Many severe genetic diseases have no cure because current therapies cannot address the underlying cause: damaged RNA. Locanabio is changing that.
Their CorrecTx™ platform targets disease-causing RNA sequences using engineered RNA-binding proteins, such as Cas13d and PUF proteins. These proteins are fused with effector domains that can selectively destroy toxic RNA, fix splicing errors, boost protein production, or correct point mutations at the RNA level. Unlike traditional gene editing, CorrecTx modifies RNA only, preserving healthy alleles and avoiding changes to DNA.
This RNA-targeting approach shows promise in diseases like Huntington’s disease, where toxic CAG repeats cause nerve cell damage, and myotonic dystrophy type 1, where toxic RNA traps key proteins. CorrecTx can also restore protein expression in exon-mutation diseases like Duchenne muscular dystrophy and Usher syndrome type 2 by skipping faulty exons.
Delivered as a one-time gene therapy, CorrecTx operates independently of the cell’s own repair machinery, making it highly versatile and precise.
3D animation by science animation vendor Life Science Animation.
