The goal is to develop therapeutics that are broadly effective and safe enough for all. But financial access is complex—most payor systems don’t know how to reward long-term health. We’ll need to push the system to redefine value as not just treating illness, but preventing it before it starts.

This future is real. It’s not even decades away, it’s within reach.

🔬Building this future is what we’re doing at Scribe.🔬

So yeah, I’m likely to keep saying it:

Get excited. 🚀 Spread the word. Support genetic medicine and help shape what comes next. The future of health is already on its way.

🌍 Universal
Safe enough to be standard. Broad enough to prevent disease across the board. We’re talking about stopping ASCVD, stroke, diabetes, kidney disease — all as part of routine care.

⚡ Liberating
Treatments won’t be daily pills or ongoing injections.
You’ll walk into a clinic, get a highly durable treatment, and walk out fundamentally healthier. Transformative care with no upkeep required.

🧬 Preventive
We’ll use genetic insights to eliminate disease before it starts.
Imagine taking the DNA of your healthiest 100-year-old relative — the one who never had heart disease or cancer — and making it yours. That’s the promise of genomic medicine: everyone gets to win the genetic lottery.

Check out this 🔥 work here! www.biorxiv.org/content/10.1...

Why I like it: This study provides a clear playbook for modern molecular engineering. Comparing this work with others in the field, it is clear that comprehensive variant mapping surpasses the effectiveness of targeted mutagenesis and assay-naïve AI engineering approaches. Plus it's just more fun!

This work elegantly demonstrates how systematic variant scanning, and combinatorial assembly, can supercharge CRISPR and is similar to engineering approaches we utilize @scribetx.bsky.social for rapidly building powerful new CRISPR tools. Check it out: www.scribetx.com/platform

This meticulous screening reveals specific mutations that greatly enhance TnpB’s nuclease activity. By stacking beneficial single mutations into one optimized variant (“enhanced TnpB”), they achieved up to 50-fold higher activity in plants—a reasonably respectable 🤯 leap in enzyme efficiency.

Quick background: TnpB is a compact RNA-guided nuclease and the evolutionary ancestor of Cas12. In this work, the team uses deep mutational scanning with a yeast-based cleavage assay to profile every RNA & protein variation.

Next, the preprint “Latent activity in TnpB revealed by mutational scanning” @brittneywthornton.bsky.social & @rfw.bsky.social et al., coming out of the Doudna & Savage labs. This work beautifully illustrates how holistic molecular engineering systematically improves CRISPR molecules

Importance: These data highlight CRISPR’s immense potential for in vivo cures. Yet, as base editors may create unintended edits, the exact correction efficiency in this trial is uncertain. PRIME Medicine recently entering this disease area with RT-based editors may offer additional precision.

At the highest dose, BEAM-302 boosted total AAT from 4.4 µM to 12.4 µM, surpassing the perceived therapeutic threshold of 11 µM. Importantly, the treatment appeared well-tolerated and is promising as correction strategy for AATD patients in need. Check it out: www.globenewswire.com/news-release...

First, let’s dive into BEAM’s results: this base-editing therapy uses a Cas9-deaminase fusion to target and mutate the PiZ mutation causing alpha-1 antitrypsin deficiency (AATD). Patients received doses of 15, 30, & 60 mg to assess in vivo editing efficacy.

💡 Want to see how these breakthroughs will help to shape the future of medicine? Don’t miss Jason Fernandes’ presentation at Keystone: Precision Genome Engineering: Translating the Human Genome to the Clinic, Tomorrow, March 4, 3:00-4:30 p.m. GMT! #KSGenomeEng25

🎤 In fact, this week at Keystone Symposia, we’re unveiling major advancements in epigenetic therapies. Our engineering engines have built thousands of possibilities, uncovering stronger, safer, and more precise therapeutics.

📰 It was great speaking with Ida Emilie Steinmark for The Economist’s 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. www.economist.com/technology-q...