News

Regenxbio’s One-time Gene Therapy RGX-181 Wins FDA’s Orphan Drug Designation

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Regenxbio’s one-time gene therapy RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease. Regenxbio expects to submit an Investigational New Drug (IND) application to the FDA…

Researchers ID 8 Potential Biomarkers of Disease Severity in Late Infantile Batten Disease

Researchers have identified eight potential biomarkers of disease severity, progression and treatment response in children with late infantile Batten disease. Considering the nature of these biomarkers, the team suggested that these patients may have a disease-associated brain energetic deficit that could drive neurodegeneration. The study, “Untargeted…