LEXINGTON, Mass. and NEW YORK, May 12, 2025 /PRNewswire/ -- Genezen, a leading gene therapy CDMO, has partnered with Elly's Team, a foundation dedicated to accelerating the translation of medical research into treatments, for an AAV9 gene replacement therapy targeting Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS).
In February 2024, Elly's Team founders Michelle and Dan Krueger received a diagnosis for their infant daughter Elly: a mutation in the Interferon regulatory factor 2 binding protein-like (IRF2BPL) gene, which causes NEDAMSS. This ultra-rare and progressive neurodevelopmental disorder affects motor skills, speech, eating, and vision and causes seizures. Fewer than 150 cases have been identified worldwide, and currently, there is no available treatment or cure.
Elly's Team board member Dr. Kathrin Meyer, previously a Principal Investigator at Nationwide Children's Hospital and currently Chief Scientific Officer at Alcyone Therapeutics, in collaboration with Elly's Team, has been researching therapeutic strategies for IRF2BPL mutations. Through additional collaborations with Dr. Zachary Grinspan from Weill Cornell Medicine, Dr. Andrea Sierra Delgado and Dr. Smita Saxena from University of Missouri, and experts from the RTW Foundation, Elly's Team selected an AAV9-based gene replacement therapy as a promising approach.
To support the IND submission, Elly's Team has leveraged Genezen's CMC expertise for development of analytical/ancillary studies and received guidance from Chief Technical and Commercial Officer Dr. Susan D'Costa throughout the development process. Says D'Costa, "We are honored to work with Elly's Team on this critical therapy, and proud to have performed our analysis within a tight timeline to support the IND submission."
On April 3, 2025, Elly became the first child to receive an IRF2BPL gene replacement therapy at Weill Cornell in New York City. Elly's mother, Michelle, co-founder of Elly's Team, says, "This achievement marks a major milestone, not only for our family but also for the entire IRF2BPL community. Elly's Team's mission is to fight for viable treatments for all children affected by NEDAMSS, and working with partners like Genezen is a critical component in achieving this goal."
Recently appointed to the Elly's Team board, Dr. D'Costa continues to provide support to Elly's Team in their endeavor to bring treatments for NEDAMSS to market as quickly as possible. Steve Favaloro, Chairman and CEO at Genezen commented, "Genezen's work on the program and the addition of Susan to the board at Elly's Team highlights our continued support of the rare disease community. It is our strategic imperative to partner with foundations like Elly's Team and grow with them as they move from early-stage to commercial."
About Genezen
Genezen is a best-in-class gene therapy CDMO. With over a decade of experience and state-of-the-art viral vector facilities, including an 86,000-ft2 commercial site, Genezen partners with innovator companies to develop and manufacture gene and cell therapies – from concept to commercial – on a global scale. For more information, visit genezen.com and follow us on LinkedIn.
About Elly's Team:
Elly's Team is a foundation established with a singular goal of translating medical research to treatment in record time. To learn more, please visit EllysTeam.org
SOURCE Genezen
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