June 9, 2026 | Workshop Day
June 10-11, 2026 | Conference & Exhibition

Boston Convention & Exhibition Center | Boston, MA

America's Largest Orphan Drug & Rare Disease Event

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280+

Speakers

2,000+

Attendees

100+

Exhibitors

16

Streams of Content

Breaking barriers & fostering collaboration to help the rare disease community

The World Orphan Drug Congress USA brings together the leading pharmaceutical and biotechnology companies, government and regulators, patient advocates, payers, investors, and solution providers – all who all depend on our event to convene and brainstorm ways to advance

Orphan Drug development and improve access to life-saving therapies for rare disease patients.

Join the world’s defining event for the Orphan Drug sector and rare disease community June 9-11, 2026.

SECURE YOUR SPOT – REGISTER NOW

This is your chance to explore the industry’s latest innovations and tech + catch the most up-to-date, keynote-driven content.

Your ticket provides access to the premium conference, exhibition and networking opportunities with thousands of industry leaders

THE CONFERENCE

Hear about the latest developments in the rare disease and orphan drug space from top thought leaders in the industry.

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THE EXHIBITION

Discover innovative products and solutions revolutionizing the future of rare disease.

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NETWORKING

Connect with fellow rare disease and orphan drug advocates from around the world – all with a shared passion to improve the lives of rare disease patients.

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Sponsor the World Orphan Drug Congress USA

Get your product or solution in front of the leaders in the rare disease industry — from pharma, biotechs, governments, payers, investors and patient/patient advocates.

The World Orphan Drug Congress USA is a place where innovation and expertise is showcased, solutions are found and learning is done.

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pharma, biotechs, governments, payers, investors and patient patient advocates.

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2026 Speakers

Mackenzie Abramson, MPH, BCPA
Patient Advocate

Rebecca Ahrens-Nicklas, MD, PhD
Assistant Professor of Pediatrics Director, Gene Therapy for Inherited Metabolic Disorders Frontier Program
Children's Hospital Of Philadelphia
Lynn Albizo
Chief Public Policy Officer
Immune Deficiency Foundation

Annette Bakker
CEO
Childrens Tumor Foundation
Anny Bedard
President
Egetis Therapeutics US

Hourinaz Behesti
Founder & CEO
Hebbian Bio
Christine Brennan
Managing Director
Vertex Ventures HC

P.J. Brooks
Deputy Director, Division of Rare Diseases Research Innovation
NIH/NCATS
Cynthia Cassandro
Vice President, Patient Advoacy
Vigil Neuroscience

James Chambers
Professor
Tufts Medical Center
Jason Colquitt
Chief Executive Officer
Across Healthcare

Rumi Desai
Director, Global Regulatory Strategy
Amgen, Inc.
Wendy Erler
Senior Vice President, Patient Affairs
Sarepta Therapeutics

Djordje Filipovic
Chief Executive Officer
AB2 Bio Ltd.
Karen Frascello
Senior Director, Medical Affairs
Alnylam Pharmaceuticals

Albert Freedman
Counseling Psychologist / Rare Dad
Rarecounseling.com
Chris Garabedian
Portfolio Manager, Venture
Perceptive Advisors

Federico Goodsaid
SVP Regulatory
Ariana Pharma
Rachel Harrison
Director, Expanded Access Program
argenx

Neil Inhaber
Head, Marketed Products, Global Medical
Takeda
Debra Jennings
Head of Patient Services Operations, North America
Kyowa Kirin

Richie Kahn
Co-Founder & COO
Canary Advisors
Patroski Lawson
Co-founder
The Just Society

Kimberly LeBlanc
Director, UDN Data Management Coordinating Center, Harvard Medical School
Harvard Medical School
Julianna LeMieux
Deputy Editor in Chief
Genetic Engineering & Biotechnology News

Robert Long
Executive Director
Uplifting Athletes
Peng Lu
Chief Medical Officer
Pharvaris

Fabiana Marzabal
Sr Director Forecasting, Analytics and Insights
Viridian Therapeutics
Maurizio Mazzi
Senior Associate Director, Patient Advocacy & Professional Relations
Böhringer Ingelheim

Jennifer McNary
Co-Founder
Canary Advisors
Kiran Musunuru, M.D., Ph.D.
Barry J. Gertz Professor for Translational Research
Perelman School of Medicine University of Pennsylvania

Brian Nappi
SVP, Strategy
Saol Therapeutics
Francis Pang
SVP Global Market Access and International Geographic Expansion
Orchard Therapeutics

Dave Pearce
Chair
International Rare Diseases Research Consortium (IRDiRC)
Dorian Readnour
Vice President, International
Sarepta Therapeutics

Frank Rivera
President
Stronger Than Sarcoidosis
Luis Rojas
Chief Clinical Development Officer
Polaryx Therapeutics

Devyn Smith
CEO
Arbor Biotechnologies
Frank Sorgi
President and Chief Executive Officer
FLAG Therapeutics

Steve St. Onge
SVP - Corporate Development
Clarametyx Biosciences
Dionne Stalling
Executive Director
Rare And Black

Ying Tam
Chief Scientific Officer
Acuitas Therapeutics
Chelsea Trengrove
CEO and Cofounder
Neoclease

Daniel Wainstock
Researcher and Rare Disease Advocate
PUC-Rio
Durhane Wong-Rieger
President, Chief Executive Officer
Canadian Organisation for Rare Disorder & Rare Diseases International

Graeme Ladds
CEO
PharSafer
Charlene Son Rigby
Chief Executive Officer
Global Genes

Fyodor Urnov
Scientific Director, Innovative Genomics Institute, Professor, Department of Molecular & Cell Biology
University of California at Berkeley
Jessica Cerullo
Senior Director, Patient Advocacy & Disease States Initiatives
Pierre Fabre Pharmaceuticals

Anne Corvelle
President, Board of Directors
FD/MAS Alliance
Amanda Forys
Managing Partner
Magnolia Market Access

Sonit Handa
CEO & Founder
Biovance Consulting

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The Start-to-Finish of Orphan Drugs
From regulation and policy to global pricing and gene therapy

PATIENT DATA

With so few patients, patient data/real-world-data (RWD/RWE) is the key element to advancing the rare disease space. What are best practices right now for data collection, ownership and sharing to benefit rare disease diagnosis, drug development and access.

NEXT GENERATION THERAPIES

The rare disease space is a breeding ground for innovative approaches in drug development and platform technologies. What are the products and approaches that will be making headlines in 5-10 years from now?

payers PAYERS

PAYERS

US-based insurers and single payer agencies around the world have a lot of impact of access and reimbursement for orphan products. Hear from top US and international payers on their strategies for orphan drugs.

CLINICAL DEVELOPMENT & REGULATORY

Understanding the regulatory and clinical landscape for rare disease drug development. How can companies, regulators and patient advocates work together to further patient voice, real world evidence, and decentralized clinical trials.

COMMERCIAL

What are the best commercial strategies for orphan products? How can companies bring products to new markets, build more efficient commercial operations, and develop effective launch strategies?

RARE DISEASE ADVOCACY WORLD

It all starts and ends with patients. So what are the challenges facing rare disease patients and patient organizations today? How can rare disease patient groups rethink funding strategies, partnerships with industry and advance policies around access?

global marketing & patient access GLOBAL MARKET & PATIENT ACCESS

GLOBAL MARKET & PATIENT ACCESS

Access is often the crucial and challenges last step for rare disease patients and developers. How can all stakeholders work together to ensure rare disease patients have access to appropriate treatments globally.

PRICING & REIMBURSEMENT

Pricing orphan products, especially one-time treatments, is complex and a key element to ensuring timely access for rare disease patients. What are the current best practices for pricing orphan products, negotiating innovative contracts and working with payers around the world?

PITCH & PARTNER

The orphan drug space is booming with innovation, M&A activity and partnerships. We play match maker for up and coming biotechs in the rare space and investors & pharma partners looking for their next blockbuster.

advanced therapies manufacturing ADVANCED THERAPIES - MANUFACTURING

ADVANCED THERAPIES - MANUFACTURING

What are the current roadblocks in manufacturing cell and gene therapies and how can companies efficiently scale-up for commercial production?

advanced therapies clinical development ADVANCED THERAPIES - CLINICAL DEVELOPMENT

ADVANCED THERAPIES - CLINICAL DEVELOPMENT

The regulatory and clinical development landscape is ever evolving for cell and gene therapies. How are companies ensuring continued development of these potentially transformative products and what clinical hurdles remain?

RARE ONCOLOGY

As the rare cancer field evolves and diagnosis becomes more precise, how can all stakeholders work together to advance drug development for rare cancer patients?

advanced therapies commercialization ADVANCED THERAPIES - COMMERCIALIZATION

ADVANCED THERAPIES - COMMERCIALIZATION

Rare disease patients are uniquely positioned to benefit from the advancements of cell and gene therapies. What are the best strategies for commercializing these innovative products and how do health systems need to evolve to accommodate patient access and reimbursement?

DIGITAL HEALTH & ARTIFICIAL INTELLIGENCE

Approaches in AI and digital health span the full development life cycle of drug development and commercialization. What are the best approaches to implementing these technologies in patient finding, clinical development and commercialization for rare diseases?

diagonis DIAGNOSIS

DIAGNOSIS

It takes an average of seven years for an accurate diagnosis. How can all stakeholders broaden access to Newborn Screening, Whole Genome Sequencing and Genetic testing to shorten the diagnostic odyssey for rare disease patients?

DISCOVERY

Technologies and processes are ever. How is the growing access to data, and use of AI and machine learning impacting drug discovery in the rare disease space?

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COMPLIMENTARY VIP ACCESS for
Patient Groups, Healthcare workers & Investors!

Apply for your free pass below to secure your spot

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Networking Is Our Sweet Spot

By App. By Facilitated Meetings. By RoundTables. By Hangin’ Out.

So you can: Exchange ideas, Build brand, Form friendships and partnerships, Grow your professional network, Explore the future, Uncover new opportunities.

FIND OUT MORE

"If there is one event you want to go to as a stakeholder in rare diseases, it is the World Orphan Drug Congress USA. This conference has helped to define the world of rare diseases, it’s not just a leading event, it has actually helped create the future for rare diseases"

President And Chief Executive Officer, Canadian Organization For Rare Disorders

"The support, kindness, courtesy and interest shown by WODC Team in our company’s drug development program for orphan disease glioblastoma, to aid us to secure funding to run registration trials"

Chief Executive Officer, Tactical Therapeutics Inc

America's Largest Orphan Drug & Rare Disease Event

280+

2,000+

100+

16

Breaking barriers & fostering collaboration to help the rare disease community

This is your chance to explore the industry’s latest innovations and tech + catch the most up-to-date, keynote-driven content.

Your ticket provides access to the premium conference, exhibition and networking opportunities with thousands of industry leaders

Sponsor the World Orphan Drug Congress USA

Past Sponsors & Exhibitors

2026 Speakers

The Start-to-Finish of Orphan Drugs
From regulation and policy to global pricing and gene therapy

COMPLIMENTARY VIP ACCESS for
Patient Groups, Healthcare workers & Investors!

Apply for your free pass below to secure your spot

Networking Is Our Sweet Spot

Join us in Boston

June 9-11, 2026

Get Involved At World Orphan Drug Congress USA

Connect with us

To Sponsor Or Exhibit

Jack Rendell
jack.rendell@terrapinn.com
t/ +44 07577689029

To Speak

Kaylee Nguyen
kaylee.nguyen@terrapinn.com

Marketing & Press

Jasper Cameron
jasper.cameron@terrapinn.com

Created by

America's Largest Orphan Drug & Rare Disease Event

280+

2,000+

100+

16

Breaking barriers & fostering collaboration to help the rare disease community

This is your chance to explore the industry’s latest innovations and tech + catch the most up-to-date, keynote-driven content.

Your ticket provides access to the premium conference, exhibition and networking opportunities with thousands of industry leaders

Sponsor the World Orphan Drug Congress USA

Past Sponsors & Exhibitors

2026 Speakers

The Start-to-Finish of Orphan Drugs From regulation and policy to global pricing and gene therapy

COMPLIMENTARY VIP ACCESS forPatient Groups, Healthcare workers & Investors!

Apply for your free pass below to secure your spot

Networking Is Our Sweet Spot

Join us in Boston

June 9-11, 2026

Get Involved At World Orphan Drug Congress USA

Connect with us

To Sponsor Or Exhibit

Jack Rendell jack.rendell@terrapinn.com t/ +44 07577689029

To Speak

Kaylee Nguyen kaylee.nguyen@terrapinn.com

Marketing & Press

Jasper Cameron jasper.cameron@terrapinn.com

Created by

The Start-to-Finish of Orphan Drugs
From regulation and policy to global pricing and gene therapy

COMPLIMENTARY VIP ACCESS for
Patient Groups, Healthcare workers & Investors!

Jack Rendell
jack.rendell@terrapinn.com
t/ +44 07577689029

Kaylee Nguyen
kaylee.nguyen@terrapinn.com

Jasper Cameron
jasper.cameron@terrapinn.com