EMA Grants Orphan Drug Designation to CLN5 Batten Disease Gene Therapy

Release Date: 04-Sep-2021

Neurogene Inc., a company receives Orphan Drug Designation from European Medicines Agency (EMA) to its adeno-associated virus (AAV) encoding a codon-optimized human CLN5 transgene (hCLN5) to treat CLN5, a subtype of Batten disease. Symptoms of CLN5 disease include cognitive and motor impairment, vision loss, and seizures.


It is given to investigational therapies that have the power to treat rare and life-threatening diseases that affect less than five in 10,000 patients in the European Union. It provides some benefits to companies, like clinical protocol advice, fee reductions, and ten years of protection from competition following regulatory approval.


Batten disease which is also called neuronal ceroid lipofuscinoses (NCL), is a rare neurodegenerative diseases affected by pathogenic changes in one of a series of genes, resulting in the accumulation of toxic deposits across multiple organ systems, including the eye, skin, brain and others. Batten disease lies within the family of lysosomal storage disorders (LSDs). LSDs are progressive, multisystemic, inherited metabolic disorders linked with premature death.

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