Andelyn Biosciences Achieves Milestone in Gene Therapy for Rare Disease After 14 Months of Determined Efforts

Andelyn Biosciences' Groundbreaking Achievement in Gene Therapy for Ultra-Rare Disease



In a remarkable display of innovation and compassion, Andelyn Biosciences, a prominent cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has successfully engineered a novel viral vector gene therapy aimed at treating a rare neurodevelopmental disorder known as NEDAMSS (Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures). This milestone comes just 14 months after the initial diagnosis of the patient, a baby named Elly Krueger, making a significant contribution to the field of gene therapy for ultra-rare diseases.

The Challenge of NEDAMSS


NEDAMSS is a debilitating condition that affects the central nervous system, characterized by a regression of motor skills, speech, and even basic functional abilities. As Elly was diagnosed at only eight months, her parents quickly realized the urgency of her condition and devoted themselves to finding a viable treatment option. In a race against time, the Krueger family formed a foundation, aptly named Elly's Team, dedicated to securing a treatment for their daughter and other children suffering from the same disorder.

Collaboration and Development


The path to developing this groundbreaking therapy involved extensive collaboration and a data-driven approach. The Kruegers, leveraging their networks and expertise, evaluated numerous treatment options before deciding to partner with Andelyn Biosciences. The company's AAV Curator® Platform was pivotal in the design and manufacturing processes, ensuring a rapid turnaround for the necessary gene therapy.

A Milestone Administration


On April 3, 2025, Elly became the first patient to receive the IRF2BPL gene replacement therapy at a facility in New York City. Her mother, Michelle Krueger, expressed overwhelming gratitude for the support and dedication displayed by the Andelyn team throughout this journey.

Topics Health)

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