Andelyn Biosciences Manufactures Gene Therapy for Ultra-Rare NEDAMSS

Elly’s Team selected a gene therapy candidate and partnered with Andelyn Biosciences for its development, scale-up, and manufacturing.

Andelyn Biosciences has developed and delivered a viral vector gene therapy using its proprietary AAV Curator Platform for a patient diagnosed with Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS), a rare and progressive neurodegenerative disorder.

According to the company, the therapy was completed within 10 months of program initiation and 14 months following the patient's diagnosis.

NEDAMSS, caused by mutations in the IRF2BPL gene, impacts the central nervous system and leads to deterioration in motor function, speech, eating ability, vision, and triggers frequent seizures. With no established treatments or cures, the condition poses a critical challenge.

The AAV Curator Platform is built to support compliant, scalable, and IP-contained development. Its Drug Master Files (DMFs), previously reviewed and accepted by the US FDA, streamline regulatory pathways and reduce time-to-clinic. The platform enables manufacturing flexibility across various patient volumes, including for ultra-rare disorders like NEDAMSS.

One such case is that of Elly Krueger, diagnosed in February 2023 at just eight months old. In response, her family founded Elly’s Team, a nonprofit committed to accelerating treatment development for NEDAMSS.

The foundation selected a gene therapy candidate and partnered with Andelyn Biosciences for its development, scale-up, and manufacturing.

The project was transferred to Andelyn’s AAV Curator Platform, which leverages a data-driven Optimisation-by-Design approach to expedite therapy production.

Michelle Krueger, Elly’s mother and co-founder of Elly’s Team, said, “On April 3rd, 2025, Elly became the first child to receive an IRF2BPL gene replacement therapy at Weill Cornell in New York City. This achievement marks a major milestone, not only for our family but also for the entire IRF2BPL community.”

Wade Macedone, CEO of Andelyn Biosciences, said, “As a company we are as proud of successfully completing single-patient programs as we are scaling a manufacturing process for 10,000 or more patients.”

As of one month post-treatment, Elly has tolerated the therapy well, with no reported adverse effects.

Andelyn Biosciences plans to share additional details on Elly’s progress and the therapy’s development in an upcoming update.

Stay tuned for more such updates on Digital Health News.

Stay tuned for more such updates on Digital Health News