Scribe Therapeutics, a genetic medicines company co-founded by Nobel Prize winner Jennifer Doudna, has announced it will present promising new data on its CRISPR-based genome editing technology for Duchenne Muscular Dystrophy (DMD) at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. The presentations, scheduled for May 13-17, 2025, in New Orleans, will showcase the company's innovative approach to treating this severe neuromuscular disorder.
Novel CRISPR Technology Targeting Duchenne Muscular Dystrophy
The first presentation will highlight Scribe's X-Editor (XE) technology and its application in targeting genetic mutations associated with DMD, a progressive condition that causes muscle degeneration and can lead to respiratory and cardiac failure. Principal Scientist Cécile Fortuny, Ph.D., will present findings demonstrating XE as an effective and versatile platform for treating DMD and potentially other inherited muscle disorders.
This research represents a significant step forward in the development of CRISPR-based therapies for genetic muscle diseases, which have historically been challenging to treat effectively. Scribe's approach leverages its proprietary "CRISPR by Design" methodology to engineer more precise and effective genome editing tools.
Collaborative Research with Eli Lilly's Prevail Therapeutics
In a separate presentation, Scribe will join forces with Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, to showcase their collaborative work on self-inactivating adeno-associated viral (AAV) vectors. Senior Scientist Kathleen Christie, Ph.D., will present data demonstrating how these engineered vectors, featuring Scribe's CRISPR-based XE technology with self-targeting guide RNAs, achieved potent, transient, and controllable on-target editing in the central nervous system of mice.
The companies recently announced reaching a success milestone in their collaboration focused on addressing neurological and neuromuscular diseases, though specific details of this achievement have not been disclosed.
"These presentations highlight our ongoing commitment to developing next-generation CRISPR technologies that can address previously untreatable genetic conditions," said a spokesperson for Scribe Therapeutics. "Our engineered XE platform offers unique advantages in specificity and deliverability that could transform treatment approaches for conditions like DMD."
Scribe's Broader CRISPR Innovation Platform
Scribe's approach differs from conventional CRISPR systems by engineering bacterial immune systems into a suite of genome and epigenome editing tools with enhanced activity, specificity, and deliverability. This "CRISPR by Design" methodology aims to create therapies with broader therapeutic windows that could potentially be safe enough for preventative treatment.
Beyond muscular dystrophies, Scribe is advancing its lead candidate, STX-1150, a liver-targeted therapy designed to epigenetically silence the PCSK9 gene for reducing LDL-C levels in cardiometabolic disease. The company has established strategic collaborations with pharmaceutical giants including Sanofi and Eli Lilly to accelerate the development of its engineered CRISPR technologies.
Upcoming Presentations at ASGCT
The DMD-focused presentation, titled "Advancing the Novel CasX-Editor for Precise Genome Editing to Address Muscular Dystrophies," will take place during the Wednesday Poster Reception on May 14, 2025, from 5:30 to 7:00 p.m. CT in Poster Hall I2.
The joint presentation with Prevail Therapeutics, "Self-Targeting CRISPR-CasX-Editor AAV Vectors Enable Potent and Controllable On-target Editing in the Murine CNS," will be held during the Thursday Poster Reception on May 15, 2025, from 5:30 to 7:00 p.m. CT, also in Poster Hall I2.
These presentations at ASGCT, one of the premier conferences for gene and cell therapy research, will provide the scientific community with valuable insights into Scribe's progress in developing potentially transformative treatments for serious genetic disorders.
