Company lists · Data refreshed September 2026
Top 25 Rare disease research organizations based in the United States
RevenueBase identified 353 rare disease research organizations headquartered in the United States as of September 2026 and tracks 15,936 professional contacts across them, including 9,308 with verified work emails. Washington is the largest hub.
This list covers rare disease research organizations headquartered in the United States, matched on what each company actually does as described in its own words. Ranked by team size, largest first. Every row shows the company's own description, its RevenueBase Smart Search rating, and when it was last checked. Data refreshed September 21, 2026.
Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase Smart Search, the natural-language company search RevenueBase customers use to build their own targeted lists and unlock verified emails and direct dials at the companies on them. How these lists are built and judged.
The 25 largest rare disease research organizations based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 6,136 contacts → | ||||
Strong evidence “Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 2 | 1,556 contacts → | ||||
Indirect evidence “Leading with purpose.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 3 | 1,212 contacts → | ||||
Strong evidence “PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 4 | 1,043 contacts → | ||||
Partial evidence “CTI Clinical Trial and Consulting Services is a global, privately held, research service organization, delivering a complete spectrum of clinical trial and consulting services…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 5 | 603 contacts → | ||||
Strong evidence “Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 6 | 582 contacts → | ||||
Strong evidence “PANTHERx Rare makes rare disease care more hyper-personalized and less overwhelming by focusing relentlessly on each patient and each therapy.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 7 | 491 contacts → | ||||
Strong evidence “Our mission is to identify, develop and deliver life-changing therapies to people living with rare disease.#InRareForLife Community Guidelines…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 8 | 326 contacts → | ||||
Partial evidence “Sobi is an international specialty healthcare company dedicated to developing treatments and extensive patient support networks for people with rare and difficult to treat…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 9 | 185 contacts → | ||||
Indirect evidence Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 10 | 168 contacts → | ||||
Strong evidence “Bionews is a digital health solutions company dedicated to empowering more than 50 rare disease communities with trusted information, news, and connections.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 11 | 156 contacts → | ||||
Strong evidence “The National Organization for Rare Disorders (NORD), a 501(c)(3) organization, is an independent patient advocacy organization dedicated to helping individuals with rare diseases…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 12 | 150 contacts → | ||||
Partial evidence “The urgent mission of the Foundation Fighting Blindness is to drive the research that will provide preventions, treatments and cures for people affected by retinitis pigmentosa…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 13 | 134 contacts → | ||||
Partial evidence “At Pinnacle Clinical Research, we are driven by a shared mission to improve lives through innovation in clinical trials.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 14 | 112 contacts → | ||||
Partial evidence “HonorHealth Research Institute is an international destination that is at the forefront of providing patients with a better quality of life through access to clinical trials and…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 15 | 84 contacts → | ||||
Partial evidence “We are a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 16 | 81 contacts → | ||||
Strong evidence “The EveryLife Foundation for Rare Diseases is powered by the rare disease community to improve health outcomes by driving change through evidence-based policy, leading…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 17 | 78 contacts → | ||||
Indirect evidence “We are a purpose-driven, commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 18 | 65 contacts → | ||||
Strong evidence “ITF Therapeutics is the US-based rare disease division of Italfarmaco S.p.A.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 19 | 63 contacts → | ||||
Partial evidence “This is a time of great hope for individuals with complex diseases such as NF1 and their families.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 20 | 61 contacts → | ||||
Partial evidence “At Citizen Health, we have a singular mission: to improve the lives of the 400+ million people navigating rare and complex conditions.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 21 | 59 contacts → | ||||
Strong evidence “At SCYNEXIS, we are committed to improving outcomes for patients living with severe, progressive, and underserved rare diseases.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 22 | 56 contacts → | ||||
Partial evidence “GEMMABio, a new therapeutics company, will serve as the research and innovation arm to speed the research of and global access to life-changing advanced therapies for those living…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 23 | 56 contacts → | ||||
Indirect evidence “Ask us what we did today, and we'll tell you that we worked hard to change someone's life.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 24 | 51 contacts → | ||||
Indirect evidence Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 25 | 50 contacts → | ||||
Strong evidence “As an independent clinical research site, we are 100% dedicated to conducting clinical research, accelerating the development of safe and effective treatments and providing rare…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
RevenueBase Smart Search
RevenueBase Smart Search assesses information from a company’s LinkedIn profile, information supplied directly by the company, its website, and other publicly available sources. It uses that information to rate how closely the company relates to this list’s category: rare disease research organizations. Every row carries its rating and the description the rating was read from, so each one can be checked rather than taken on trust.
- Strong evidence
- The company's own public description of its business names rare disease research organizations.
- Partial evidence
- Its own public description names part of rare disease research organizations, so it works in or next to the category without describing itself that way.
- Indirect evidence
- The description shown does not name rare disease research organizations. The match rests on the rest of the company's public profile rather than the one sentence quoted here, so this is the rating to check first.
RevenueBase Smart Search is how these lists are built: RevenueBase's data engine and AI assemble the companies that match a category defined in a user's own words, so a list can be about what companies actually do rather than about whichever industry code they were filed under.
Showing 25 of 353 rare disease research organizations based in the United States
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Rare disease research organizations based in the United States at a glance
As of Sep 21, 2026Companies by employee count
Email recency for this list is measured worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 353 companies.
How this list was built
Company categories are rarely black and white. RevenueBase reads each company's own description of its business to decide whether it belongs among rare disease research organizations, so some rows will be adjacent to the category rather than pure plays. The description and RevenueBase Smart Search rating on every row are there so you can check each match yourself.
- Source
- The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase Smart Search, the natural-language company search available to every RevenueBase user.
- What qualifies
- Companies headquartered in the United States whose own description of what they do matches “rare disease research organizations”. Matching reads each company's description, not its industry code or its name.
- RevenueBase Smart Search
- RevenueBase Smart Search is how this list is assembled and how each row is rated. RevenueBase's data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to a category defined in a user's own words. Each row is rated strong evidence (its own description names rare disease research organizations), partial evidence (it names part of the category) or indirect evidence (the description shown names none of it and the match rests on the rest of the company's profile). The description supporting each rating is quoted on the row.
- What we exclude
- Companies whose own description does not clearly place them among rare disease research organizations, and records with no verified work email. Companies headquartered outside United States are excluded even when they keep an office there.
- Match quality
- In blind human judging of this matching method, 86% of matched companies were on target. Expect roughly 1 in 7 rows to be adjacent to the category rather than a pure play. Every row shows the company's own description and its RevenueBase Smart Search rating so you can judge each match yourself. Read the full methodology: how the lists are built, how the judging worked, and what the figure means.
- Ranking
- Team size, largest first: companies are ordered by the number of professionals RevenueBase tracks at each one. Employee ranges shown are reported bands, for context.
- Verification
- Every listed company carries at least one verified work email, 9,308 across the full list. Contacts re-verify on a rolling cadence, and each contact's own verification date is shown inside RevenueBase.
- Refresh
- This page's figures were pulled from the company graph on September 21, 2026. Lists refresh periodically as the graph is re-checked.
- Corrections
- Wrong category, wrong headquarters, or a company that has closed? Use “Report an issue” on any row. Reports go to the RevenueBase Data Team and feed back into the company graph.
Frequently asked
What is RevenueBase Smart Search?
RevenueBase Smart Search is how RevenueBase decides which companies belong on this list and how strongly each one fits. Its data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to the category the list is about. Each row is rated strong evidence when the company's own description names rare disease research organizations, partial evidence when it names part of the category, and indirect evidence when the description shown names none of it and the match rests on the rest of the company's profile.
How many rare disease research organizations are there based in the United States?
RevenueBase identified 353 rare disease research organizations headquartered in the United States as of September 2026, matched on each company's own description of what it does rather than its industry code.
What is the largest company on this list?
Alexion Pharmaceuticals, INC, headquartered in Boston, tops this list with the largest team on record.
Which city has the most rare disease research organizations?
Washington leads with 17 headquartered companies, followed by New York (14) and Boston (13).
How do I get contact information for these companies?
A free RevenueBase account lets you run this exact search and work the top results, verified emails and direct dials included. Upgrading unlocks all 9,308 verified decision-maker contacts tracked across these 353 companies.
How current is this list?
The list refreshes periodically as the company graph is re-checked. This page's figures were pulled on September 21, 2026.
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Rare disease research organizations by geography
- Rare disease research organizations based in Australia
- Rare disease research organizations based in Canada
- Rare disease research organizations based in France
- Rare disease research organizations based in Italy
- Rare disease research organizations based in the Netherlands
- Rare disease research organizations based in Spain
- Rare disease research organizations based in the United Kingdom
About this data
RevenueBase is a B2B data infrastructure company. Its company graph covers 60 million companies and 398.9 million professional contacts, with emails verified on a rolling cadence rather than scraped once and left to decay. These company lists are generated directly from that graph with RevenueBase Smart Search, the same natural-language company search every RevenueBase user has: describe a market in plain words, get the companies that match, and add verified emails and direct dials for the people at them. It is the same data that powers RevenueBase's B2B data products, and a free account includes 500 credits to start. How these lists are built and judged.
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- Rare disease research organizations based in Australia
- Rare disease research organizations based in Canada
- Rare disease research organizations based in France
- Rare disease research organizations based in Italy
- Rare disease research organizations based in the Netherlands
- Rare disease research organizations based in Spain