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.

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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.

14 Months to Treatment: Breaking Records in Rare Disease Therapy

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14 Months to Treatment: Breaking Records in Rare Disease Therapy