Company lists · Data refreshed September 2026
Top 25 Rare disease research organizations worldwide
RevenueBase identified 1,156 rare disease research organizations worldwide as of September 2026 and tracks 26,139 professional contacts across them, including 11,975 with verified work emails. The United States is the largest market and Paris the largest hub.
This list covers rare disease research organizations worldwide, 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. Country and city lists for rare disease research organizations follow the table.
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 worldwide
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 | 976 contacts → | ||||
Strong evidence “Pharming Group is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 6 | 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. | |||||
| 7 | 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. | |||||
| 8 | 571 contacts → | ||||
Partial evidence “L’AFM-Téléthon est une association de parents et de malades qui mène un combat sans relâche contre des maladies génétiques, rares et lourdement invalidantes.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 9 | 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. | |||||
| 10 | 409 contacts → | ||||
Strong evidence “LifeArc® is a self-funded medical research organisation with one clear purpose – transforming the lives of people living with rare diseases and drug-resistant infections.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 11 | 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. | |||||
| 12 | 321 contacts → | ||||
Indirect evidence “The Institut Pasteur de Lille foundation was created in 1894 by the will of Louis Pasteur, Albert Calmette and the local officials.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 13 | 293 contacts → | ||||
Indirect evidence “Généthon est un laboratoire à but non lucratif, créé et soutenu par l’AFM-Téléthon depuis 1990.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 14 | 257 contacts → | ||||
Strong evidence “TIGEM is a leading research institute dedicated to uncovering the molecular mechanisms of rare genetic diseases and translating these discoveries into innovative therapies, with a…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 15 | 217 contacts → | ||||
Partial evidence “Focused on complex therapies and orphan diseases, Varifarma is a company which is devoted to improving the patients' quality of life.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 16 | 196 contacts → | ||||
Indirect evidence “We’re committed to supporting patients and their caregivers in every way we can.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 17 | 185 contacts → | ||||
Indirect evidence Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 18 | 173 contacts → | ||||
Strong evidence “Founded in 1996, the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between Fondazione Telethon, a major Italian charity funding research on rare…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 19 | 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. | |||||
| 20 | 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. | |||||
| 21 | 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. | |||||
| 22 | 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. | |||||
| 23 | 118 contacts → | ||||
Strong evidence “Transforming Rare Disease Treatment At Orphalan we identify, develop, and provide access to innovative treatments for patients with rare diseases to make a positive and meaningful…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 24 | 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. | |||||
| 25 | 110 contacts → | ||||
Strong evidence “Ågrenska is a national centre of competence for rare diseases and other disabilities.” 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 1,156 rare disease research organizations worldwide
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Rare disease research organizations worldwide at a glance
As of Sep 21, 2026Companies by employee count
Email recency is reported on each country list linked below.
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.
- Coverage
- Every country. Country and city lists for rare disease research organizations are linked below.
- What qualifies
- Companies worldwide 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.
- 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, 11,975 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.
Rare disease research organizations by location
8 lists · largest firstEvery Rare disease research organizations list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
- Rare disease research organizations based in the United States353
- Rare disease research organizations based in France86
- Rare disease research organizations based in the United Kingdom62
- Rare disease research organizations based in Spain45
- Rare disease research organizations based in the Netherlands40
- Rare disease research organizations based in Italy39
- Rare disease research organizations based in Australia27
- Rare disease research organizations based in Canada25
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 worldwide?
RevenueBase identified 1,156 rare disease research organizations worldwide 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 country has the most rare disease research organizations?
The United States leads with 353 companies, followed by France (86) and the United Kingdom (62). Every rare disease research organizations list by country and city is linked on this page.
Which city has the most rare disease research organizations?
Paris leads with 27 headquartered companies, followed by Washington (17) and London (16).
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 11,975 verified decision-maker contacts tracked across these 1,156 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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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.