Rare disease at Meeting on the Mesa 2026
71 organizations at Meeting on the Mesa 2026 present work in rare disease: 55 therapy developers, 9 academic, hospital and public organizations, 6 suppliers and service providers and 1 investor. Between them they bring 49 presentations to the program. Most represented: Arbor Biotechnologies (1 on the program), Children’s National Hospital (1 on the program), CTI Clinical Trial & Consulting Services (1 on the program). 47 of them have their own page in this guide.
Format
Top represented
Showing 1–17 of 71 organizations
| Organization | Meeting on the Mesa 2026 Attendance |
|---|---|
Arbor Biotechnologies Cambridge, MA | ◇Silver Sponsor |
Develops gene-editing therapeutics for genetic diseases, including liver and central nervous system conditions and rare disorders. | Spotlight Mon, Oct 5 10:45am Innovation Spotlight company presentation Panel Wed, Oct 7 4:15pm |
Children’s National Hospital Washington, DC | ◇Silver Sponsor |
Provides pediatric hospital care and develops treatments for genetic and rare diseases through clinical and translational research. | Spotlight Mon, Oct 5 5:15pm Innovation Spotlight company presentation Panel Wed, Oct 7 10:45am |
CTI Clinical Trial & Consulting Services Covington, KY | ◆Diamond Sponsor |
Provides clinical research, regulatory consulting, site management, and laboratory services for biopharmaceutical trials, including cell and gene therapies and rare diseases. | Spotlight Mon, Oct 5 3:00pm Innovation Spotlight company presentation |
Precision for Medicine Bethesda, MD | ◆Platinum Sponsor |
Provides biomarker-driven CRO services, clinical trials, specialty laboratories, biospecimens, data science, and companion diagnostic support. | Spotlight Mon, Oct 5 12:00pm Innovation Spotlight company presentation |
REGENXBIO Rockville, MD | ◇Silver Sponsor |
Develops AAV gene therapies for retinal and rare diseases, including programs for wet AMD, diabetic retinopathy, Duchenne muscular dystrophy, and MPS II. | Spotlight Mon, Oct 5 11:00am Innovation Spotlight company presentation |
Rocket Pharmaceuticals, Inc. Cranbury, NJ | ◇Silver Sponsor |
Develops gene therapies for rare cardiovascular diseases, with additional programs in hematology and immunology and proprietary AAV manufacturing capabilities. | Spotlight Mon, Oct 5 11:30am Innovation Spotlight company presentation Panel Wed, Oct 7 |
uniQure Amsterdam, Netherlands | ◇Silver Sponsor |
Develops one-time gene therapies for hemophilia, Huntington’s disease, epilepsy, Fabry disease, and other severe conditions. | Spotlight Mon, Oct 5 3:15pm Innovation Spotlight company presentation |
Ocugen Malvern, PA | ◇Silver Sponsor |
Develops gene and cell therapies for inherited retinal diseases, blindness and other serious conditions. | Panel Wed, Oct 7 |
BridgeBio Palo Alto, CA | ◇Silver Sponsor |
Develops medicines for genetic conditions through autonomous teams focused on underserved and rare diseases. | Sponsor In the meeting’s sponsor directory, with no session on the public program |
EveryLife Foundation Washington, DC | ◇Silver Sponsor |
Advocates for policies and research funding that improve diagnosis, treatment, and access for people with rare diseases. | Sponsor In the meeting’s sponsor directory, with no session on the public program |
◇Silver Sponsor | |
Develops and commercializes therapies for cancer, transplant-related disease, and rare genetic disorders. | Sponsor In the meeting’s sponsor directory, with no session on the public program |
Premier Research Durham, NC | ◆Gold Sponsor |
Provides clinical development, trial management, regulatory, and market-access services, with expertise in rare diseases and complex therapies. | Sponsor In the meeting’s sponsor directory, with no session on the public program |
○Event Supporter | |
Supports children and families affected by rare diseases through Canadian patient advocacy, research connections, and care resources. | Sponsor In the meeting’s sponsor directory, with no session on the public program |
Solid Biosciences Charlestown, MA | ◇Silver Sponsor |
Develops genetic medicines for Duchenne muscular dystrophy and other serious diseases, including gene therapies and genetic medicines for cardiac conditions. | Sponsor In the meeting’s sponsor directory, with no session on the public program |
Patient advocacy nonprofit advancing therapies for Angelman syndrome and other rare monogenic neurodevelopmental disorders through venture philanthropy. | Spotlight Mon, Oct 5 9:45am Innovation Spotlight company presentation Panel Wed, Oct 7 3:30pm |
Skylark Bio Cambridge, MA | |
Develops locally delivered gene therapies for monogenic diseases, including genetic hearing loss and CNS disorders. | Spotlight Tue, Oct 6 2:45pm Innovation Spotlight company presentation Emerging Track Workshop Wed, Oct 7 |
Develops allogeneic CAR-iNKT cell therapies for blood cancers and solid tumors, including its lead CD19-targeting program ALA-101. | Spotlight Tue, Oct 6 10:00am Innovation Spotlight company presentation |
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