Biotechnology
a Baltimore, Maryland-based biotechnology company and Johns Hopkins University spinout dedicated to developing pioneering gene therapies and biomarkers for neurodegenerative diseases driven by TDP-43 dysfunction. The company’s flagship program, AAV9-CTR, is engineered as a one-time intrathecal gene therapy designed to rescue transcriptome-wide splicing errors and restore normal cellular function across hundreds of targets, initially focusing on amyotrophic lateral sclerosis (ALS) with future plans to expand into frontotemporal dementia (FTD) and Alzheimer’s disease.