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Scribe Achieves Milestone for In Vivo Program in Collaboration with Prevail Therapeutics, a part of Lilly’s Gene Therapy Division, and Announces ASGCT Joint Presentation on Self-Inactivating AAV Technology for Neurological and Neuromuscular Disorders

via Business Wire
  • Success milestone reached in Scribe and Prevail’s ongoing collaboration to accelerate in vivo therapeutics for neurological and neuromuscular diseases using Scribe’s CRISPR technology
  • Companies to jointly present new preclinical data on self-inactivating AAV (siXAAV) technology from their collaboration at the 28th ASGCT Annual Meeting

Scribe Therapeutics Inc., a genetic medicines company unlocking the potential of CRISPR to transform human health, today announced the achievement of a success milestone for one of the targets in its research collaboration with Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company (NYSE: LLY), to develop in vivo CRISPR-based therapeutics for neurological and neuromuscular disorders. The success milestone marks Scribe’s second such partnership achievement in 2025.

At the 28th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, the companies will have a joint presentation titled “Self-Targeting CRISPR-CasX-Editor AAV Vectors Enable Potent and Controllable On-target Editing in the Murine CNS.” The presentation will take place during the poster reception between 5:30 and 7:00 p.m. CT on Thursday, May 15, 2025, at the New Orleans Ernest N. Morial Convention Center.

"We're thrilled by our strong progress with the Prevail and Lilly teams in developing CRISPR-based therapies and delivery systems for devastating neurological diseases," said Benjamin Oakes, Ph.D., CEO of Scribe. "This milestone and presentation represent more than the continued validation of Scribe's holistic engineering approaches; they represent a leap towards safer and more effective in vivo genetic medicines for neurodegenerative disorders."

CRISPR by Design™ is Scribe’s data-driven design and engineering approach for optimizing its CRISPR-based platforms and assets, including the X-Editor (XE) technology for genome editing, to drive forward a new era of transformative genetic medicines.

"Achieving this success milestone and having a joint presentation at ASGCT together underscore the powerful synergy between Scribe's cutting-edge CRISPR technology and Prevail's expertise in addressing neurological and neuromuscular diseases," said Svetlana Lucas, Ph.D., Chief Business Officer at Scribe. "We are confident that this collaboration will continue to unlock new solutions and deliver on our shared commitment to bringing transformative genetic medicines to patients worldwide."

As part of the company’s research collaboration with Prevail Therapeutics, a part of Lilly’s Gene Therapy Division, 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.

About Scribe Therapeutics

Scribe Therapeutics is revolutionizing medicine by developing optimized in vivo CRISPR-based genetic medicines designed to become standard of care treatments for patients suffering from highly prevalent diseases, starting with cardiometabolic disease. The company is on a mission to build the first CRISPR-based therapeutics that are effective and safe enough to transform everyone’s lifetime risk for disease. Scribe’s CRISPR by Design™ approach engineers bacterial immune systems into a premier suite of genome and epigenome editing tools built for unique molecular advantages in activity, specificity, and deliverability, enabling the creation of therapies with a broader therapeutic window and safe for use as a preventative treatment. The company’s lead candidate, STX-1150, is a novel liver-targeted therapy designed to epigenetically silence the PCSK9 gene, resulting in significant and durable reduction of LDL-C levels. 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 engineering the future of genetic medicine. To learn more, visit www.scribetx.com.

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