NORD® Rare Diseases and Orphan Products Breakthrough Summit
2025-10-20 - 2025-10-21
Shape the future of rare disease treatments, research and policy at the NORD® Rare Diseases and Orphan Products Breakthrough Summit.® Together, we can advance equity and innovation for the more than 30 million Americans – and more than 400 million people worldwide – with rare diseases!
Washington DC
nordsummit.org

Molecular Therapy Presents: Advancing AAV Technology
2025-10-22 10:00 - 11:00 (America/Chicago)
“Advancing AAV Technology: From Capsid Design to Scalable Manufacturing” – An ASGCT Webinar
Virtual | Free

Breakthroughs in Targeted In Vivo Gene Editing
2025-11-20 - 2025-11-21
Join ASGCT at the Marriott Marquis San Diego Marina, Nov. 20-21, 2025, for an unparalleled opportunity to immerse yourself in cutting-edge solutions for targeted delivery of gene editing technology in vivo in San Diego. Hear from SCGE researchers Bill Peranteau, Sonia Vallabh, Yong-Hui Jiang, Kiran Musunuru, and Rebecca Ahrens-Nicklas at the event.
Virtual and In Person (San Diego, CA)
https://www.asgct.org/events/breakthroughs

Around the World: Technological Frontiers in South American Gene and Cell Therapy
2025-12-16 10:00 - 11:00
As gene and cell therapies revolutionize modern medicine, South America is emerging as a dynamic player on the global stage—leveraging scientific innovation, regulatory evolution, and regional collaboration. This free virtual program explores the technological frontiers shaping the development and deployment of advanced therapies across the continent.
Webinar | Free

Public Meeting: FDA Rare Disease Day 2026
2026-02-23 08:00 - 15:00
FDA will host Rare Disease Day, a virtual public meeting, on Monday, February 23, 2026 in global observance of Rare Disease Week. The theme is: “Moving Forward. Looking Ahead. An Event for Patients.” The goal of this year’s Rare Disease Day is to explore ways to engage and collaborate with patients and their communities to support and accelerate the development of medical products for rare diseases. We want patients to connect with FDA and stay involved with our outreach and engagement programs to ensure the patient voice informs medical product development and regulation throughout FDA. Panels will discuss:

Patient Focused FDA Initiatives
Patient Engagement Opportunities
Addressing Challenges and Opportunities with AI Technology
Utilizing Real-World Data and Real-World Evidence at FDA

More info at https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/public-meeting-fda-rare-disease-day-2026-02232026

Rare Disease Day at NIH
2026-02-27 08:00 - 16:00
Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect and NIH collaborations that address scientific challenges and advance research for new treatments.
The event will be held at NIH Main Campus (Natcher Conference Center), and there will also be a virtual livestream, with the event archived for replay afterward. The event is free and open to the public.
More info at: https://ncats.nih.gov/news-events/events/rdd

Empowering Patients 2026: A Cell and Gene Therapies Summit
2026-03-11 - 2026-03-12
This free, virtual summit from ASGCT will bring together patients, caregivers, advocates, researchers, and industry leaders for two dynamic half-days of learning the latest insights in cell and gene therapy.
Hear from SCGE researchers Steve Gray and Kiran Musunuru at the event.

9th Annual Gene Therapy Development Summit
2026-03-24 - 2026-03-26
The 9th Gene Therapy Development Summit returns to Boston as the meeting point for this community. Join 100+ leading voices from the likes of Bayer, Astellas Pharmaceuticals, REGENXBIO, Ocugen, AskBio, CureDuchenne Ventures, and more to come together to share ideas, restore confidence, and chart a sustainable path forward for gene therapy. Connect with your peers in pre-clinical development, manufacturing, analytical development, clinical operations, and regulatory affairs for an unrivalled learning and networking opportunity across the end-to-end of gene therapy development.
Boston, MA

NORD® Rare Disease Scientific Symposium 2026
2026-04-14 - 2026-04-15
The NORD® Rare Disease Scientific Symposium is bringing together a dynamic community of researchers, clinicians, industry leaders, and advocates to spark the conversations and connections that drive science forward.
This immersive, 1.5 day event is designed to push boundaries and bridge gaps across the rare disease landscape. Through engaging case studies, interactive panel discussions, and extended audience Q&A, participants will explore critical topics such as:

Innovative Study Designs and Methods for Rare Disease Research
Approaches to Address the Challenges of Small Trial Enrollment and Disease Heterogeneity
Successes and Challenges of Global Clinical Trials
Using Alternative and Real-World Data for Rare Disease Research
Multi-Stakeholder Collaboration in Research
Strategies for Earlier Pediatric Inclusion in Clinical Trials

Learn more at: https://nordscience.org/

ISCT 2026
2026-05-06 - 2026-05-09
International Society for Cell & Gene Therapy 2026 Annual Meeting
Dublin, Ireland
https://www.isctglobal.org/meetings/annual-meeting/isct-2026-dublin

ASGCT 2026 Annual Meeting
2026-05-12 - 2026-05-16
Boston, MA or Virtual
https://annualmeeting.asgct.org/

World Orphan Drug Congress USA 2026
2026-06-09 - 2026-06-11
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.
Boston, MA

CRISPR 2026
2026-06-10 - 2026-06-12
The CRISPR meeting has a distinguished history. It was the first meeting focusing on the emerging topic of CRISPR-Cas. It was at this podium that researchers shared their groundbreaking discoveries on CRISPR-Cas and witnessed the birth of the genome editing revolution.
SCGE researcher Benjamin Kleinstiver is scheduled to be speaking at the event.
Omni New Haven Hotel at Yale
Learn more: https://web.cvent.com/event/ac20b6f2-79c9-4ab7-87ae-f650579f4e42/summary?environment=P2

3rd Global Cell & Gene Therapy Summit
2026-07-06 - 2026-07-08
This three-day conference aims to bring together a global audience to discuss and exchange ideas on the origins, development, clinical applications, and translational science of cell and gene therapy.
Boston, MA
