September 14-16, 2026

Rare Trials Summit

InterContinental Boston

Bringing together key stakeholders in the clinical research community to accelerate the development of new 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.
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

  • Raymond A. Huml, DVM

    VP and Head of Rare Disease Strategy. Sciensus

  • Richie Kahn

    Owner, Canary Advisors

  • Sandeep Menon, PhD

    Chief Development Officer, Alnylam 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

  • Karin Hoelzer, DVM PhD

    Senior Director, Patient Advocacy, Biotechnology Innovation Organization

  • Sy Pretorius, MD MBA

    CEO, Ergomed

  • Patti Shugarts, MHI

    VP, Rare Disease Pillar Head, Neuroscience, PPD ThermoFisher

  • Rachel Smith

    VP, Rare and Genetic Disease, Parexel

  • Brandon Michael Henry, MD FACSc FAPCR IFAPP Fellow

    Medical Director, Cell & Gene Center of Excellence, IQVIA

  • Angi Robinson

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

  • Holly Peay, PhD

    Senior Director, Faegre Drinker Consulting

  • Jason Colquitt

    Founder & CEO, Across Healthcare

  • Dan Levy, MD PhD

    Founder, ATCG Rare

Sample 2025 Session Video:

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