Andelyn Biosciences has successfully developed and manufactured a gene therapy using its AAV Curator Platform for a young patient with NEDAMSS (Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures), a rare neurodegenerative disorder. The therapy was delivered just 10 months after development began, and only 14 months following the child's diagnosis with an IRF2BPL gene mutation.

NEDAMSS severely impacts the nervous system, leading to symptoms such as vision loss, speech regression, seizures, and motor decline. Currently, there is no cure or established treatment for this condition. Eight-month-old Elly Krueger, diagnosed in February 2023, became the first recipient of the IRF2BPL gene therapy at Weill Cornell in April 2025.

Elly’s family initiated “Elly’s Team,” a foundation to support research and therapy development. After assessing options, Andelyn Biosciences was chosen to produce the therapy using its data-driven, Optimisation-by-Design AAV Curator Platform, which enables fast, scalable, and regulatory-compliant production.

Michelle Krueger, Elly’s mother, expressed deep gratitude for the team’s commitment and care throughout the process. One month after treatment, Elly has shown no negative side effects, and the family remains optimistic as regulatory efforts aim to expand access.

CEO Wade Macedone emphasized that supporting individual patients is core to Andelyn’s mission. The AAV Curator Platform, with FDA-reviewed Drug Master Files, supports rapid development and manufacturing for both large-scale and rare disease therapies, offering hope to families affected by ultra-rare conditions.