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Andelyn Manufactures Novel Gene Therapy for Ultra-Rare NEDAMSS Disease Patient

The therapy for Elly Krueger was produced and released only 10 months from the start of development and 14 months after the patient’s initial diagnosis.

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By: Charlie Sternberg

Associate Editor

Elly Krueger.

Andelyn Biosciences Inc., a patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has successfully 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). NEDAMSS is a neurodegenerative disease affecting the central nervous system and is a regressive disorder with no known cures or ...

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