Company lists · Data refreshed September 2026
Top 25 Rare disease biotech investors based in the United States
RevenueBase identified 138 company matches for rare disease biotech investors associated with the United States as of September 2026 and tracks 31,408 professional contacts across them, including 16,765 with verified work emails. New York is the largest hub.
This list covers company matches for rare disease biotech investors 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 disease biotech investors based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 17,217 contacts → | ||||
Exact match “About Genentech We're passionate about finding solutions for people facing the world's most difficult-to-treat conditions.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 2 | 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 | |||||
| 3 | 1,556 contacts → | ||||
Exact match “Leading with purpose.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 4 | 1,212 contacts → | ||||
Exact match “PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 5 | 837 contacts → | ||||
Exact match “We exist to bring transformative medicines to people living with genetic conditions.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 6 | 657 contacts → | ||||
Exact match “Arrowhead Pharmaceuticals is a clinical stage, Nasdaq listed (ticker ARWR) company developing medicines that treat intractable diseases by silencing the genes that cause them.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 7 | 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 | |||||
| 8 | 491 contacts → | ||||
Exact match “Our mission is to identify, develop and deliver life-changing therapies to people living with rare disease.#InRareForLife Community Guidelines…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 9 | 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 | |||||
| 10 | 185 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 11 | 115 contacts → | ||||
Exact match “huMannity Medtec (The Alfred E.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 12 | 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 | |||||
| 13 | 84 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 14 | 81 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 15 | 78 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 16 | 69 contacts → | ||||
Exact match “We operate with a “No Analog” mindset-continually pushing boundaries, navigating new terrain and unleashing fresh thinking.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 17 | 66 contacts → | ||||
Exact match “The ALS Therapy Development Institute (ALS TDI) and its researchers discover and develop potential treatments for ALS.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 18 | 65 contacts → | ||||
Exact match “ITF Therapeutics is the US-based rare disease division of Italfarmaco S.p.A.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 19 | 59 contacts → | ||||
Exact match “At SCYNEXIS, we are committed to improving outcomes for patients living with severe, progressive, and underserved rare diseases.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 20 | 56 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 21 | 54 contacts → | ||||
Exact match “As of October, 2022, Forma Therapeutics has been acquired by Novo Nordisk.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 22 | 53 contacts → | ||||
Exact match “Eton Pharmaceuticals is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 23 | 51 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 24 | 50 contacts → | ||||
Exact match “Aditum Bio, from the Latin word for “access”, was founded on a simple mission: to give patients access to medicines that may not otherwise be developed.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 25 | 46 contacts → | ||||
Exact match “Spero Therapeutics is a clinical-stage biopharmaceutical company focused on advancing next-generation medicines in immunology and inflammation for patients with serious diseases…” 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 disease biotech investors”. 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 138 rare disease biotech investors based in the United States
See the full list, filter by size, metro and specialty, and access 16,765 tracked professional contacts with verified work emails. Direct dials are available where recorded.
Free signup · no credit card
Rare disease biotech investors 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 138 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 disease biotech investors” 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 16,765 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 disease biotech investors”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many rare disease biotech investors are there based in the United States?
RevenueBase identified 138 company matches for rare disease biotech investors 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?
Genentech tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.
Which city has the most rare disease biotech investors?
New York leads with 12 company records, followed by Boston (8) and Cambridge (5).
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 16,765 professional contacts with verified work emails tracked across these 138 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 138 rare disease biotech investors 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 disease biotech investors 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 138 matching company records for rare disease biotech investors based in the United States, with 31,408 tracked contacts and 16,765 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 disease biotech investors 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 |  Genentechgene.comHQ: South San Francisco, CA, United StatesReported employees: 10001+ | South San Francisco, CA, United States | 10001+ | Exact match | 17,217 contacts → |
“About Genentech We're passionate about finding solutions for people facing the world's most difficult-to-treat conditions.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 |  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. | |||||
| 3 | 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. | |||||
| 4 |  PTC Therapeutics, INCptcbio.comHQ: Warren, NJ, United StatesReported employees: 501-1,000 | Warren, NJ, United States | 501-1,000 | Exact match | 1,212 contacts → |
“PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 |  Bridgebiobridgebio.comHQ: Palo Alto, CA, United StatesReported employees: 501-1,000 | Palo Alto, CA, United States | 501-1,000 | Exact match | 837 contacts → |
“We exist to bring transformative medicines to people living with genetic conditions.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Arrowhead Pharmaceuticalsarrowheadpharma.com HQ: Pasadena, CA, United StatesReported employees: 501-1,000 | Pasadena, CA, United States | 501-1,000 | Exact match | 657 contacts → |
“Arrowhead Pharmaceuticals is a clinical stage, Nasdaq listed (ticker ARWR) company developing medicines that treat intractable diseases by silencing the genes that cause them.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 |  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. | |||||
| 8 | Travere Therapeuticstravere.com HQ: San Diego, CA, United StatesReported employees: 201-500 | San Diego, CA, United States | 201-500 | Exact match | 491 contacts → |
“Our mission is to identify, develop and deliver life-changing therapies to people living with rare disease.#InRareForLife Community Guidelines…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 |  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. | |||||
| 10 | Catalyst Pharmaceuticals, INCcatalystpharma.com HQ: Coral Gables, FL, United StatesReported employees: 51-200 | Coral Gables, FL, United States | 51-200 | Exact match | 185 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 11 | Humannity Medtechumannitymedtec.org HQ: Valencia, CA, United StatesReported employees: 51-200 | Valencia, CA, United States | 51-200 | Exact match | 115 contacts → |
“huMannity Medtec (The Alfred E.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 |  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. | |||||
| 13 | Fulcrum Therapeuticsfulcrumtx.com HQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 84 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Everylife Foundation For Rare Diseaseseverylifefoundation.org HQ: Washington, DC, United StatesReported employees: 11-50 | Washington, DC, United States | 11-50 | Exact match | 81 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 |  Zevra Therapeuticszevra.comHQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 78 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 |  Intarcia Therapeutics, INCintarcia.comHQ: Boston, MA, United StatesReported employees: 201-500 | Boston, MA, United States | 201-500 | Exact match | 69 contacts → |
“We operate with a “No Analog” mindset-continually pushing boundaries, navigating new terrain and unleashing fresh thinking.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | HQ: Watertown, MA, United StatesReported employees: 11-50 | Watertown, MA, United States | 11-50 | Exact match | 66 contacts → |
“The ALS Therapy Development Institute (ALS TDI) and its researchers discover and develop potential treatments for ALS.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | ITF Therapeutics LLCitftherapeutics.com HQ: Concord, MA, United StatesReported employees: 51-200 | Concord, MA, United States | 51-200 | Exact match | 65 contacts → |
“ITF Therapeutics is the US-based rare disease division of Italfarmaco S.p.A.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Scynexis, INCscynexis.com HQ: Jersey City, NJ, United StatesReported employees: 11-50 | Jersey City, NJ, United States | 11-50 | Exact match | 59 contacts → |
“At SCYNEXIS, we are committed to improving outcomes for patients living with severe, progressive, and underserved rare diseases.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 |  Gemma Biotherapeuticsgemmabiotx.comHQ: Philadelphia, PA, United StatesReported employees: 51-200 | Philadelphia, PA, United States | 51-200 | Exact match | 56 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 |  Forma Therapeutics NOW Novo Nordisknovonordisk.comHQ: Lexington, MA, United StatesReported employees: 51-200 | Lexington, MA, United States | 51-200 | Exact match | 54 contacts → |
“As of October, 2022, Forma Therapeutics has been acquired by Novo Nordisk.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | Eton Pharmaceuticalsetonpharma.com HQ: Deer Park, IL, United StatesReported employees: 51-200 | Deer Park, IL, United States | 51-200 | Exact match | 53 contacts → |
“Eton Pharmaceuticals is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 |  Leadiant Biosciences, INCleadiant.comHQ: Rockville, MD, United StatesReported employees: 51-200 | Rockville, MD, United States | 51-200 | Exact match | 51 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 24 |  Aditum BIOaditumbio.comHQ: Oakland, CA, United StatesReported employees: 51-200 | Oakland, CA, United States | 51-200 | Exact match | 50 contacts → |
“Aditum Bio, from the Latin word for “access”, was founded on a simple mission: to give patients access to medicines that may not otherwise be developed.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Spero Therapeuticssperotherapeutics.com HQ: Cambridge, MA, United StatesReported employees: 11-50 | Cambridge, MA, United States | 11-50 | Exact match | 46 contacts → |
“Spero Therapeutics is a clinical-stage biopharmaceutical company focused on advancing next-generation medicines in immunology and inflammation for patients with serious diseases…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 138 company matches
Build a targeted audience, find relevant contacts and export available records to your workflow.
Free signup · 500 credits · limited free results
Rare disease biotech investors: 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
138 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 138 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 disease biotech investors?
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 disease biotech investors did RevenueBase find based in the United States?
The saved snapshot records 138 matching company records, 31,408 tracked contacts and 16,765 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 disease biotech investors based in the United States. Snapshot: September 17, 2026. https://revenuebase.ai/companies/rare-disease-biotech-investors/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 disease biotech investors based in the United States
RevenueBase records 138 matching company records for rare disease biotech investors based in the United States, with 31,408 tracked contacts and 16,765 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 disease biotech investors 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 | Genentechgene.com HQ: South San Francisco, CA, United StatesReported employees: 10001+ | South San Francisco, CA, United States | 10001+ | Exact match | 17,217 contacts → |
“About Genentech We're passionate about finding solutions for people facing the world's most difficult-to-treat conditions.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | 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. | |||||
| 3 | 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. | |||||
| 4 | PTC Therapeutics, INCptcbio.com HQ: Warren, NJ, United StatesReported employees: 501-1,000 | Warren, NJ, United States | 501-1,000 | Exact match | 1,212 contacts → |
“PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | Bridgebiobridgebio.com HQ: Palo Alto, CA, United StatesReported employees: 501-1,000 | Palo Alto, CA, United States | 501-1,000 | Exact match | 837 contacts → |
“We exist to bring transformative medicines to people living with genetic conditions.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Arrowhead Pharmaceuticalsarrowheadpharma.com HQ: Pasadena, CA, United StatesReported employees: 501-1,000 | Pasadena, CA, United States | 501-1,000 | Exact match | 657 contacts → |
“Arrowhead Pharmaceuticals is a clinical stage, Nasdaq listed (ticker ARWR) company developing medicines that treat intractable diseases by silencing the genes that cause them.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | 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. | |||||
| 8 | Travere Therapeuticstravere.com HQ: San Diego, CA, United StatesReported employees: 201-500 | San Diego, CA, United States | 201-500 | Exact match | 491 contacts → |
“Our mission is to identify, develop and deliver life-changing therapies to people living with rare disease.#InRareForLife Community Guidelines…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | 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. | |||||
| 10 | Catalyst Pharmaceuticals, INCcatalystpharma.com HQ: Coral Gables, FL, United StatesReported employees: 51-200 | Coral Gables, FL, United States | 51-200 | Exact match | 185 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 11 | Humannity Medtechumannitymedtec.org HQ: Valencia, CA, United StatesReported employees: 51-200 | Valencia, CA, United States | 51-200 | Exact match | 115 contacts → |
“huMannity Medtec (The Alfred E.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | 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. | |||||
| 13 | Fulcrum Therapeuticsfulcrumtx.com HQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 84 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Everylife Foundation For Rare Diseaseseverylifefoundation.org HQ: Washington, DC, United StatesReported employees: 11-50 | Washington, DC, United States | 11-50 | Exact match | 81 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | Zevra Therapeuticszevra.com HQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 78 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Intarcia Therapeutics, INCintarcia.com HQ: Boston, MA, United StatesReported employees: 201-500 | Boston, MA, United States | 201-500 | Exact match | 69 contacts → |
“We operate with a “No Analog” mindset-continually pushing boundaries, navigating new terrain and unleashing fresh thinking.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | HQ: Watertown, MA, United StatesReported employees: 11-50 | Watertown, MA, United States | 11-50 | Exact match | 66 contacts → |
“The ALS Therapy Development Institute (ALS TDI) and its researchers discover and develop potential treatments for ALS.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | ITF Therapeutics LLCitftherapeutics.com HQ: Concord, MA, United StatesReported employees: 51-200 | Concord, MA, United States | 51-200 | Exact match | 65 contacts → |
“ITF Therapeutics is the US-based rare disease division of Italfarmaco S.p.A.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Scynexis, INCscynexis.com HQ: Jersey City, NJ, United StatesReported employees: 11-50 | Jersey City, NJ, United States | 11-50 | Exact match | 59 contacts → |
“At SCYNEXIS, we are committed to improving outcomes for patients living with severe, progressive, and underserved rare diseases.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Gemma Biotherapeuticsgemmabiotx.com HQ: Philadelphia, PA, United StatesReported employees: 51-200 | Philadelphia, PA, United States | 51-200 | Exact match | 56 contacts → |
“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…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | Forma Therapeutics NOW Novo Nordisknovonordisk.com HQ: Lexington, MA, United StatesReported employees: 51-200 | Lexington, MA, United States | 51-200 | Exact match | 54 contacts → |
“As of October, 2022, Forma Therapeutics has been acquired by Novo Nordisk.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | Eton Pharmaceuticalsetonpharma.com HQ: Deer Park, IL, United StatesReported employees: 51-200 | Deer Park, IL, United States | 51-200 | Exact match | 53 contacts → |
“Eton Pharmaceuticals is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Leadiant Biosciences, INCleadiant.com HQ: Rockville, MD, United StatesReported employees: 51-200 | Rockville, MD, United States | 51-200 | Exact match | 51 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 24 | Aditum BIOaditumbio.com HQ: Oakland, CA, United StatesReported employees: 51-200 | Oakland, CA, United States | 51-200 | Exact match | 50 contacts → |
“Aditum Bio, from the Latin word for “access”, was founded on a simple mission: to give patients access to medicines that may not otherwise be developed.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Spero Therapeuticssperotherapeutics.com HQ: Cambridge, MA, United StatesReported employees: 11-50 | Cambridge, MA, United States | 11-50 | Exact match | 46 contacts → |
“Spero Therapeutics is a clinical-stage biopharmaceutical company focused on advancing next-generation medicines in immunology and inflammation for patients with serious diseases…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 138 company matches
Build a targeted audience, find relevant contacts and export available records to your workflow.
Free signup · 500 credits · limited free results
Rare disease biotech investors: 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
138 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 138 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 disease biotech investors?
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 disease biotech investors did RevenueBase find based in the United States?
The saved snapshot records 138 matching company records, 31,408 tracked contacts and 16,765 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 disease biotech investors based in the United States. Snapshot: September 17, 2026. https://revenuebase.ai/companies/rare-disease-biotech-investors/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