Scribe Therapeutics to Highlight In Vivo CRISPR-based Genome Editing Data for Duchenne Muscular Dystrophy at ASGCT 2025, in Addition to Joint Presentation with Prevail Therapeutics, a Part of Lilly’s Gene Therapy Division

• Company will highlight the potential for Scribe’s X-Editor (XE) technology to address muscular dystrophies such as Duchenne Muscular Dystrophy
• Joint presentation with Prevail Therapeutics, a part of Lilly’s Gene Therapy Division, to highlight Scribe’s engineered self-inactivating AAV technology for use in vivo

ALAMEDA, Calif.--(BUSINESS WIRE)--Scribe Therapeutics Inc. (Scribe), a genetic medicines company unlocking the potential of CRISPR to transform human health, today announced its participation in the 28th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place May 13-17, 2025, at the Ernest N. Morial Convention Center in New Orleans, LA.

Scribe will deliver two presentations that showcase the company’s ongoing advances enabled by its CRISPR by Design^™ approach to engineer novel in vivo genetic medicines. In one demonstration of potent therapeutic application, Scribe will present novel genome editing strategies using its X-Editor (XE) technology to target mutations in Duchenne Muscular Dystrophy (DMD), a severe neuromuscular disorder that can lead to respiratory and cardiac failure. Findings from this study will showcase XE as an effective and versatile platform for the treatment of DMD and other inherited muscle disorders.

The joint presentation will describe work by Scribe and Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company (NYSE: LLY), to engineer self-inactivating adeno-associated viral (AAV) vectors featuring Scribe’s CRISPR-based XE with self-targeting guide RNAs and results demonstrating potent, transient, and controllable on-target in vivo editing in the central nervous system of mice. The companies recently reached a success milestone in their collaboration to address neurological and neuromuscular diseases. Additional details can be found on Scribe’s website.

The presentation details are listed below. Full abstracts are available on the ASGCT website.

Title: Advancing the Novel CasX-Editor for Precise Genome Editing to Address Muscular Dystrophies
Session title: Wednesday Poster Reception
Presenting company: Scribe Therapeutics
Date: Wednesday, May 14, 2025
Time: 5:30 - 7:00 p.m. CT
Location: Poster Hall I2
Speaker: Cécile Fortuny, Ph.D., Principal Scientist

Title: Self-Targeting CRISPR-CasX-Editor AAV Vectors Enable Potent and Controllable On-target Editing in the Murine CNS
Session title: Thursday Poster Reception
Presenting companies: Scribe Therapeutics, Prevail Therapeutics
Date: Thursday, May 15, 2025
Time: 5:30 - 7:00 p.m. CT
Location: Poster Hall I2
Speaker: Kathleen Christie, Ph.D., Senior Scientist I

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.