Andelyn Biosciences manufactures gene therapy for ultra-rare NEDAMSS disease using AAV curator platform

Andelyn Biosciences has announced the successful manufacture and delivery of a viral vector gene therapy using its AAV Curator Platform for a patient diagnosed with Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS), an ultra-rare neurodegenerative disease. The therapy was developed and released 10 months from the start of the programme, and just 14 months after the patient’s initial diagnosis.

NEDAMSS affects the central nervous system, with symptoms that can include loss of motor function, speech, eating ability, vision, and frequent seizures. There are currently no known cures or established treatments for this condition. Elly Krueger was diagnosed with NEDAMSS on 8 February 2023 at the age of eight months. Her condition, caused by a mutation in the IRF2BPL gene, was rapidly progressing.

In response, the Krueger family launched Elly’s Team, a foundation dedicated to advancing treatment for NEDAMSS. After evaluating several treatment options, a gene therapy candidate was selected and Andelyn Biosciences was chosen to develop, scale, and manufacture the therapy. The Andelyn team transferred the programme in and began manufacturing using the AAV Curator Platform, which uses a data-driven Optimisation-by-Design approach to accelerate development.

Michelle Krueger, Elly’s mother, 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. Throughout this experience, the Andelyn Biosciences team became like part of our family. Everyone at the company knew the importance of speed. Many of them met Elly, held her, and prayed for her. Every step of the way, they knew there was a purpose beyond the vial – for Elly and all the other children affected by this devastating disease.”

One month post-treatment, Elly has tolerated the therapy well, with no adverse effects reported. Although efficacy is yet to be assessed, the family remains hopeful, bolstered by regulatory support to potentially expand access to similar patients.

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.”

The AAV Curator Platform allows scalable, compliant, and IP-contained development processes. Its Drug Master Files (DMFs) have previously been reviewed and approved by the US FDA, enabling faster regulatory review and reducing delays in clinical readiness. The platform’s structure enables manufacturing for a wide range of patient needs, including those with ultra-rare diseases such as NEDAMSS.

Further details on Elly’s journey and the therapy development process will be shared by Andelyn Biosciences in a forthcoming update.