Andelyn Biosciences uses AAV curator platform to deliver novel gene therapy for ultra-rare NEDAMSS disease
The platform allows for the ability to support the exacting viral vector manufacturing needs of different types of patients
Andelyn Biosciences, a cell and gene therapy Contract Development and Manufacturing Organisation (CDMO), has 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). The therapy was produced and released within 10 months from the start of development and 14 months after the patient’s initial diagnosis.
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.
Elly Krueger’s form of NEDAMSS, caused by a mutation in the IRF2BPL (Interferon regulatory factor 2 binding protein-like) gene, was diagnosed on Feb 8, 2023, at 8 months old. After evaluating multiple possible treatment options, a potential gene therapy was chosen, and Andelyn Biosciences was selected to develop, scale, and manufacture the drug. On April 3rd, 2025, Elly became the first child to receive an IRF2BPL gene replacement therapy at Weill Cornell in New York City. One-month post-treatment, the therapy has been safe and well tolerated, while it is still too early to assess efficacy.
Andelyn’s AAV Curator Platform utilises Optimisation-by-Design, a data-driven approach to size processes for scale and speed to the clinic. The platform is highly predictable in terms of yield and quality and eliminates intellectual property concerns because the technology has all been developed internally. Furthermore, review of investigational new drug (IND) applications is streamlined as the referenced Drug Master Files (DMFs) have been reviewed and approved by the FDA previously. The platform allows for the ability to support the exacting viral vector manufacturing needs of different types of patients, including for ultra-rare diseases like NEDAMSS.