NEW YORK – Genetic medicines firm Locanabio on Monday announced an equity investment from nonprofit CureDuchenne's investment arm, CureDuchenne Ventures.
Locanabio, through a spokesperson, declined to share financial details of the investment.
The investment will support development of San Diego-based Locanabio's RNA-targeted gene therapies for Duchenne muscular dystrophy (DMD), a rare degenerative neuromuscular disorder caused by mutations in the DMD gene that result in dysfunctional dystrophin, a protein that's integral to muscle function.
Locanabio has two gene therapies in its pipeline designed to restore muscle health for patients with DMD. The therapies use an adeno-associated virus (AAV) vector to deliver engineered small nuclear RNAs (snRNAs) that target splicing regulatory sites in the dystrophin mRNA, promoting exon skipping and producing functional dystrophin.
One of the therapies, which is in investigational new drug (IND)-enabling studies, is designed to skip exon 51 on the DMD gene. The other is designed to skip exons 44, 45, and 53 and is in a preclinical phase.
"We look forward to advancing product candidates for additional exon mutations leveraging our snRNA-mediated exon skipping approach in DMD," Locanabio CEO Jim Burns said in a statement.