RTW
RTW Foundation Works with Elly's Team to Achieve One of the Fastest Gene Therapy Development Efforts in History
On March 19, 2025, the U.S. Food and Drug Administration (FDA) approved an Investigational New Drug (IND) application to allow the use of an experimental gene replacement therapy in a NEDAMSS patient, Elly Krueger. This clearance came just 13 months after Elly’s diagnosis, marking an unusually rapid timeline in the context of gene therapy development. Elly's parents, Michelle and Dan Krueger, connected with the rare disease advisory team at RTW Foundation in March 2024, and on April 3, 2025, Elly Krueger became the first child to receive an IRF2BPL gene replacement therapy.
01 Rare Disease
Supporting scientific initiatives to aid in the development of medicines for neglected rare diseases
There are over 10,000 rare diseases, yet less than 5% of them have effective treatments. We collaborate with research teams, families, and advocacy organizations from around the world to work towards treatment for these conditions.
02 Community
Building partnerships with local organizations to advance health equity in New York City
We are committed to supporting underserved communities. We build partnerships with community-based organizations to respond to the needs of our neighbors.
BioQuest: Empowering Future Innovators
BioQuest is our STEM mentoring program designed to give middle and high school students opportunities to explore careers in science, biotechnology, and medicine. Click below to explore more about the program and the students from our inaugural year!
About us
Our mission is to power community initiatives and scientific research to improve the health of underserved populations.
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Contact us for information on open grants and how to apply
