Daniela Roth

Locanabio, Inc.

At Locanabio, we are developing a new class of genetic medicines that has the potential to significantly improve the lives of patients with devastating genetic diseases by correcting the message of disease-causing RNA. Our proprietary platform uses gene therapy to deliver RNA-binding systems, including snRNA, Cas13d and PUF, that can be engineered to selectively manipulate disease-causing RNA by multiple mechanisms. Our systems are designed to provide a durable therapy with a single administration without altering a cell's DNA. Locanabio's platform has applications across a range of tissues and diseases, and we are currently advancing programs in rare genetic neuromuscular and neurodegenerative diseases. With world leadership in RNA biology and RNA-mediated disease and a top tier Board of Directors and panel of Scientific Advisors, we are poised to develop the next generation of genomic medicines for functional correction of disease.