Company lists · Data refreshed September 2026
Top 21 Rare genetic disease treatment companies based in the United Kingdom
RevenueBase identified 29 rare genetic disease treatment companies headquartered in the United Kingdom as of September 2026 and tracks 1,288 professional contacts across them, including 808 with verified work emails. London is the largest hub.
This list covers rare genetic disease treatment companies headquartered in the United Kingdom, 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 21, 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 21 largest rare genetic disease treatment companies based in the United Kingdom
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 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 21, 2026Thanks. The Data Team will review it. | |||||
| 2 | 221 contacts → | ||||
Strong 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 21, 2026Thanks. The Data Team will review it. | |||||
| 3 | 196 contacts → | ||||
Indirect evidence “We’re committed to supporting patients and their caregivers in every way we can.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 4 | 98 contacts → | ||||
Indirect evidence “Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 5 | 70 contacts → | ||||
Strong evidence “We are committed to transforming the lives of people affected by cancer or rare disease, globally.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 6 | 60 contacts → | ||||
Strong evidence “Healx is a mission-driven technology company pioneering the next generation of drug discovery in order to bring novel, effective treatments to rare disease patients around the…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 7 | 50 contacts → | ||||
Strong evidence “As the UK and Ireland affiliate of Sobi, an international biopharmaceutical company, we are transforming life for people with rare diseases.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 8 | 40 contacts → | ||||
Partial evidence “Welcome to Mereo BioPharma, we are an agile biopharmaceutical company, focused on finding and bringing to market therapies that will improve the lives of people living with a rare…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 9 | 26 contacts → | ||||
Indirect evidence “Harnessing an innate mechanism of gene activation, MiNA Therapeutics' platform enables the development of new medicines that restore normal function to patients’ cells.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 10 | 21 contacts → | ||||
Partial evidence “Biodexa Pharmaceuticals PLC (listed on NASDAQ: BDRX) is a clinical stage biopharmaceutical company developing innovative products focused on the treatment or prevention of…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 11 | 16 contacts → | ||||
Indirect evidence “Pencil Biosciences is developing a truly innovative gene modulation technology that can have an impact across a range of applications, including new therapeutic options for…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 12 | 13 contacts → | ||||
Partial evidence “Freeline is a clinical-stage biotechnology company focused on developing transformative gene therapies for chronic debilitating diseases.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 13 | 11 contacts → | ||||
Indirect evidence “Mironid Limited is an innovative new drug discovery company that develops proprietary drug candidate molecules by modulating the activity of key cell signalling proteins.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 14 | 9 contacts → | ||||
Partial evidence “RareCan supports people living with rare cancers to better understand their situation and make informed decisions with confidence.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 15 | 8 contacts → | ||||
Indirect evidence “Axovia Therapeutics is dedicated to the research and development of novel AAV gene therapies that target key aspects of ciliopathies, addressing the devastating impact of the…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 16 | 7 contacts → | ||||
Strong evidence “Overview SynaptixBio is a rare disease biotech company that is pioneering ground-breaking treatments for severe leukodystrophies and other childhood neurodegenerative diseases.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 17 | 5 contacts → | ||||
Strong evidence “We are an entrepreneurial pharmaceutical company working to drive change in rare diseases.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 18 | 4 contacts → | ||||
Strong evidence “Bloomsbury Genetic Therapies ("Bloomsbury") is a clinical-stage biotechnology company, developing potentially curative treatments for patients suffering from rare neurological and…” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 19 | 3 contacts → | ||||
Strong evidence “Oranda Therapeutics is a rare disease company, focused on acquiring, developing and commercialising medicines that improve the lives of patients in areas of high unmet need.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 20 | 1 contacts → | ||||
Strong evidence “We help companies developing new therapies for rare and genetic diseases to find and hire executive level talent for their senior medical and commercial roles across Europe.” Source: RevenueBase Database · checked Sep 21, 2026Thanks. The Data Team will review it. | |||||
| 21 | 1 contacts → | ||||
Strong evidence “ZYTHERA is at the forefront of developing groundbreaking treatments for lysosomal storage diseases (LSDs), a diverse group rare metabolic disorders caused by enzyme deficiencies…” Source: RevenueBase Database · checked Sep 21, 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: rare genetic disease 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 rare genetic disease treatment companies.
- Partial evidence
- Its own public description names part of rare genetic disease treatment companies, so it works in or next to the category without describing itself that way.
- Indirect evidence
- The description shown does not name rare genetic disease 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 21 of 29 rare genetic disease treatment companies based in the United Kingdom
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Rare genetic disease treatment companies based in the United Kingdom at a glance
As of Sep 21, 2026Companies by employee count
Email recency for this list is measured worldwide rather than for United Kingdom, so it is reported there. Company counts and contact totals on this page are exact for United Kingdom, over 29 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 rare genetic disease 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.
- What qualifies
- Companies headquartered in the United Kingdom whose own description of what they do matches “rare genetic disease 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 rare genetic disease 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 rare genetic disease treatment companies, and records with no verified work email. Companies headquartered outside United Kingdom 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, 808 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 21, 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 rare genetic disease 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 rare genetic disease treatment companies are there based in the United Kingdom?
RevenueBase identified 29 rare genetic disease treatment companies headquartered in the United Kingdom 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?
Meiragtx, headquartered in York, tops this list with the largest team on record.
Which city has the most rare genetic disease treatment companies?
London leads with 10 headquartered companies, followed by Cambridge (2) and Stevenage (2).
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 808 verified decision-maker contacts tracked across these 29 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 21, 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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