LBIO-115

LBIO-115 is a preclinical adeno-associated virus serotype 9 (AAV9) gene therapy candidate from Locanabio that delivers engineered small nuclear RNAs (snRNAs) to promote exon 51 skipping in the dystrophin (DMD) pre-mRNA for the treatment of Duchenne muscular dystrophy patients with mutations amenable to exon 51 correction.[1][3][5][7][8] Using a vectorized snRNA exon-skipping approach, LBIO-115 achieves high levels of exon 51 skipping (>80–90%) and robust dystrophin restoration in skeletal and cardiac muscle in patient-derived cells and DMD mouse models, with associated improvements in muscle pathology and creatine kinase reductions and minimal off‑target activity reported in vivo.[1][3][5][7] The program is in IND-enabling studies and represents Locanabio’s lead application of its RNA-targeted AAV platform, with the same snRNA backbone planned for expansion to DMD exons 53, 45, and 44.