Condition: Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD · Sponsor: Precision BioSciences, Inc.
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.
This description comes directly from the study's public registry record.
Precision BioSciences Clin Ops · (800) 593-0346 · function-DMD@precisionbiosciences.com
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| Arkansas Children's Hospital | Little Rock, Arkansas, United States | Recruiting |
| Washington University School of Medicine | St Louis, Missouri, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07429240