LEADING THE WAY IN
RNA TARGETED GENE THERAPIES
Locanabio is creating a new class of genetic medicines that combines the two leading approaches for treating genetic diseases —RNA modification and gene therapy. Locanabio’s new approach to RNA modification leverages our proprietary platform of RNA-binding protein systems. These versatile systems are designed to precisely correct dysfunctional RNA in a variety of ways. The genetic instructions to produce these systems are packaged into a single adeno-associated virus (AAV) vector and administered as a one-time treatment.