14 Months to Treatment: Breaking Records in Rare Disease Therapy

From a Thanksgiving night emergency to one of the fastest gene therapies ever deployed, this is the story of a family’s relentless pursuit to help their daughter, and how RTW Foundation made it possible.This episode was recorded on June 11, 2025, and the clinical trial details discussed may have changed; please see ellysteam.org for the most current information on Elly’s case.PODCAST TEAMProducer & Director: Devon LeaverProduction Coordinator: YingYu LinEditor: Dominique GuerraResearch Consultants: Joe Katakowski, PhD, and Nicole LittStory Consultant: Samantha DarrisTheme music: Ganymede by Yehezkel RazAdditional music: APM MusicFeaturing: Michelle Krueger, Managing Director at Alvarez & Marsal Transaction Advisory Group, and the co-founder of Elly’s Team, moderated by Stephanie A. Sirota, Partner, Chief Business Officer at RTW Investments, LP.FOLLOW USNow you can follow along in the format you prefer. Watch the video feed of this podcast on YouTube or read the transcript at rtwfunds.com/podcasts.To make sure you never miss an episode, you can subscribe wherever you get your podcasts. Follow us on LinkedIn at RTW Investments, LP and check out our website for the latest updates and insights.CHAPTERS00:00 - Introduction01:16 – Elly’s Diagnosis04:37 - Finding a treatment with RTW Foundation09:42 - Elly’s journey: from research to treatment14:50 - Becoming the first child to receive an IRF2BPL gene replacement therapy17:17 - Expanding the clinical trial for NEDAMSS19:12 - How to improve drug research and development21:18 - Outro and disclaimerDISCLAIMERThis interview was given by Michelle Krueger, Managing Director at Alvarez & Marsal Transaction Advisory Group, and the co-founder of Elly’s Team, and moderated by Stephanie A. Sirota, Partner and Chief Business Officer at RTW Investments, LP. Statements reflect RTW's views and opinions as of the date hereof and not as of any future date. All expressions of opinion are subject to change without notice and are not intended to be a forecast of future events or results. The views expressed by guests are their own, and their appearance on the program does not imply an endorsement of them or any entity they represent.