Company lists · Data refreshed September 2026
Top 25 Genetic disorder treatment companies worldwide
RevenueBase identified 705 genetic disorder treatment companies worldwide as of September 2026 and tracks 28,012 professional contacts across them, including 13,098 with verified work emails. The United States is the largest market and Cambridge the largest hub.
This list covers genetic disorder treatment companies worldwide, 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. Country and city lists for genetic disorder treatment companies follow the table.
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 genetic disorder treatment companies worldwide
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 3,943 contacts → | ||||
Partial 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 | 1,556 contacts → | ||||
Indirect evidence “Leading with purpose.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 3 | 1,359 contacts → | ||||
Partial 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. | |||||
| 4 | 1,048 contacts → | ||||
Partial evidence “We see a world where every genetic condition is understood, and every patient receives the care they need to live their healthiest life through genomics.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 5 | 957 contacts → | ||||
Partial evidence “Ambry Genetics, a Tempus company, translates scientific research into clinically actionable test results based upon a deep understanding of the human genome and the biology behind…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 6 | 837 contacts → | ||||
Partial 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. | |||||
| 7 | 510 contacts → | ||||
Indirect 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. | |||||
| 8 | 470 contacts → | ||||
Partial evidence “CENTOGENE is a leading company in the field of genetic diagnostics and precision medicine, dedicated to transforming clinical, genetic, and biochemical data into medical solutions…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 9 | 456 contacts → | ||||
Indirect 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. | |||||
| 10 | 398 contacts → | ||||
Partial evidence “MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicine company with a broad pipeline of late-stage clinical programs supported by end-to-end…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 11 | 374 contacts → | ||||
Indirect evidence “Mirum Pharmaceuticals is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 12 | 372 contacts → | ||||
Partial evidence “TGen, a part of City of Hope, is a non-profit 501(c)(3) organization focused on developing earlier diagnostics and smarter treatments.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 13 | 349 contacts → | ||||
Partial evidence “uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 14 | 326 contacts → | ||||
Partial evidence “Sobi is an international specialty healthcare company dedicated to developing treatments and extensive patient support networks for people with rare and difficult to treat…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 15 | 311 contacts → | ||||
Partial 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. | |||||
| 16 | 302 contacts → | ||||
Indirect 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. | |||||
| 17 | 300 contacts → | ||||
Partial 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. | |||||
| 18 | 293 contacts → | ||||
Indirect evidence “Généthon est un laboratoire à but non lucratif, créé et soutenu par l’AFM-Téléthon depuis 1990.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 19 | 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. | |||||
| 20 | 270 contacts → | ||||
Partial evidence “For Sangamo Therapeutics, science is a means to develop new medicines with the potential to transform the lives of patients living with serious genetic diseases.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 21 | 258 contacts → | ||||
Indirect 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. | |||||
| 22 | 239 contacts → | ||||
Indirect 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. | |||||
| 23 | 236 contacts → | ||||
Partial 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. | |||||
| 24 | 221 contacts → | ||||
Partial evidence “Orchard Therapeutics, a Kyowa Kirin company, is a global gene therapy leader focused on ending the devastation caused by genetic and other severe diseases by discovering…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 25 | 217 contacts → | ||||
Partial evidence “Health in Code is a leading biotechnology company with a clear mission: to identify health conditions that can benefit from genetic diagnosis and to develop and deliver advanced…” 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: genetic disorder treatment 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 genetic disorder treatment companies.
- Partial evidence
- Its own public description names part of genetic disorder treatment companies, so it works in or next to the category without describing itself that way.
- Indirect evidence
- The description shown does not name genetic disorder treatment 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 705 genetic disorder treatment companies worldwide
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Genetic disorder treatment companies worldwide at a glance
As of Sep 19, 2026Companies by employee count
Email recency is reported on each country list linked below.
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 genetic disorder treatment 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.
- Coverage
- Every country. Country and city lists for genetic disorder treatment companies are linked below.
- What qualifies
- Companies worldwide whose own description of what they do matches “genetic disorder treatment 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 genetic disorder treatment 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 genetic disorder treatment companies, and records with no verified work email.
- 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, 13,098 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.
Genetic disorder treatment companies by location
2 lists · largest firstEvery Genetic disorder treatment companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
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 genetic disorder treatment 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 genetic disorder treatment companies are there worldwide?
RevenueBase identified 705 genetic disorder treatment companies worldwide 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 country has the most genetic disorder treatment companies?
The United States leads with 300 companies, followed by the United Kingdom (29). Every genetic disorder treatment companies list by country and city is linked on this page.
Which city has the most genetic disorder treatment companies?
Cambridge leads with 23 headquartered companies, followed by Boston (15) 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 13,098 verified decision-maker contacts tracked across these 705 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.