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 TEAM Producer & Director: Devon Leaver Production Coordinator: YingYu Lin Editor: Dominique Guerra Research Consultants: Joe Katakowski, PhD, and Nicole Litt Story Consultant: Samantha Darris Theme music: Ganymede by Yehezkel Raz Additional music: APM Music Featuring: 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 US Now 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. CHAPTERS 00:00 - Introduction 01:16 – Elly’s Diagnosis 04:37 - Finding a treatment with RTW Foundation 09:42 - Elly’s journey: from research to treatment 14:50 - Becoming the first child to receive an IRF2BPL gene replacement therapy 17:17 - Expanding the clinical trial for NEDAMSS 19:12 - How to improve drug research and development 21:18 - Outro and disclaimer DISCLAIMER This 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.