Company lists · Data refreshed September 2026
Top 25 Gene therapy companies based in the United States
The 280 gene therapy companies based in the United States are listed with 17,933 contacts and 7,699 email addresses. Updated September 2026, the directory identifies Cambridge as the single most-represented city among the companies on the list.
Gene therapy companies develop, manufacture, or support treatments that modify, replace, add, or regulate genetic material to address disease. This list covers companies that develop, manufacture, test, commercialize, or directly support the research and development of gene therapy products. Ranked by team size, largest first. Data current as of September 7, 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 gene therapy companies based in the United States
Ranked by team size| # | Company | RevenueBase Smart Search | Contacts | ||
|---|---|---|---|---|---|
| 1 | Indirect evidence | 3,943 contacts → | |||
“Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 2 | Partial evidence | 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…” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 3 | Partial evidence | 605 contacts → | |||
“Beam Therapeutics, launched in 2018, is pioneering the use of CRISPR base editing to develop a broad portfolio of advanced genetic medicines.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 4 | Strong evidence | 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…” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 5 | Strong evidence | 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…” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 6 | Strong evidence | 393 contacts → | |||
“REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 7 | Indirect evidence | 311 contacts → | |||
“Corporate Overview Lexicon Pharmaceuticals is a biopharmaceutical company that has harnessed the power of genetics for drug discovery.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 8 | Strong evidence | 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…” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 9 | Indirect evidence | 300 contacts → | |||
“A commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines for high unmet medical needs.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 10 | Indirect evidence | 287 contacts → | |||
“Sana Biotechnology is focused on utilizing engineered cells as medicines for patients.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 11 | Strong evidence | 281 contacts → | |||
“Andelyn Biosciences is a gene therapy CDMO dedicated to Turning Hope Into Reality™.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 12 | Partial evidence | 279 contacts → | |||
“ArsenalBiosciences, Inc, is a clinical stage programmable cell therapy company focused on the realization of solid tumor cell therapy to defeat cancer.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 13 | Strong evidence | 258 contacts → | |||
“Rocket Pharma (NASDAQ: RCKT) is a fully-integrated, clinical-stage company seeking gene therapy cures.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 14 | Partial evidence | 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…” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 15 | Indirect evidence | 211 contacts → | |||
“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: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 16 | Partial evidence | 208 contacts → | |||
“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: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 17 | Partial evidence | 207 contacts → | |||
“Abeona Therapeutics Inc.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 18 | Partial evidence | 200 contacts → | |||
“Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using our proprietary, comprehensive metagenomics-derived genome…” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 19 | Partial evidence | 189 contacts → | |||
“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: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 20 | Strong evidence | 177 contacts → | |||
“Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the…” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 21 | Indirect evidence | 172 contacts → | |||
“Poseida was acquired by Roche in early 2025 and is now part of the Roche Group.” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 22 | Strong evidence | 160 contacts → | |||
“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: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 23 | Strong evidence | 155 contacts → | |||
“Century Therapeutics is an innovative biotechnology company that is building an industry-leading, allogeneic, iPSC-derived cell therapy platform that integrates cutting-edge gene…” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 24 | Strong evidence | 155 contacts → | |||
“Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation…” Source: company website · checked Sep 7, 2026Thanks. The Data Team will review it. | |||||
| 25 | Partial evidence | 138 contacts → | |||
“Caribou Biosciences is a leading clinical-stage CRISPR genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating…” Source: company website · checked Sep 7, 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: 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 gene therapy companies.
- Partial evidence
- Its own public description names part of 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 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 280 gene therapy companies based in the United States
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Gene therapy companies based in the United States at a glance
As of Sep 7, 2026Companies by employee count
Freshness & accuracy
How recently the business emails on this list were verified
Email last verified
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 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 “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 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 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, 7,699 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 7, 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 do gene therapy companies do?
Gene therapy companies develop or support treatments that modify, replace, add, or regulate genetic material to address disease.
How many gene therapy companies are based in the United States?
The directory lists 280 gene therapy companies based in the United States.
Which city has the most gene therapy companies?
Cambridge is the single most-represented city among the companies on the list.
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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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