LBIO-135

LBIO-135 is an adeno-associated virus (AAV) gene therapy candidate developed by Locanabio for the treatment of Duchenne muscular dystrophy (DMD). It utilizes a proprietary vectorized small nuclear RNA (snRNA) platform to deliver multiple snRNA sequences designed to induce skipping of exon 53 in the dystrophin pre-mRNA. This mechanism aims to restore the reading frame of the dystrophin gene, allowing for the production of a truncated but functional dystrophin protein in patients with specific mutations. LBIO-135 was designed to overcome the packaging constraints of AAV vectors by efficiently delivering multiple snRNA constructs. Although it showed promise in preclinical models, development was halted following Locanabio's decision to cease operations in late 2023.