Scribe Therapeutics Achieves Second Success Milestone for In Vivo Program in Collaboration with Eli Lilly and Company

Key Terms

in vivo medical

In vivo describes tests or experiments performed inside a living organism, such as an animal or human, to observe how a drug, device or biological process behaves in a real, functioning body. Investors care because in vivo results reveal safety, effectiveness and possible side effects that lab tests cannot, much like road-testing a prototype car in traffic rather than only on a bench — outcomes can strongly influence regulatory approval, clinical success and a company’s valuation.

CRISPR medical

CRISPR is a gene‑editing technology that works like precise molecular scissors to change DNA in living cells. For investors, it matters because it can speed development of new therapies, lower research costs, and create valuable intellectual property, but it also carries scientific, regulatory, ethical, and commercial risks that can strongly affect the value and prospects of companies working with the technology.

neuromuscular medical

Relating to the connection and interaction between nerves and muscles, neuromuscular describes how signals from the nervous system make muscles move and function. For investors, neuromuscular issues and treatments matter because they drive demand for drugs, devices and diagnostic tests, shape development timelines and regulatory risks, and can change long-term revenue prospects in healthcare—think of it like the wiring and motors in a machine where problems or fixes affect overall performance and value.

genetic medicines medical

Genetic medicines are treatments that work by changing, replacing, or reprogramming a person’s genes or the instructions those genes send, with the goal of preventing or curing disease rather than just easing symptoms. For investors, they matter because they can offer transformative, often one-time therapies that command high prices and durable outcomes, but they also carry long development timelines, regulatory scrutiny and complex manufacturing risks — like backing a high-reward, high-risk technology startup.

milestone payments financial

Milestone payments are predetermined sums a company agrees to pay or receive when specific development, regulatory, or commercial goals are reached in a partnership or license deal. Think of them like progress bonuses: they turn uncertain future outcomes into conditional cash events, so investors track them as potential sources of revenue, value inflection points, and risk—payments only arrive if the agreed milestones are actually achieved.

royalties financial

Payments made to the owner of an asset or intellectual property each time that asset is used or a product is sold, often calculated as a percentage of sales or a set amount per unit. Royalties matter to investors because they create predictable, ongoing income streams and affect a company’s cash flow and valuation—like a landlord collecting rent or an author getting a steady cut whenever a book is sold.

pipeline technical

A pipeline is the organized list of products, drugs or projects a company is developing but has not yet fully launched or commercialized. For investors it acts like a forecasted inventory of future revenue sources — showing what could become earnings, how far along each item is, and the risk and time before they might pay off; think of it as a company’s product roadmap that helps gauge growth potential and uncertainty.

cardiovascular medical

Relating to the heart and the network of blood vessels that pump and carry blood through the body — like the body’s engine and plumbing working together. Investors pay attention because conditions, treatments, devices, and drugs tied to this system drive large health care spending, regulatory reviews, and market demand; breakthroughs, safety issues, or trial results can quickly change a company’s revenue prospects and stock value.

• Milestone achieved in Scribe and Lilly’s ongoing collaboration to develop in vivo CRISPR-based therapeutics for neurological and neuromuscular diseases
• Achievement validates Scribe’s technologies and strengthens momentum across its genetic medicine portfolio, including its internal pipeline advances for cardiovascular and metabolic disease

ALAMEDA, Calif.--(BUSINESS WIRE)-- Scribe Therapeutics, Inc. (Scribe), a biotechnology company pioneering highly engineered CRISPR technologies designed to reshape the treatment of disease by enabling earlier intervention, improved outcomes, and longer, healthier lives, today announced the achievement of a second success milestone for one of the targets in its collaboration with Eli Lilly and Company (NYSE: LLY). The companies are working towards developing in vivo CRISPR-based genetic medicines for neurological and neuromuscular disorders.

This latest milestone is a testament to the companies’ ongoing progress in accelerating the development of next-generation therapies by using Scribe’s proprietary X-Editor (XE) technology and is the second such event in Scribe and Lilly’s collaboration. The achievement further validates CRISPR by Design™, Scribe’s holistic, data-driven, and iterative engineering approach to deliberately optimize CRISPR-based medicines and apply them to specified targets that drive prevalent, high-burden diseases with significant unmet need.

“This milestone reflects strong execution and continued validation of our in vivo CRISPR technologies by a partner with deep technical and clinical rigor,” said Benjamin Oakes, Ph.D., co-founder and Chief Executive Officer of Scribe Therapeutics. “Advancing this program with Lilly reinforces our conviction that purpose-built CRISPR technologies can clear the potency and specificity thresholds required for transformative therapies in neurological and neuromuscular diseases. In parallel, we are deploying the same engineering engine across our wholly owned cardiovascular and metabolic pipeline, where we see a significant opportunity to build durable, differentiated medicines for large, high-impact patient populations.”

Previously, the companies signaled progress in their collaboration in the form of an initial milestone achievement and a joint scientific presentation at ASGCT in 2025. Under the terms of the original agreement announced in 2023, Scribe is eligible to receive more than $1.5 billion across all programs in milestone payments for the achievement of certain research, development, regulatory, and commercial milestones, as well as low-double-digit royalties.

“With the achievement of this second success milestone, Scribe is further advancing a productive collaboration with Lilly,” said Svetlana Lucas, Ph.D., Chief Business Officer at Scribe. “We are leveraging the complementary strengths of our teams and a shared commitment to innovation in genetic medicine to improve therapeutic options for patients.”

About Scribe’s X-Editor (XE)

XE is Scribe’s novel CRISPR-based gene editing technology designed for precise and versatile genetic modification. It uses a staggered DNA cleavage mechanism to achieve high on-target activity, specificity, and flexibility across a range of applications, including gene knock-out, knock-down, knock-in, exon skipping, genetic excision, and other targeted modifications. Scribe has engineered its novel CasX enzyme, XE, with improved nuclease stability, DNA binding, cleavage activity, and specificity, resulting in greater than 100-fold higher editing than naturally occurring CasX in cell-based assays while maintaining exquisite specificity across target sites. XE’s compact size and engineered PAM recognition together provide a differentiated combination of potency, specificity, delivery, flexibility, and a broad therapeutic window, providing the potential for durable, one-time treatments for genetically defined diseases.

About Scribe Therapeutics

Scribe Therapeutics is a biotechnology company developing optimized in vivo CRISPR-based technologies and genetic medicines designed to become standard of care treatments for patients suffering from highly prevalent diseases, starting with cardiometabolic disease. Leveraging its CRISPR by Design™ approach and nature’s blueprint for improved cardiovascular health, Scribe’s initial programs focus on addressing the key drivers of ASCVD such as elevated LDL-C, lipoprotein(a), and triglycerides. The company’s lead candidate, STX-1150, is a novel liver-targeted therapy designed to epigenetically silence the PCSK9 gene and reduce LDL-C levels without inducing permanent DNA changes. Scribe expects to enter the clinic in mid-2026 with STX-1150 for the treatment of hypercholesterolemia. To broaden and accelerate the impact of its engineered CRISPR technologies for patients, Scribe has formed strategic collaborations with world-leading pharmaceutical companies including Sanofi and Eli Lilly. Co-founded by Nobel Prize winner Jennifer Doudna and backed by leading life sciences investors, Scribe is advancing scalable, transformative, and preventative genetic medicines with the goal of improving outcomes and democratizing access to the protective effects of beneficial human genetics. To learn more, visit www.scribetx.com.

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Media Contact:

Thermal for Scribe Therapeutics

media@scribetx.com

Source: Scribe Therapeutics, Inc.