September 14-16, 2026

2026 Rare Trials Summit

InterContinental Boston

The Rare Disease Drug Development Summit

The US meeting for clinical development, regulatory, operations, evidence and patient leaders responsible for advancing rare disease therapies.

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A conference hall during the 2025 Rare Trials Summit with a large audience, a stage with two presenters, and screens displaying sponsor logos.
Panel of six patient advocacy leaders and a moderator, sitting on stage at the 2025 Rare Trials Summit, with Pat Furlong, on the far right speaking into a microphone, dark curtains backdrop, blue-themed stage with a banner displaying the event logo.
Exhibit Hall during lunch time, with round tables, attendees talking, and large vendor exhibits in the background.
2025 Session Videos
People sitting at round tables in a conference room, engaging in conversations, with a woman in a teal blazer speaking to a woman with dark hair, a flower arrangement on the table, and attendees listening or drinking coffee.

2026 Steering Committee

  • Han Phan, MD

    Founder & CEO, Rare Disease Research

  • Miro Pastrnak, PhD

    COO, Gooseberry Research

  • Annie Kennedy

    Chief of Policy, Advocacy, and Patient Engagement, EveryLife Foundation for Rare Diseases

  • Patricia Furlong

    President & Founder, Parent Project Muscular Dystrophy

  • Craig Lipset, MPH

    Co-Chair, Decentralized Trials & Research Alliance (DTRA)

  • Raymond A. Huml, MS DVM RAC

    CRO Executive, FSHD Advocate and Author

  • Sandeep Menon, PhD

    Chief Research and Development Officer, BioCryst Pharmaceuticals

  • Sam Hopkins, PhD

    SVP, Therapeutics, AskBio, part of Bayer AG

  • David Jacoby, MD PhD

    SVP Head of Clinical Development, Ultragenyx

  • Thomas Defay, PhD

    Deputy Head Diagnostics Strategy & Development, Alexion Pharmaceuticals

  • Ebony Dashiell-Aje, PhD

    Executive Director and Head, Patient Centered Outcomes Science, BioMarin Pharmaceutical

  • Wendy Erler, MBA

    SVP, Patient Affairs, Sarepta Therapeutics

  • Michael Binks, MD

    Chief Medical Officer, Capricor Therapeutics

  • Dan Levy, MD PhD

    Chief Medical Officer, Torque Bio

  • Karin Hoelzer, DVM PhD

    Senior Director, Patient Advocacy, Biotechnology Innovation Organization

  • Deb Kientop, MBA

    SVP, Strategic Solutions and Patient Centricity, ERGOMED

  • Patti Shugarts, MHI

    VP, Rare Disease Pillar Head, Neuroscience, PPD ThermoFisher

  • Rachel Smith

    VP, Rare and Genetic Disease, Parexel

  • Brandon Henry, MD FACSc IFAPP Fellow

    Founder & CEO, Aurelix

  • Angi Robinson

    SVP, Specialty Areas Business Unit (Rare Disease, Pediatrics, CGT), Premier Research

  • Richie Kahn

    Owner, Canary Advisors

  • Iwona Tongbhoyai, MSc

    Chief Client Solutions Officer, FutureMeds

  • Holly Peay, PhD

    Senior Director, Faegre Drinker Consulting

  • Jason Colquitt

    Founder & CEO, Across Healthcare

Sample 2025 Session Video:

Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials