Dear Fellow IRF2BPL Parents & Community,
We are the parents of Elly, a brave and resilient almost two-year-old girl diagnosed with NEDAMSS on February 8th, 2024. We are also the founders of Elly’s Team, a foundation dedicated to translating medical research into treatment in record time.
Today, we are thrilled to share some groundbreaking news: on April 3rd, 2025, Elly became the first child to receive an IRF2BPL gene replacement therapy at Weill Cornell in New York City. This achievement marks a major milestone, not only for our family but also for the entire IRF2BPL community.
After receiving Elly’s diagnosis and evaluating all potential treatment options, we decided to pursue the development of a gene replacement therapy in collaboration with Dr. Kathrin Meyer, Dr. Zachary Grinspan and many others, as it presented the most promising treatment option for Elly given the severity of her disease. We would like to thank the parents, scientists, and foundations who came before us whose hard work and contributions laid significant groundwork in understanding our disease and enabling first steps on the path to generating a gene therapy.
Before proceeding with Elly’s treatment, we rigorously tested the therapy in mice and pigs to evaluate its safety and conducted efficacy studies in patient-derived cells, mice, and tadpoles. After months of hard work, encouraging data, and interactions with the FDA, we received approval in March 2025 to proceed with the therapy in humans and provided the first dose to Elly, a mere 14 months after her diagnosis and 10 months after we began our gene therapy pursuit. A timeline that was only accomplished due to relentless effort, a lot of prayer, divine intervention and a series of miracles.
We are excited to report that one-month post-treatment, the therapy has been safe and well tolerated. Elly is doing very well. While it is still too early to assess efficacy, we are filled with hope and optimism. This is an extraordinary moment for all of us, and we are encouraged by the FDA’s support to expand this treatment for other children.
As a faith-based foundation, Elly’s Team’s mission is to fight for viable treatments for all children affected by NEDAMSS. We believe in the power of unity within our community, and we are committed to collaborating as we continue to push forward with our mission to translate medical research into novel treatments. We are excited to share that additional doses of the gene therapy will be made available to NEDAMSS patients in the scope of a phase 1/2 clinical trial following FDA sign off.
We want to express our deepest gratitude to all members of Elly’s Team who made this incredible treatment a reality, including but not limited to the researchers and clinicians: Dr. Meyer – former Nationwide Children’s Hospital, now Alcyone Therapeutics and University of Missouri, Dr. Andrea Sierra Delgado and Dr. Smita Saxena – NextGen Precision Health Medical Center University of Missouri, Dr. Grinspan, Dr. Michael Kaplitt, Dr. Sara Strauss and the full team at Weill Cornell Medicine and New York Presbyterian Hospital as well as Alcyone Therapeutics, Unravel Biosciences, Jackson Labs, and La Jolla Labs; and the gene therapy manufacturers: Andelyn Biosciences, Genezen, & Charles River Laboratories. We also thank Canopy Biosciences and the N=1 collaborative for regulatory and writing support.
Important funding and support for this project was provided by our fellow partners and colleagues at Alvarez & Marsal and Ernst & Young, the RTW Foundation, the Reiter Family Foundation, Andelyn Fore One Purpose, Environment Mechanical, Lake Shore III Corporation/ Hiawatha National Bank, and all our friends, families and supporters. Elly’s Team has and will continue to use 100% of all funds toward scientific research and developing treatments for patients.
Further, we could not have accomplished this without the hundreds of people who have prayed for us daily- it matters!
Elly’s Team will host a webinar in the near future to share more details on our treatment path and next steps for the clinical trial. In order to receive further information about the webinar please register here: Elly’s Team Gene Therapy Webinar.
While we do not yet know whether this treatment will have the intended outcome, we place our trust and faith in God, and remain steadfast in our mission, knowing that we are all part of this journey together. We look forward to working together to find treatments for every child impacted by this disease!
Sincerely,
Michelle & Dan Krueger
Parents to Elly and Founders of Elly’s Team
Romans 12:12: Be joyful in hope, patient in affliction, faithful in prayer.
