Company lists · Data refreshed September 2026
Top 25 Monogenic disease gene therapy companies based in the United States
RevenueBase identified 230 monogenic disease gene therapy companies headquartered in the United States as of September 2026 and tracks 19,068 professional contacts across them, including 8,103 with verified work emails. Cambridge is the largest hub.
This list covers monogenic disease gene therapy companies headquartered in the United States, matched on what each company actually does as described in its own words. Ranked by team size, largest first. Every row shows the company's own description, its RevenueBase Smart Search rating, and when it was last checked. Data refreshed September 19, 2026.
Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase Smart Search, the natural-language company search RevenueBase customers use to build their own targeted lists and unlock verified emails and direct dials at the companies on them. How these lists are built and judged.
The 25 largest monogenic disease gene therapy companies based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 3,943 contacts → | ||||
Indirect evidence “Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 2 | 1,359 contacts → | ||||
Strong evidence “Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 3 | 1,313 contacts → | ||||
Partial evidence “For three decades, Ionis has invented medicines that bring better futures to people with serious diseases.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 4 | 837 contacts → | ||||
Indirect evidence “We exist to bring transformative medicines to people living with genetic conditions.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 5 | 605 contacts → | ||||
Partial evidence “Beam Therapeutics, launched in 2018, is pioneering the use of CRISPR base editing to develop a broad portfolio of advanced genetic medicines.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 6 | 510 contacts → | ||||
Strong evidence “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…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 7 | 474 contacts → | ||||
Strong evidence “Intellia Therapeutics is a leading clinical-stage biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 8 | 456 contacts → | ||||
Strong evidence “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…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 9 | 393 contacts → | ||||
Strong evidence “REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 10 | 373 contacts → | ||||
Strong evidence “Since its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 11 | 311 contacts → | ||||
Indirect evidence “Corporate Overview Lexicon Pharmaceuticals is a biopharmaceutical company that has harnessed the power of genetics for drug discovery.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 12 | 302 contacts → | ||||
Strong evidence “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…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 13 | 300 contacts → | ||||
Indirect evidence “A commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines for high unmet medical needs.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 14 | 287 contacts → | ||||
Indirect evidence “Sana Biotechnology is focused on utilizing engineered cells as medicines for patients.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 15 | 258 contacts → | ||||
Strong evidence “Rocket Pharma (NASDAQ: RCKT) is a fully-integrated, clinical-stage company seeking gene therapy cures.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 16 | 239 contacts → | ||||
Partial evidence “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…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 17 | 236 contacts → | ||||
Strong evidence “4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 18 | 232 contacts → | ||||
Partial evidence “Prime Medicine was founded to bring the promise of gene editing to patients.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 19 | 211 contacts → | ||||
Partial evidence “Our mission is to revolutionize medicine, with the ultimate goal of eliminating human suffering and enabling people to live without the burden of disease.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 20 | 208 contacts → | ||||
Partial evidence “Voyager Therapeutics, Inc.(Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure –…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 21 | 207 contacts → | ||||
Partial evidence “Abeona Therapeutics Inc.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 22 | 200 contacts → | ||||
Partial evidence “Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using our proprietary, comprehensive metagenomics-derived genome…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 23 | 189 contacts → | ||||
Strong evidence “Verve Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, is developing a new class of genetic medicines for cardiovascular disease with the potential to transform…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 24 | 172 contacts → | ||||
Indirect evidence “Poseida was acquired by Roche in early 2025 and is now part of the Roche Group.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 25 | 160 contacts → | ||||
Strong evidence “Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
RevenueBase Smart Search
RevenueBase Smart Search assesses information from a company’s LinkedIn profile, information supplied directly by the company, its website, and other publicly available sources. It uses that information to rate how closely the company relates to this list’s category: monogenic disease gene therapy companies. Every row carries its rating and the description the rating was read from, so each one can be checked rather than taken on trust.
- Strong evidence
- The company's own public description of its business names monogenic disease gene therapy companies.
- Partial evidence
- Its own public description names part of monogenic disease gene therapy companies, so it works in or next to the category without describing itself that way.
- Indirect evidence
- The description shown does not name monogenic disease gene therapy companies. The match rests on the rest of the company's public profile rather than the one sentence quoted here, so this is the rating to check first.
RevenueBase Smart Search is how these lists are built: RevenueBase's data engine and AI assemble the companies that match a category defined in a user's own words, so a list can be about what companies actually do rather than about whichever industry code they were filed under.
Showing 25 of 230 monogenic disease gene therapy companies based in the United States
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Monogenic disease gene therapy companies based in the United States at a glance
As of Sep 19, 2026Companies by employee count
Email recency for this list is measured worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 230 companies.
How this list was built
Company categories are rarely black and white. RevenueBase reads each company's own description of its business to decide whether it belongs among monogenic disease gene therapy companies, so some rows will be adjacent to the category rather than pure plays. The description and RevenueBase Smart Search rating on every row are there so you can check each match yourself.
- Source
- The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase Smart Search, the natural-language company search available to every RevenueBase user.
- What qualifies
- Companies headquartered in the United States whose own description of what they do matches “monogenic disease gene therapy companies”. Matching reads each company's description, not its industry code or its name.
- RevenueBase Smart Search
- RevenueBase Smart Search is how this list is assembled and how each row is rated. RevenueBase's data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to a category defined in a user's own words. Each row is rated strong evidence (its own description names monogenic disease gene therapy companies), partial evidence (it names part of the category) or indirect evidence (the description shown names none of it and the match rests on the rest of the company's profile). The description supporting each rating is quoted on the row.
- What we exclude
- Companies whose own description does not clearly place them among monogenic disease gene therapy companies, and records with no verified work email. Companies headquartered outside United States are excluded even when they keep an office there.
- Match quality
- In blind human judging of this matching method, 86% of matched companies were on target. Expect roughly 1 in 7 rows to be adjacent to the category rather than a pure play. Every row shows the company's own description and its RevenueBase Smart Search rating so you can judge each match yourself. Read the full methodology: how the lists are built, how the judging worked, and what the figure means.
- Ranking
- Team size, largest first: companies are ordered by the number of professionals RevenueBase tracks at each one. Employee ranges shown are reported bands, for context.
- Verification
- Every listed company carries at least one verified work email, 8,103 across the full list. Contacts re-verify on a rolling cadence, and each contact's own verification date is shown inside RevenueBase.
- Refresh
- This page's figures were pulled from the company graph on September 19, 2026. Lists refresh periodically as the graph is re-checked.
- Corrections
- Wrong category, wrong headquarters, or a company that has closed? Use “Report an issue” on any row. Reports go to the RevenueBase Data Team and feed back into the company graph.
Frequently asked
What is RevenueBase Smart Search?
RevenueBase Smart Search is how RevenueBase decides which companies belong on this list and how strongly each one fits. Its data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to the category the list is about. Each row is rated strong evidence when the company's own description names monogenic disease gene therapy companies, partial evidence when it names part of the category, and indirect evidence when the description shown names none of it and the match rests on the rest of the company's profile.
How many monogenic disease gene therapy companies are there based in the United States?
RevenueBase identified 230 monogenic disease gene therapy companies headquartered in the United States as of September 2026, matched on each company's own description of what it does rather than its industry code.
What is the largest company on this list?
Biomarin Pharmaceutical INC, headquartered in San Rafael, tops this list with the largest team on record.
Which city has the most monogenic disease gene therapy companies?
Cambridge leads with 21 headquartered companies, followed by Boston (16) and San Diego (14).
How do I get contact information for these companies?
A free RevenueBase account lets you run this exact search and work the top results, verified emails and direct dials included. Upgrading unlocks all 8,103 verified decision-maker contacts tracked across these 230 companies.
How current is this list?
The list refreshes periodically as the company graph is re-checked. This page's figures were pulled on September 19, 2026.
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About this data
RevenueBase is a B2B data infrastructure company. Its company graph covers 60 million companies and 398.9 million professional contacts, with emails verified on a rolling cadence rather than scraped once and left to decay. These company lists are generated directly from that graph with RevenueBase Smart Search, the same natural-language company search every RevenueBase user has: describe a market in plain words, get the companies that match, and add verified emails and direct dials for the people at them. It is the same data that powers RevenueBase's B2B data products, and a free account includes 500 credits to start. How these lists are built and judged.
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