Company lists · Data refreshed September 2026
Top 25 Rare disease gene therapy companies based in the United States
RevenueBase identified 313 company matches for rare disease gene therapy companies associated with the United States as of September 2026 and tracks 56,812 professional contacts across them, including 29,055 with verified work emails. Boston is the largest hub.
This list covers company matches for rare disease gene therapy 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 21, 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 gene therapy companies 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 21, 2026 · View company records in RevenueBase | |||||
| 2 | 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 21, 2026 · View company records in RevenueBase | |||||
| 3 | 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 21, 2026 · View company records in RevenueBase | |||||
| 4 | 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 21, 2026 · View company records in RevenueBase | |||||
| 5 | 1,556 contacts → | ||||
Exact match “Leading with purpose.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 6 | 1,359 contacts → | ||||
Exact match “Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 7 | 1,313 contacts → | ||||
Exact match “For three decades, Ionis has invented medicines that bring better futures to people with serious diseases.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 8 | 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 21, 2026 · View company records in RevenueBase | |||||
| 9 | 837 contacts → | ||||
Exact match “We exist to bring transformative medicines to people living with genetic conditions.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 10 | 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 21, 2026 · View company records in RevenueBase | |||||
| 11 | 510 contacts → | ||||
Exact match “AskBio Inc, a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 12 | 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 21, 2026 · View company records in RevenueBase | |||||
| 13 | 474 contacts → | ||||
Exact match “Intellia Therapeutics is a leading clinical-stage biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 14 | 456 contacts → | ||||
Exact match “Forge, a member of Ajinomoto Bio-Pharma Services, is a gene therapy development engine, focused on enabling access to life changing gene therapies and helping bring them from idea…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 15 | 393 contacts → | ||||
Exact match “REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 16 | 392 contacts → | ||||
Exact match “SpringWorks Therapeutics, a healthcare company of Merck KGaA, Darmstadt, Germany, is a commercial-stage biopharmaceutical company dedicated to improving the lives of patients with…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 17 | 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 21, 2026 · View company records in RevenueBase | |||||
| 18 | 311 contacts → | ||||
Exact match “Corporate Overview Lexicon Pharmaceuticals is a biopharmaceutical company that has harnessed the power of genetics for drug discovery.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 19 | 302 contacts → | ||||
Exact match “Tessera Therapeutics is a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, with the aim…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 20 | 300 contacts → | ||||
Exact match “A commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines for high unmet medical needs.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 21 | 287 contacts → | ||||
Exact match “Sana Biotechnology is focused on utilizing engineered cells as medicines for patients.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 22 | 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 21, 2026 · View company records in RevenueBase | |||||
| 23 | 258 contacts → | ||||
Exact match “Rocket Pharma (NASDAQ: RCKT) is a fully-integrated, clinical-stage company seeking gene therapy cures.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 24 | 239 contacts → | ||||
Exact match “Headquartered in Boston’s Seaport community, Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 25 | 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 21, 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 gene therapy 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 313 rare disease gene therapy companies based in the United States
See the full list, filter by size, metro and specialty, and access 29,055 tracked professional contacts with verified work emails. Direct dials are available where recorded.
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Rare disease gene therapy companies based in the United States at a glance
As of Sep 21, 2026Companies by employee count
Email recency for this list is measured worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 313 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 gene therapy 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 29,055 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 21, 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 gene therapy companies”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many rare disease gene therapy companies are there based in the United States?
RevenueBase identified 313 company matches for rare disease gene therapy 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?
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 gene therapy companies?
Boston leads with 26 company records, followed by Cambridge (21) and San Diego (18).
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 29,055 professional contacts with verified work emails tracked across these 313 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 21, 2026.
Ready to reach these companies? Verified emails and direct dials for all 313 rare disease gene therapy companies based in the United States. Start with a free account, no credit card.
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Rare disease gene therapy companies by geography
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
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- Lists by location
- Lists A-Z
- New lists
- Rare disease gene therapy companies based in France
- Rare disease gene therapy companies based in the United Kingdom
- 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
Company intelligence · public preview
Top 25 rare disease gene therapy 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 313 matching company records for rare disease gene therapy companies based in the United States, with 56,812 tracked contacts and 29,055 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 21, 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 gene therapy 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 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 |  Sarepta Therapeuticssarepta.comHQ: Cambridge, MA, United StatesReported employees: 501-1,000 | Cambridge, MA, United States | 501-1,000 | Exact match | 1,359 contacts → |
“Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | Ionis Pharmaceuticals, INCionis.com HQ: Carlsbad, CA, United StatesReported employees: 501-1,000 | Carlsbad, CA, United States | 501-1,000 | Exact match | 1,313 contacts → |
“For three decades, Ionis has invented medicines that bring better futures to people with serious diseases.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 |  Askbio INCaskbio.comHQ: Research Triangle Park, NC, United StatesReported employees: 501-1,000 | Research Triangle Park, NC, United States | 501-1,000 | Exact match | 510 contacts → |
“AskBio Inc, a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | Intellia Therapeutics, INCintelliatx.com HQ: Cambridge, MA, United StatesReported employees: 201-500 | Cambridge, MA, United States | 201-500 | Exact match | 474 contacts → |
“Intellia Therapeutics is a leading clinical-stage biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 |  Forge Biologicsforgebiologics.comHQ: Columbus, OH, United StatesReported employees: 201-500 | Columbus, OH, United States | 201-500 | Exact match | 456 contacts → |
“Forge, a member of Ajinomoto Bio-Pharma Services, is a gene therapy development engine, focused on enabling access to life changing gene therapies and helping bring them from idea…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 |  Regenxbioregenxbio.comHQ: Rockville, MD, United StatesReported employees: 201-500 | Rockville, MD, United States | 201-500 | Exact match | 393 contacts → |
“REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Springworks Therapeuticsspringworkstx.com HQ: Stamford, CT, United StatesReported employees: 201-500 | Stamford, CT, United States | 201-500 | Exact match | 392 contacts → |
“SpringWorks Therapeutics, a healthcare company of Merck KGaA, Darmstadt, Germany, is a commercial-stage biopharmaceutical company dedicated to improving the lives of patients with…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 |  Lexicon Pharmaceuticals, INClexpharma.comHQ: The Woodlands, TX, United StatesReported employees: 51-200 | The Woodlands, TX, United States | 51-200 | Exact match | 311 contacts → |
“Corporate Overview Lexicon Pharmaceuticals is a biopharmaceutical company that has harnessed the power of genetics for drug discovery.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 |  Tessera Therapeuticstesseratx.comHQ: Somerville, MA, United StatesReported employees: 201-500 | Somerville, MA, United States | 201-500 | Exact match | 302 contacts → |
“Tessera Therapeutics is a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, with the aim…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 |  Krystal Biotech, INCkrystalbio.comHQ: Pittsburgh, PA, United StatesReported employees: 201-500 | Pittsburgh, PA, United States | 201-500 | Exact match | 300 contacts → |
“A commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines for high unmet medical needs.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 |  Sana Biotechnology, INCsana.comHQ: Seattle, WA, United StatesReported employees: 201-500 | Seattle, WA, United States | 201-500 | Exact match | 287 contacts → |
“Sana Biotechnology is focused on utilizing engineered cells as medicines for patients.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 |  Rocket Pharmaceuticalsrocketpharma.comHQ: Cranbury, NJ, United StatesReported employees: 201-500 | Cranbury, NJ, United States | 201-500 | Exact match | 258 contacts → |
“Rocket Pharma (NASDAQ: RCKT) is a fully-integrated, clinical-stage company seeking gene therapy cures.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | Entrada Therapeuticsentradatx.com HQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 239 contacts → |
“Headquartered in Boston’s Seaport community, Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 313 company matches
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Rare disease gene therapy companies: dataset insights
September 21, 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
313 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 313 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 21, 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 gene therapy 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 disease gene therapy companies did RevenueBase find based in the United States?
The saved snapshot records 313 matching company records, 56,812 tracked contacts and 29,055 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 21, 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
Rare disease gene therapy companies by geography
Cite this list
RevenueBase Data Team. Top 25 rare disease gene therapy companies based in the United States. Snapshot: September 21, 2026. https://revenuebase.ai/companies/rare-disease-gene-therapy-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
- Rare disease gene therapy companies based in France
- Rare disease gene therapy companies based in the United Kingdom
- 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
Company intelligence · public preview
Top 25 rare disease gene therapy companies based in the United States
RevenueBase records 313 matching company records for rare disease gene therapy companies based in the United States, with 56,812 tracked contacts and 29,055 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 21, 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 gene therapy 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 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Sarepta Therapeuticssarepta.com HQ: Cambridge, MA, United StatesReported employees: 501-1,000 | Cambridge, MA, United States | 501-1,000 | Exact match | 1,359 contacts → |
“Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | Ionis Pharmaceuticals, INCionis.com HQ: Carlsbad, CA, United StatesReported employees: 501-1,000 | Carlsbad, CA, United States | 501-1,000 | Exact match | 1,313 contacts → |
“For three decades, Ionis has invented medicines that bring better futures to people with serious diseases.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | Askbio INCaskbio.com HQ: Research Triangle Park, NC, United StatesReported employees: 501-1,000 | Research Triangle Park, NC, United States | 501-1,000 | Exact match | 510 contacts → |
“AskBio Inc, a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | Intellia Therapeutics, INCintelliatx.com HQ: Cambridge, MA, United StatesReported employees: 201-500 | Cambridge, MA, United States | 201-500 | Exact match | 474 contacts → |
“Intellia Therapeutics is a leading clinical-stage biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Forge Biologicsforgebiologics.com HQ: Columbus, OH, United StatesReported employees: 201-500 | Columbus, OH, United States | 201-500 | Exact match | 456 contacts → |
“Forge, a member of Ajinomoto Bio-Pharma Services, is a gene therapy development engine, focused on enabling access to life changing gene therapies and helping bring them from idea…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | Regenxbioregenxbio.com HQ: Rockville, MD, United StatesReported employees: 201-500 | Rockville, MD, United States | 201-500 | Exact match | 393 contacts → |
“REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Springworks Therapeuticsspringworkstx.com HQ: Stamford, CT, United StatesReported employees: 201-500 | Stamford, CT, United States | 201-500 | Exact match | 392 contacts → |
“SpringWorks Therapeutics, a healthcare company of Merck KGaA, Darmstadt, Germany, is a commercial-stage biopharmaceutical company dedicated to improving the lives of patients with…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Lexicon Pharmaceuticals, INClexpharma.com HQ: The Woodlands, TX, United StatesReported employees: 51-200 | The Woodlands, TX, United States | 51-200 | Exact match | 311 contacts → |
“Corporate Overview Lexicon Pharmaceuticals is a biopharmaceutical company that has harnessed the power of genetics for drug discovery.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Tessera Therapeuticstesseratx.com HQ: Somerville, MA, United StatesReported employees: 201-500 | Somerville, MA, United States | 201-500 | Exact match | 302 contacts → |
“Tessera Therapeutics is a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, with the aim…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Krystal Biotech, INCkrystalbio.com HQ: Pittsburgh, PA, United StatesReported employees: 201-500 | Pittsburgh, PA, United States | 201-500 | Exact match | 300 contacts → |
“A commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines for high unmet medical needs.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | Sana Biotechnology, INCsana.com HQ: Seattle, WA, United StatesReported employees: 201-500 | Seattle, WA, United States | 201-500 | Exact match | 287 contacts → |
“Sana Biotechnology is focused on utilizing engineered cells as medicines for patients.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Rocket Pharmaceuticalsrocketpharma.com HQ: Cranbury, NJ, United StatesReported employees: 201-500 | Cranbury, NJ, United States | 201-500 | Exact match | 258 contacts → |
“Rocket Pharma (NASDAQ: RCKT) is a fully-integrated, clinical-stage company seeking gene therapy cures.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | Entrada Therapeuticsentradatx.com HQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 239 contacts → |
“Headquartered in Boston’s Seaport community, Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 313 company matches
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Rare disease gene therapy companies: dataset insights
September 21, 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
313 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 313 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 21, 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 gene therapy 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 disease gene therapy companies did RevenueBase find based in the United States?
The saved snapshot records 313 matching company records, 56,812 tracked contacts and 29,055 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 21, 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
Rare disease gene therapy companies by geography
Cite this list
RevenueBase Data Team. Top 25 rare disease gene therapy companies based in the United States. Snapshot: September 21, 2026. https://revenuebase.ai/companies/rare-disease-gene-therapy-companies/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
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