#United States #New York #Genezen #Elly's Team #AAV9 Therapy

Genezen and Elly's Team Unite for Innovative Gene Therapy

Genezen, a prominent contract development and manufacturing organization (CDMO) focused on gene therapies, has announced a partnership with Elly's Team, a nonprofit dedicated to transforming medical research into viable treatments. This collaboration centers around the development of a gene replacement therapy utilizing AAV9 vectors, targeting a severe neurodevelopmental disorder termed Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS).

The NEDAMSS Condition

NEDAMSS is a challenging condition characterized by a combination of neurological symptoms including severe developmental delays, loss of motor skills, and seizures, largely due to a mutation in the IRF2BPL gene. In February 2024, the founders of Elly's Team, Michelle and Dan Krueger, received heartbreaking news when their infant daughter, Elly, was diagnosed with this ultra-rare disorder. Currently, fewer than 150 cases have been reported globally, highlighting the urgent need for effective treatments.

Research and Development Journey

Elly's Team has been fortunate to collaborate with some of the leading experts in the field, such as Dr. Kathrin Meyer and Dr. Zachary Grinspan, who have conducted significant research on therapeutic strategies for the IRF2BPL mutation. Supported by international researchers, the team identified AAV9-based gene replacement therapy as a promising avenue for treatment.

Furthermore, Genezen has played a vital role in the developmental process, utilizing its contract manufacturing expertise to assist in the analytical studies needed for Investigational New Drug (IND) submission. According to Genezen's Chief Technical Officer, Dr. Susan D'Costa, the team's commitment to this project has enabled the research to progress rapidly.

A Milestone Achievement

A significant breakthrough occurred on April 3, 2025, when Elly became the first child to receive the AAV9 gene therapy at Weill Cornell Medicine in New York City. This milestone is not just a personal victory for the Krueger family, but a beacon of hope for families across the globe affected by NEDAMSS. Michelle Krueger articulates the emotional weight of this achievement, stating, "This marks a vital step not only for our family but also for the entire IRF2BPL community. Our mission is to advocate for effective treatments for all children facing this disorder."

Continuing Support and Future Goals

Dr. D'Costa's recent appointment to the board of Elly's Team underscores Genezen's ongoing commitment to the rare disease community. Steve Favaloro, Genezen's Chairman and CEO, emphasized the importance of partnerships like the one with Elly's Team, expressing a desire to grow alongside them as they transition from foundational research to potential treatments available to patients.

As Elly's Team moves forward, their collaboration with Genezen will be pivotal in driving further research and facilitating the market introduction of viable treatments for NEDAMSS, potentially changing the lives of many affected families.

About Genezen and Elly's Team

Genezen is a leader in the gene therapy manufacturing sector, boasting over a decade of experience in the development of gene and cell therapies. The nonprofit Elly's Team focuses on transforming medical research into tangible treatment solutions in an expedited manner.

For further updates on their progress and initiatives, you can visit Genezen’s official website or Elly's Team's platform.