Neurogene Announces EMA Grants Orphan Drug Designation to CLN5 Batten Disease Gene Therapy

 Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its adeno-associated virus (AAV) encoding a codon-optimized human CLN5 transgene (hCLN5) for patients diagnosed with CLN5, a subtype of Batten disease.

“Signs and symptoms of CLN5 disease include vision loss, cognitive and motor impairment, and seizures. The disease often takes a rapidly progressive course within a few years of symptom onset, leading to premature death. With no available treatment options, the outcome is devastating for children and their families,” said Angela Schulz, M.D., Ph.D., Children’s Hospital, NCL Specialty Clinic, International Center of Lysosomal Storage Disorders, University Medical Center Hamburg-Eppendorf.

“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,” said Rachel McMinn, Ph.D., Founder and CEO of Neurogene. “Neurogene is collaborating with Batten disease experts, regulatory authorities, and caregivers to provide a safe and effective gene therapy for this disease as quickly as possible.”

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