Andelyn Biosciences Uses AAV Curator® for NEDAMSS Gene Therapy 14 Months Post-Diagnosis

COLUMBUS, Ohio, May 12, 2025 -- Andelyn Biosciences, Inc., a leader in cell and gene therapy development, has achieved a significant milestone with the successful production of a viral vector gene therapy using its AAV Curator® Platform. This therapy targets Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS), a rare and debilitating neurodegenerative condition. The therapy was developed and released in just 10 months, following the patient’s initial diagnosis 14 months prior, highlighting the potential of gene therapies to offer rapid and effective solutions for rare diseases.

NEDAMSS is a severe disorder affecting the central nervous system, leading to a decline in motor and speech abilities, as well as issues with eating and eyesight, often resulting in seizures. Diagnosed at only eight months old on February 8, 2023, Elly Krueger was facing a swift progression of the disease caused by a mutation in the IRF2BPL gene. Her parents, driven by the urgency of her condition, established Elly's Team, a foundation dedicated to finding a treatment for NEDAMSS. Through their efforts and collaboration with Andelyn Biosciences, a promising gene therapy was identified, and Andelyn was chosen to develop and manufacture the treatment.

On April 3, 2025, Elly became the first recipient of the IRF2BPL gene replacement therapy at Weill Cornell in New York City. Her mother, Michelle Krueger, expressed gratitude for the milestone, recognizing its significance not just for their family but for the entire community affected by the IRF2BPL mutation. She emphasized the dedication of the Andelyn team, who worked tirelessly and personally engaged with Elly, understanding the urgency and impact beyond the laboratory.

A month after the treatment, Elly is reportedly in good health with the therapy being well-tolerated. While it is too soon to determine the long-term efficacy, the outcome so far has instilled hope within her family and supporters. The success of this endeavor has been buoyed by the support of the FDA, raising optimism about expanding the treatment to benefit other children with similar conditions.

Wade Macedone, CEO of Andelyn Biosciences, remarked on the importance of their work with individual patient projects, underscoring the company's commitment to addressing the needs of patients, particularly children, who rely on these treatments for their survival. The company's AAV Curator® Platform is pivotal in this process, offering a scalable, efficient, and reliable methodology for developing viral vector therapies. This platform is designed to yield high-quality results while expediting the regulatory review process, thanks to its established Drug Master Files with the FDA.

Andelyn Biosciences, operating from Columbus, Ohio, is a comprehensive cell and gene therapy CDMO, boasting over two decades of experience. The company provides expertise in developing and producing viral vectors for gene therapies, supporting projects from initial concept through to clinical and commercial manufacturing. Their configurable AAV Curator® Platform allows for streamlined development and manufacturing processes, further supported by a robust quality system and regulatory assistance.

Elly's Team, founded with a mission to accelerate medical research into treatment, continues to support research and development of therapies for NEDAMSS. The foundation aims to fund additional studies to better understand the disease and develop viable treatments for all children affected by this condition.