Biopharmaceutical company developing genomic medicines for neurodegenerative diseases, initially targeting ALS
Biopharmaceutical company developing genomic medicines for neurodegenerative diseases, initially targeting ALS. Its lead antisense oligonucleotide program aims to correct UNC13A mRNA splicing and restore UNC13A protein function and synaptic communication. The company is also researching biomarkers to identify patients likely to benefit from its approach.