe, we're engineering #CRISPR-based therapies designed to transform everyone's lifetime risk for disease.
Learn more about causes for World Health Day from @WHO: who.int/campaigns/world-healโฆ
This week, Iโll highlight two significant recent CRISPR advances: 1) BEAMโs exciting Phase 1 clinical data for BEAM-302. 2) Impressive work from the labs of Scribeโs co-founders Dave Savage & Jennifer Doudnaโproviding a textbook example on engineering CRISPR enzymes from zero to hero.
1/ ๐งฌ In the near future, it may be the we can all win the genetic lottery for healthโnot by chance, but by design. At Scribe, weโre focused on making genetic medicines safe enough for disease prevention at scale and moving beyond gene editing into epigenetic editing.
#CRISPR can change the worldโwithout editing your DNA. A new @TheEconomist report highlights epigenetic editing as a transformative technology and our work at Scribe engineering epigenetic editors (ELXRs) as genetic medicines potent and safe enough for broad patient populations.
2/ ๐ฐ It was great speaking with Ida Emilie Steinmark for The Economistโs The Age of CRISPR. The piece highlights how Scribe's epigenetic editors could give everyone access to protective genetic traits and dramatically reduce ASCVD risk. economist.com/technology-quaโฆ
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1
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As someone with close family affected by ASCVDโand therefore at high risk myselfโthis mission is personal, urgent and real. We will solve this.
Explore our vision for the future of ASCVD treatmentโand, more importantly, preventionโbelow.
Did you know that elevated LDL-C can increase risk for cardiovascular disease, the leading cause of death worldwide?
As we observe #HeartMonth, weโre reminded of the importance of heart health and the ongoing fight against cardiometabolic disease โฌ๏ธ