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Neurogene secures EMA orphan designation for gene therapy in rare, inherited aspartylglucosaminuria

Published on 03/12/20 at 01:03pm

The European Medicines Agency (EMA) has bestowed Orphan Drug Designation (ODD) to Neurogene’s adeno-associated virus vector (AAV) gene therapy in the treatment of aspartylglucosaminuria (AGU), a rare hereditary disease with no currently approved treatments.

A progressive neurodegenerative lysosomal storage disorder, AGU is caused by a variant in the human AGA gene, an engineered transgene encoding of which is included in Neurogene’s therapy.

Characterised primarily by developmental delays, the condition causes an accumulation of toxic material in the brain and other organs, leading to symptoms including cognitive defects, chronic ear and respiratory infections, behavioural issues, seizures and more, leading eventually to premature death.

“Due to the lack of approved treatment options and the severity of symptoms, the prognosis and quality of life for patients and families affected by AGU can often be very poor,” said Dr Minna Laine, Child Neurologist at Helsinki University Hospital. “The EMA granting Orphan Drug Designation for a potential gene therapy for AGU represents promise while underscoring the urgent, unmet medical need for patients around the world diagnosed with this condition.”

According to the company, the designation is Neurogene’s third for a neurological disease with unmet need, after the FDA granted ODD to two of the company’s gene therapies for two sub-types of the neurodegenerative condition Batten’s disease.

“Gaining this important regulatory milestone designation means we are one step closer to moving the first AGU treatment option into the clinic,” said Dr Rachel McMinn, Neurogene’s Founder and Chief Executive Officer. “Neurogene is collaborating with regulatory authorities, families and patients to make a safe and effective gene therapy for AGU available as quickly as possible.”

Matt Fellows

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