Andelyn Biosciences Inc., a patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has successfully manufactured a novel viral vector gene therapy with its AAV Curator Platform to treat a baby with the ultra-rare disease Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS).

NEDAMSS is a neurodegenerative disease affecting the central nervous system and is a regressive disorder with no known cures or treatments available. It can impact motor skills, speech, eating, and eyesight, among other functions, and often causes seizures. Diagnosed on Feb 8, 2023 at just 8 months old, Elly Krueger’s form of NEDAMSS, caused by a mutation in the IRF2BPL (Interferon regulatory factor 2 binding protein-like) gene, was rapidly progressing, making every day more pressing than the one before.

The therapy was produced and released only 10 months from the start of development and 14 months after the patient’s initial diagnosis.

Michelle Krueger, mother of Elly 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, “Supporting single-patient projects is part of the DNA of Andelyn Biosciences and the lifestyle of everyone who works at the organization. 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. Individually, we all know that with these projects, there is someone, usually a young child, whose life depends on us.”

One-month post-treatment, the therapy has been safe and well tolerated, and Elly is doing very well. While it is still too early to assess efficacy, the Kruegers are filled with hope and optimism. Everyone is encouraged by the FDA’s support to expand this treatment for other children.