EMA Names Potential Gene Therapy for CLN5 Batten an Orphan Drug

EMA Names Potential Gene Therapy for CLN5 Batten an Orphan Drug

298289

EMA Names Potential Gene Therapy for CLN5 Batten an Orphan Drug

The European Medicines Agency (EMA) designated Neurogene’s gene therapy candidate for CLN5, a form of late-infantile Batten disease, an orphan drug as a potential treatment of the disorder. “This regulatory designation is an important milestone toward addressing the urgent, unmet need of families and patients living with CLN5, and follows the orphan drug designation previously granted in 2020 by the U.S. Food and Drug Administration (FDA) for this same gene therapy candidate,” Rachel McMinn, PhD, Neurogene’s…

You must be logged in to read/download the full post.