Company lists · Data refreshed September 2026
Top 25 Rare blood disease biotech companies based in the United States
RevenueBase identified 230 company matches for rare blood disease biotech companies associated with 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 company matches for rare blood disease biotech companies associated with the United States, found by RevenueBase SmartSearch using the meaning of business descriptions in its company graph at Exact. Ranked by professionals tracked, largest first. Rows show recorded descriptions where available, Exact search membership and the snapshot date. Data refreshed September 17, 2026.
Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase SmartSearch, 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 → | ||||
Exact match “At Bristol Myers Squibb, we work every day to transform patients’ lives through science.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 2 | 33,433 contacts → | ||||
Exact match “Amgen harnesses the best of biology and technology to fight the world’s toughest diseases, and make people’s lives easier, fuller and longer.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 3 | 8,099 contacts → | ||||
Exact match “Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and has made significant…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 4 | 6,136 contacts → | ||||
Exact match “Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 5 | 3,943 contacts → | ||||
Exact match “Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 6 | 1,556 contacts → | ||||
Exact match “Leading with purpose.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 7 | 1,361 contacts → | ||||
Exact match “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.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 8 | 929 contacts → | ||||
Exact match “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.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 9 | 603 contacts → | ||||
Exact match “Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 10 | 326 contacts → | ||||
Exact match “Sobi is an international specialty healthcare company dedicated to developing treatments and extensive patient support networks for people with rare and difficult to treat…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 11 | 291 contacts → | ||||
Exact match “Rigel Pharmaceuticals, Inc, is a biotechnology company dedicated to discovering, developing and providing novel small molecule drugs that significantly improve the lives of…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 12 | 282 contacts → | ||||
Exact match “Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 13 | 281 contacts → | ||||
Exact match “Designing Proteins.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 14 | 263 contacts → | ||||
Exact match “We are committed to discovering, developing, and commercializing a pipeline of first-in-class therapeutics to change the lives of people globally.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 15 | 236 contacts → | ||||
Exact match “4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 16 | 139 contacts → | ||||
Exact match “Disc Medicine is a biopharmaceutical company dedicated to transforming the lives of patients with hematologic disorders.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 17 | 133 contacts → | ||||
Exact match “Precigen (Nasdaq: PGEN) is a biopharmaceutical company specializing in the advancement of innovative precision medicines to address difficult-to-treat diseases with high unmet…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 18 | 130 contacts → | ||||
Exact match “At Inhibrx, our mission is to deliver optimized, biologic therapeutics to people with life-threatening conditions.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 19 | 130 contacts → | ||||
Exact match “IGI, Inc.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 20 | 117 contacts → | ||||
Exact match “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.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 21 | 97 contacts → | ||||
Exact match “We are a fully integrated, commercial-stage biopharmaceutical company driving progress for people living with rare immune disorders.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 22 | 97 contacts → | ||||
Exact match “Company Profile LFB USA, Inc.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 23 | 96 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 24 | 92 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 25 | 91 contacts → | ||||
Exact match “Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation chemoproteomics RAPID platform to discover medicines for highly validated…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
RevenueBase SmartSearch
Describe a niche business in your own words. RevenueBase SmartSearch compares the meaning of your description with company business descriptions compiled in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit.
This list uses Exact for “rare blood disease biotech companies”. Each Exact match badge shows membership in that saved search; it is not an individual similarity score or a service-quality rating. Recorded descriptions are shown where available.
Showing 25 of 230 rare blood disease biotech companies based in the United States
See the full list, filter by size, metro and specialty, and access 46,742 tracked professional contacts with verified work emails. Direct dials are available where recorded.
Free signup · no credit card
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
The recorded descriptions help you assess these company matches. Check a company’s website for its current services and locations.
- Source
- The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase SmartSearch, the natural-language company search available to every RevenueBase user.
- What qualifies
- Company records associated with the United States whose business descriptions match “rare blood disease biotech companies” by meaning at the Exact setting. Descriptions are compiled in the RevenueBase company graph from public websites, profiles and company information where available; they are not guaranteed quotations from the company's website.
- RevenueBase SmartSearch
- Describe a niche company in your own words to find businesses by meaning. Exact, Similar, Related and Broad control the breadth of RevenueBase SmartSearch. This saved list uses Exact. Each row's Exact match badge shows inclusion in that search, not an individual score or a service-quality judgment.
- Public preview
- The public preview omits records without a verified work email. Where headquarters information is recorded, preview rows are also filtered to this page’s location. Dataset totals and geographic summaries describe the full matched records before these preview filters.
- Match quality
- RevenueBase's August 25, 2026 study assessed 4,198 description/company pairs across matching settings. The publisher reports 86% good or better at Exact. This evaluates the method across queries; it does not audit this individual list. Recorded company descriptions, where available, help you judge each match yourself. Read the full methodology: how the lists are built and what the study measures.
- 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 displayed company carries at least one verified work email. RevenueBase tracks 46,742 professional contacts with verified work emails across the full matched dataset. 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.
- Record context
- Company records and descriptions can change. Check the company's website for its current services and locations.
Frequently asked
What is RevenueBase SmartSearch?
RevenueBase SmartSearch finds niche companies by the meaning of their business descriptions in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit your description. This list uses Exact for “rare blood disease biotech companies”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many rare blood disease biotech companies are there based in the United States?
RevenueBase identified 230 company matches for rare blood disease biotech companies associated with the United States as of September 2026, using the meaning of business descriptions at Exact. This is the recorded matched set in the RevenueBase company graph.
What is the largest company on this list?
Bristol Myers Squibb tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.
Which city has the most rare blood disease biotech companies?
Cambridge leads with 16 company records, 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 limited free results with 500 credits to start. Upgrading provides access to the 46,742 professional contacts with verified work emails tracked across these 230 companies; direct dials are available where recorded.
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.
Ready to reach these companies? Verified emails and direct dials for all 230 rare blood disease biotech companies based in the United States. Start with a free account, no credit card.
Download this list →Related lists
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 SmartSearch, 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.
More company lists
- All company lists
- Lists by location
- Lists A-Z
- New lists
- AI companies based in the United States
- Cybersecurity companies based in the United States
- Manufacturing companies based in the United States
- Media companies based in the United States
- Oil and gas companies based in the United States
- Real estate companies based in the United States
Company intelligence · public preview
Top 25 rare blood disease biotech companies based in the United States
Find companies in any niche.
Describe the businesses you want in your own words. RevenueBase SmartSearch matches company descriptions by meaning.
RevenueBase records 230 matching company records for rare blood disease biotech companies based in the United States, with 100,691 tracked contacts and 46,742 verified work emails.
This public preview shows 25 selected records ordered by tracked contacts. RevenueBase SmartSearch finds company descriptions that match by meaning at Exact. Snapshot: September 17, 2026.
RevenueBase compiles business descriptions for niche-company discovery; each row shows a recorded excerpt where available. Adapt this search to find the companies and contacts you want.
Top 25 rare blood disease biotech companies based in the United States
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 |  Bristol Myers Squibbbms.comHQ: Lawrence Township, NJ, United StatesReported employees: 10001+ | Lawrence Township, NJ, United States | 10001+ | Exact match | 36,379 contacts → |
“At Bristol Myers Squibb, we work every day to transform patients’ lives through science.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 |  AMGENamgen.comHQ: Thousand Oaks, CA, United StatesReported employees: 10001+ | Thousand Oaks, CA, United States | 10001+ | Exact match | 33,433 contacts → |
“Amgen harnesses the best of biology and technology to fight the world’s toughest diseases, and make people’s lives easier, fuller and longer.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 |  Vertex Pharmaceuticalsvrtx.comHQ: Boston, MA, United StatesReported employees: 5,001-10,000 | Boston, MA, United States | 5,001-10,000 | Exact match | 8,099 contacts → |
“Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and has made significant…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 |  Alexion Pharmaceuticals, INCalexion.comHQ: Boston, MA, United StatesReported employees: 1,001-5,000 | Boston, MA, United States | 1,001-5,000 | Exact match | 6,136 contacts → |
“Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 |  Biomarin Pharmaceutical INCbiomarin.comHQ: San Rafael, CA, United StatesReported employees: 1,001-5,000 | San Rafael, CA, United States | 1,001-5,000 | Exact match | 3,943 contacts → |
“Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Ultragenyxultragenyx.com HQ: Novato, CA, United StatesReported employees: 1,001-5,000 | Novato, CA, United States | 1,001-5,000 | Exact match | 1,556 contacts → |
“Leading with purpose.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 |  United Therapeutics Corporationunither.comHQ: Silver Spring, MD, United StatesReported employees: 1,001-5,000 | Silver Spring, MD, United States | 1,001-5,000 | Exact match | 1,361 contacts → |
“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.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | Fenwal, Inc., A Fresenius Kabi Companyfenwalinc.com HQ: Lake Zurich, IL, United StatesReported employees: 10001+ | Lake Zurich, IL, United States | 10001+ | Exact match | 929 contacts → |
“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.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 |  Agios Pharmaceuticalsagios.comHQ: Cambridge, MA, United StatesReported employees: 501-1,000 | Cambridge, MA, United States | 501-1,000 | Exact match | 603 contacts → |
“Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 |  SOBIsobi-northamerica.comHQ: Waltham, MA, United StatesReported employees: 201-500 | Waltham, MA, United States | 201-500 | Exact match | 326 contacts → |
“Sobi is an international specialty healthcare company dedicated to developing treatments and extensive patient support networks for people with rare and difficult to treat…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 |  Rigel Pharmaceuticals INCrigel.comHQ: South San Francisco, CA, United StatesReported employees: 51-200 | South San Francisco, CA, United States | 51-200 | Exact match | 291 contacts → |
“Rigel Pharmaceuticals, Inc, is a biotechnology company dedicated to discovering, developing and providing novel small molecule drugs that significantly improve the lives of…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 |  Viridian Therapeutics, INCviridiantherapeutics.comHQ: Waltham, MA, United StatesReported employees: 201-500 | Waltham, MA, United States | 201-500 | Exact match | 282 contacts → |
“Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | Xencorxencor.com HQ: Pasadena, CA, United StatesReported employees: 201-500 | Pasadena, CA, United States | 201-500 | Exact match | 281 contacts → |
“Designing Proteins.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Fibrogen, INCfibrogen.com HQ: San Francisco, CA, United StatesReported employees: 201-500 | San Francisco, CA, United States | 201-500 | Exact match | 263 contacts → |
“We are committed to discovering, developing, and commercializing a pipeline of first-in-class therapeutics to change the lives of people globally.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 |  4d Molecular Therapeutics4dmoleculartherapeutics.comHQ: Emeryville, CA, United StatesReported employees: 51-200 | Emeryville, CA, United States | 51-200 | Exact match | 236 contacts → |
“4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Disc Medicinediscmedicine.com HQ: Watertown, MA, United StatesReported employees: 51-200 | Watertown, MA, United States | 51-200 | Exact match | 139 contacts → |
“Disc Medicine is a biopharmaceutical company dedicated to transforming the lives of patients with hematologic disorders.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 |  Precigenprecigen.comHQ: Germantown, MD, United StatesReported employees: 51-200 | Germantown, MD, United States | 51-200 | Exact match | 133 contacts → |
“Precigen (Nasdaq: PGEN) is a biopharmaceutical company specializing in the advancement of innovative precision medicines to address difficult-to-treat diseases with high unmet…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 |  Inhibrx Biosciences, INCinhibrx.comHQ: San Diego, CA, United StatesReported employees: 51-200 | San Diego, CA, United States | 51-200 | Exact match | 130 contacts → |
“At Inhibrx, our mission is to deliver optimized, biologic therapeutics to people with life-threatening conditions.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 |  IGIiginnovate.comHQ: New York, NY, United StatesReported employees: 51-200 | New York, NY, United States | 51-200 | Exact match | 130 contacts → |
“IGI, Inc.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 |  CTI Biopharmasobi.comHQ: Seattle, WA, United StatesReported employees: 51-200 | Seattle, WA, United States | 51-200 | Exact match | 117 contacts → |
“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.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 |  X4 Pharmaceuticalsx4pharma.comHQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 97 contacts → |
“We are a fully integrated, commercial-stage biopharmaceutical company driving progress for people living with rare immune disorders.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | LFB USAlfb-usa.com HQ: Framingham, MA, United StatesReported employees: 51-200 | Framingham, MA, United States | 51-200 | Exact match | 97 contacts → |
“Company Profile LFB USA, Inc.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Versiti Blood Research Instituteversiti.org HQ: Milwaukee, WI, United StatesReported employees: 201-500 | Milwaukee, WI, United States | 201-500 | Exact match | 96 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | National Bleeding Disorders Foundationbleeding.org HQ: New York, NY, United StatesReported employees: 51-200 | New York, NY, United States | 51-200 | Exact match | 92 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 |  Jnana Therapeuticsjnanatx.comHQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 91 contacts → |
“Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation chemoproteomics RAPID platform to discover medicines for highly validated…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 230 company matches
Build a targeted audience, find relevant contacts and export available records to your workflow.
Free signup · 500 credits · limited free results
Rare blood disease biotech companies: dataset insights
September 17, 2026These summaries describe recorded coverage in this saved dataset. This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Reported company sizes
230 records in the displayed employee bands.
0 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
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 company list was built
Describe a niche in your own words: companies installing commercial solar, or manufacturers of sustainable packaging. RevenueBase SmartSearch finds companies whose compiled business descriptions match by meaning. This list is one saved example to adapt.
Where the company descriptions come from
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
RevenueBase SmartSearch matching settings
- Exact
- Closest fits to the business described.
- Similar
- Similar businesses with a wider range of fits.
- Related
- Related businesses in and around the category.
- Broad
- A broader set of businesses connected to the description.
RevenueBase SmartSearch · Exact match identifies inclusion in this search's closest semantic matching setting. It is not a per-company confidence score, a literal keyword match, or a quality endorsement.
Selection, ordering and scope
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Recorded company descriptions support research; check company websites for current services. The selected preview is ordered by tracked contacts and is not a list of the best service providers.
Snapshot: September 17, 2026.
RevenueBase is a B2B data infrastructure company. Its September 3, 2026 published methodology describes a graph of 60 million companies and 398.9 million professional contacts; these platform figures differ from this page's totals. Published methodology ↗
Questions about this company list
What does Exact match mean for rare blood disease biotech companies?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many rare blood disease biotech companies did RevenueBase find based in the United States?
The saved snapshot records 230 matching company records, 100,691 tracked contacts and 46,742 verified emails. The public preview displays 25 selected records.
Where do the company descriptions come from?
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
How are the public companies ordered?
The selected public records are ordered by contacts tracked in RevenueBase, highest first. Contact coverage is a prospecting measure, not an assessment of the company's services or quality.
How can I get contact information?
Select a company's contact count to open its filtered contacts in RevenueBase. Free signup is required and includes 500 credits and limited free results. Verified emails, direct dials and mobile numbers depend on available coverage.
How current is this list?
Snapshot: September 17, 2026. Review each company's website for current services and locations. A recorded business description does not establish that every company offers every service named in the search.
What do headquarters and employee bands mean?
These are recorded company-level fields, rather than a map of every branch or a count of specialists. Missing headquarters or employee bands are labeled; older records may show a location separately from recorded headquarters.
Build your next target company list
Adapt this niche search and work with available contacts in RevenueBase.
Related lists
Cite this list
RevenueBase Data Team. Top 25 rare blood disease biotech companies based in the United States. Snapshot: September 17, 2026. https://revenuebase.ai/companies/rare-blood-disease-biotech-companies/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
More company lists
- All company lists
- Lists by location
- Lists A-Z
- New lists
- AI companies based in the United States
- Cybersecurity companies based in the United States
- Manufacturing companies based in the United States
- Media companies based in the United States
- Oil and gas companies based in the United States
- Real estate companies based in the United States
Company intelligence · public preview
Top 25 rare blood disease biotech companies based in the United States
RevenueBase records 230 matching company records for rare blood disease biotech companies based in the United States, with 100,691 tracked contacts and 46,742 verified work emails.
This public preview shows 25 selected records ordered by tracked contacts. RevenueBase SmartSearch finds company descriptions that match by meaning at Exact. Snapshot: September 17, 2026.
RevenueBase compiles business descriptions for niche-company discovery; each row shows a recorded excerpt where available. Adapt this search to find the companies and contacts you want.
Top 25 rare blood disease biotech companies based in the United States
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 | Bristol Myers Squibbbms.com HQ: Lawrence Township, NJ, United StatesReported employees: 10001+ | Lawrence Township, NJ, United States | 10001+ | Exact match | 36,379 contacts → |
“At Bristol Myers Squibb, we work every day to transform patients’ lives through science.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | AMGENamgen.com HQ: Thousand Oaks, CA, United StatesReported employees: 10001+ | Thousand Oaks, CA, United States | 10001+ | Exact match | 33,433 contacts → |
“Amgen harnesses the best of biology and technology to fight the world’s toughest diseases, and make people’s lives easier, fuller and longer.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 | Vertex Pharmaceuticalsvrtx.com HQ: Boston, MA, United StatesReported employees: 5,001-10,000 | Boston, MA, United States | 5,001-10,000 | Exact match | 8,099 contacts → |
“Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and has made significant…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | Alexion Pharmaceuticals, INCalexion.com HQ: Boston, MA, United StatesReported employees: 1,001-5,000 | Boston, MA, United States | 1,001-5,000 | Exact match | 6,136 contacts → |
“Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | Biomarin Pharmaceutical INCbiomarin.com HQ: San Rafael, CA, United StatesReported employees: 1,001-5,000 | San Rafael, CA, United States | 1,001-5,000 | Exact match | 3,943 contacts → |
“Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Ultragenyxultragenyx.com HQ: Novato, CA, United StatesReported employees: 1,001-5,000 | Novato, CA, United States | 1,001-5,000 | Exact match | 1,556 contacts → |
“Leading with purpose.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | United Therapeutics Corporationunither.com HQ: Silver Spring, MD, United StatesReported employees: 1,001-5,000 | Silver Spring, MD, United States | 1,001-5,000 | Exact match | 1,361 contacts → |
“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.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | Fenwal, Inc., A Fresenius Kabi Companyfenwalinc.com HQ: Lake Zurich, IL, United StatesReported employees: 10001+ | Lake Zurich, IL, United States | 10001+ | Exact match | 929 contacts → |
“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.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | Agios Pharmaceuticalsagios.com HQ: Cambridge, MA, United StatesReported employees: 501-1,000 | Cambridge, MA, United States | 501-1,000 | Exact match | 603 contacts → |
“Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | SOBIsobi-northamerica.com HQ: Waltham, MA, United StatesReported employees: 201-500 | Waltham, MA, United States | 201-500 | Exact match | 326 contacts → |
“Sobi is an international specialty healthcare company dedicated to developing treatments and extensive patient support networks for people with rare and difficult to treat…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | Rigel Pharmaceuticals INCrigel.com HQ: South San Francisco, CA, United StatesReported employees: 51-200 | South San Francisco, CA, United States | 51-200 | Exact match | 291 contacts → |
“Rigel Pharmaceuticals, Inc, is a biotechnology company dedicated to discovering, developing and providing novel small molecule drugs that significantly improve the lives of…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | Viridian Therapeutics, INCviridiantherapeutics.com HQ: Waltham, MA, United StatesReported employees: 201-500 | Waltham, MA, United States | 201-500 | Exact match | 282 contacts → |
“Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | Xencorxencor.com HQ: Pasadena, CA, United StatesReported employees: 201-500 | Pasadena, CA, United States | 201-500 | Exact match | 281 contacts → |
“Designing Proteins.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Fibrogen, INCfibrogen.com HQ: San Francisco, CA, United StatesReported employees: 201-500 | San Francisco, CA, United States | 201-500 | Exact match | 263 contacts → |
“We are committed to discovering, developing, and commercializing a pipeline of first-in-class therapeutics to change the lives of people globally.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | 4d Molecular Therapeutics4dmoleculartherapeutics.com HQ: Emeryville, CA, United StatesReported employees: 51-200 | Emeryville, CA, United States | 51-200 | Exact match | 236 contacts → |
“4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Disc Medicinediscmedicine.com HQ: Watertown, MA, United StatesReported employees: 51-200 | Watertown, MA, United States | 51-200 | Exact match | 139 contacts → |
“Disc Medicine is a biopharmaceutical company dedicated to transforming the lives of patients with hematologic disorders.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Precigenprecigen.com HQ: Germantown, MD, United StatesReported employees: 51-200 | Germantown, MD, United States | 51-200 | Exact match | 133 contacts → |
“Precigen (Nasdaq: PGEN) is a biopharmaceutical company specializing in the advancement of innovative precision medicines to address difficult-to-treat diseases with high unmet…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Inhibrx Biosciences, INCinhibrx.com HQ: San Diego, CA, United StatesReported employees: 51-200 | San Diego, CA, United States | 51-200 | Exact match | 130 contacts → |
“At Inhibrx, our mission is to deliver optimized, biologic therapeutics to people with life-threatening conditions.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | IGIiginnovate.com HQ: New York, NY, United StatesReported employees: 51-200 | New York, NY, United States | 51-200 | Exact match | 130 contacts → |
“IGI, Inc.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | CTI Biopharmasobi.com HQ: Seattle, WA, United StatesReported employees: 51-200 | Seattle, WA, United States | 51-200 | Exact match | 117 contacts → |
“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.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | X4 Pharmaceuticalsx4pharma.com HQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 97 contacts → |
“We are a fully integrated, commercial-stage biopharmaceutical company driving progress for people living with rare immune disorders.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | LFB USAlfb-usa.com HQ: Framingham, MA, United StatesReported employees: 51-200 | Framingham, MA, United States | 51-200 | Exact match | 97 contacts → |
“Company Profile LFB USA, Inc.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Versiti Blood Research Instituteversiti.org HQ: Milwaukee, WI, United StatesReported employees: 201-500 | Milwaukee, WI, United States | 201-500 | Exact match | 96 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | National Bleeding Disorders Foundationbleeding.org HQ: New York, NY, United StatesReported employees: 51-200 | New York, NY, United States | 51-200 | Exact match | 92 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Jnana Therapeuticsjnanatx.com HQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 91 contacts → |
“Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation chemoproteomics RAPID platform to discover medicines for highly validated…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 230 company matches
Build a targeted audience, find relevant contacts and export available records to your workflow.
Free signup · 500 credits · limited free results
Rare blood disease biotech companies: dataset insights
September 17, 2026These summaries describe recorded coverage in this saved dataset. This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Reported company sizes
230 records in the displayed employee bands.
0 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
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 company list was built
Describe a niche in your own words: companies installing commercial solar, or manufacturers of sustainable packaging. RevenueBase SmartSearch finds companies whose compiled business descriptions match by meaning. This list is one saved example to adapt.
Where the company descriptions come from
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
RevenueBase SmartSearch matching settings
- Exact
- Closest fits to the business described.
- Similar
- Similar businesses with a wider range of fits.
- Related
- Related businesses in and around the category.
- Broad
- A broader set of businesses connected to the description.
RevenueBase SmartSearch · Exact match identifies inclusion in this search's closest semantic matching setting. It is not a per-company confidence score, a literal keyword match, or a quality endorsement.
Selection, ordering and scope
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Recorded company descriptions support research; check company websites for current services. The selected preview is ordered by tracked contacts and is not a list of the best service providers.
Snapshot: September 17, 2026.
RevenueBase is a B2B data infrastructure company. Its September 3, 2026 published methodology describes a graph of 60 million companies and 398.9 million professional contacts; these platform figures differ from this page's totals. Published methodology ↗
Questions about this company list
What does Exact match mean for rare blood disease biotech companies?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many rare blood disease biotech companies did RevenueBase find based in the United States?
The saved snapshot records 230 matching company records, 100,691 tracked contacts and 46,742 verified emails. The public preview displays 25 selected records.
Where do the company descriptions come from?
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
How are the public companies ordered?
The selected public records are ordered by contacts tracked in RevenueBase, highest first. Contact coverage is a prospecting measure, not an assessment of the company's services or quality.
How can I get contact information?
Select a company's contact count to open its filtered contacts in RevenueBase. Free signup is required and includes 500 credits and limited free results. Verified emails, direct dials and mobile numbers depend on available coverage.
How current is this list?
Snapshot: September 17, 2026. Review each company's website for current services and locations. A recorded business description does not establish that every company offers every service named in the search.
What do headquarters and employee bands mean?
These are recorded company-level fields, rather than a map of every branch or a count of specialists. Missing headquarters or employee bands are labeled; older records may show a location separately from recorded headquarters.
Build your next target company list
Adapt this niche search and work with available contacts in RevenueBase.
Related lists
Cite this list
RevenueBase Data Team. Top 25 rare blood disease biotech companies based in the United States. Snapshot: September 17, 2026. https://revenuebase.ai/companies/rare-blood-disease-biotech-companies/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
More company lists
- All company lists
- Lists by location
- Lists A-Z
- New lists
- AI companies based in the United States
- Cybersecurity companies based in the United States
- Manufacturing companies based in the United States
- Media companies based in the United States
- Oil and gas companies based in the United States
- Real estate companies based in the United States