Andelyn Biosciences Achieves Breakthrough in Gene Therapy for Ultra-Rare Neurodegenerative Disorder

Diagnosed at just 8 months old, Elly Krueger's form of Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS) was rapidly progressing, with no known cures or treatments available. Leveraging their expertise and network, the Kruegers formed Elly's Team, a foundation dedicated to saving their child and others like her. After evaluating multiple treatment options, Andelyn Biosciences was selected to develop, scale up, and manufacture a gene therapy using their AAV Curator(r) Platform. This feat marks a major accomplishment, with the therapy produced and released in just 10 months from development start and 14 months after the patient's initial diagnosis.

Key Takeaways:

• Andelyn Biosciences successfully manufactured a novel viral vector gene therapy to treat NEDAMSS, a neurodegenerative disease with no known cures or treatments available.
• The therapy was produced and released in 10 months from development start and 14 months after the patient's initial diagnosis.
• Elly Krueger, the patient, received the therapy on April 3rd, 2025, at Weill Cornell in New York City.
• The therapy has been safe and well-tolerated one month post-treatment, with Elly doing very well, although efficacy assessments are still ongoing.
• Andelyn's AAV Curator(r) Platform utilizes Optimization-by-DesignTM, a data-driven approach to correct process sizing for scale and speed to the clinic.
• The platform has been reviewed and approved by the FDA, eliminating unnecessary hurdles or delays in IND applications.
• The well-established platform supports exacting viral vector manufacturing needs for ultra-rare diseases like NEDAMSS.
• Andelyn Biosciences prioritizes single-patient projects, with everyone at the organization understanding the importance of speed in these projects.

Statistics:

• 10 months: The time it took to develop and release the gene therapy from development start.
• 14 months: The time from the patient's initial diagnosis to the therapy's release.
• April 3rd, 2025: The date Elly Krueger received the gene therapy at Weill Cornell in New York City.
• 1 month post-treatment: The time by which the therapy has been safe and well-tolerated, with Elly doing very well.

Sources:

• Andelyn Biosciences news release
• Michelle Krueger's quote in the Andelyn Biosciences news release
• Wade Macedone's quote in the Andelyn Biosciences news release