Company lists · Data refreshed September 2026
Top 25 Rare blood disease biotech companies based in the United States
RevenueBase identified 230 rare blood disease biotech companies headquartered in the United States as of September 2026 and tracks 100,691 professional contacts across them, including 46,742 with verified work emails. Cambridge is the largest hub.
This list covers rare blood disease biotech companies 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 17, 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 blood disease biotech companies based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 36,379 contacts → | ||||
Indirect evidence “At Bristol Myers Squibb, we work every day to transform patients’ lives through science.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 2 | 33,433 contacts → | ||||
Partial evidence “Amgen harnesses the best of biology and technology to fight the world’s toughest diseases, and make people’s lives easier, fuller and longer.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 3 | 8,099 contacts → | ||||
Strong evidence “Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and has made significant…” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 4 | 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 17, 2026Thanks. The Data Team will review it. | |||||
| 5 | 3,943 contacts → | ||||
Partial evidence “Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 6 | 1,556 contacts → | ||||
Indirect evidence “Leading with purpose.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 7 | 1,361 contacts → | ||||
Strong evidence “Founded to find a cure for a daughter’s rare disease, United Therapeutics is a biotechnology company transforming care for people with chronic and life-threatening conditions.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 8 | 929 contacts → | ||||
Indirect evidence “Fresenius Kabi is a leading global health care company that focuses on medicines and medical devices used to care for critically and chronically ill patients.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 9 | 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 17, 2026Thanks. The Data Team will review it. | |||||
| 10 | 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 17, 2026Thanks. The Data Team will review it. | |||||
| 11 | 291 contacts → | ||||
Partial evidence “Rigel Pharmaceuticals, Inc, is a biotechnology company dedicated to discovering, developing and providing novel small molecule drugs that significantly improve the lives of…” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 12 | 282 contacts → | ||||
Strong evidence “Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 13 | 281 contacts → | ||||
Indirect evidence “Designing Proteins.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 14 | 263 contacts → | ||||
Indirect evidence “We are committed to discovering, developing, and commercializing a pipeline of first-in-class therapeutics to change the lives of people globally.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 15 | 236 contacts → | ||||
Strong evidence “4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 16 | 139 contacts → | ||||
Indirect evidence “Disc Medicine is a biopharmaceutical company dedicated to transforming the lives of patients with hematologic disorders.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 17 | 133 contacts → | ||||
Partial evidence “Precigen (Nasdaq: PGEN) is a biopharmaceutical company specializing in the advancement of innovative precision medicines to address difficult-to-treat diseases with high unmet…” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 18 | 130 contacts → | ||||
Indirect evidence “At Inhibrx, our mission is to deliver optimized, biologic therapeutics to people with life-threatening conditions.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 19 | 130 contacts → | ||||
Indirect evidence “IGI, Inc.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 20 | 117 contacts → | ||||
Strong evidence “We have merged with Sobi AB creating a biopharma company positioned to provide innovative medicines that transform the lives of people with rare and debilitating diseases.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 21 | 97 contacts → | ||||
Partial evidence “We are a fully integrated, commercial-stage biopharmaceutical company driving progress for people living with rare immune disorders.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 22 | 97 contacts → | ||||
Indirect evidence “Company Profile LFB USA, Inc.” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 23 | 96 contacts → | ||||
Partial evidence “The Versiti Blood Research Institute’s work is dedicated to hematology in all its facets, spanning basic, translational and clinical research, all with one goal: to make people’s…” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 24 | 92 contacts → | ||||
Partial evidence “The National Bleeding Disorders Foundation is dedicated to finding better treatments and cures for inheritable blood and bleeding disorders and to preventing the complications of…” Source: RevenueBase Database · checked Sep 17, 2026Thanks. The Data Team will review it. | |||||
| 25 | 91 contacts → | ||||
Partial evidence “Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation chemoproteomics RAPID platform to discover medicines for highly validated…” Source: RevenueBase Database · checked Sep 17, 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 blood disease biotech companies. 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 blood disease biotech companies.
- Partial evidence
- Its own public description names part of rare blood disease biotech companies, so it works in or next to the category without describing itself that way.
- Indirect evidence
- The description shown does not name rare blood disease biotech companies. 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 230 rare blood disease biotech companies based in the United States
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Rare blood disease biotech companies based in the United States at a glance
As of Sep 17, 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 230 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 blood disease biotech companies, 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 blood disease biotech companies”. 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 blood disease biotech companies), 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 blood disease biotech companies, 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, 46,742 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 17, 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 blood disease biotech companies, 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 blood disease biotech companies are there based in the United States?
RevenueBase identified 230 rare blood disease biotech companies 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?
Bristol Myers Squibb, headquartered in Lawrence Township, tops this list with the largest team on record.
Which city has the most rare blood disease biotech companies?
Cambridge leads with 16 headquartered companies, followed by San Diego (13) and South San Francisco (10).
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 46,742 verified decision-maker contacts tracked across these 230 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 17, 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.
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