Locanabio develops RNA-targeted genetic medicines delivered by AAV, using engineered small nuclear RNAs (snRNAs) alongside Cas13d and PUF platforms to selectively manipulate disease-causing RNA. Its vectorized snRNA platform can deliver multiple snRNAs in a single AAV to increase potency or target multiple RNAs and is designed for a one-time administration without altering cellular DNA. Locanabio describes snRNAs as RNA-exclusive, precise, and non-immunogenic tools that can effect exon skipping, RNA editing, and other mechanisms. The company is advancing programs in rare genetic neuromuscular and neurodegenerative diseases, including DMD exon-skipping programs. Its exon 51 program, intended to enable production of a near full-length dystrophin protein, is in IND-enabling studies. Locanabio expects to present additional data at an upcoming peer-reviewed scientific conference and recently received an equity investment from CureDuchenne Ventures to support its snRNA-mediated exon skipping work. Locanabio is a San Diego-based RNA-targeting gene therapy company focused on developing therapies for patients with severe neurodegenerative, neuromuscular and retinal diseases. The company is advancing a portfolio of RNA-targeted gene therapies aimed at multiple genetic diseases that currently have no approved therapeutic alternatives. Indications specifically mentioned include Huntington’s disease, spinocerebellar ataxia type 1, myotonic dystrophy type 1, genetic forms of amyotrophic lateral sclerosis and retinal diseases. Locanabio recently raised $100M in a Series B financing to advance its pipeline and expand the underlying technology platform to pursue a broader range of therapeutic indications. Jim Burns, Ph.D., serves as chief executive officer. Rajul Jain, M.D., director of Vida Ventures, will join Locanabio’s board of directors.

Total raised

$100M

Funding rounds

2

Latest round

Series B

Latest activity

Dec 2020