Company lists · Data refreshed September 2026
Top 25 Neurodegenerative disorder gene therapy companies worldwide
RevenueBase identified 513 neurodegenerative disorder gene therapy companies worldwide as of September 2026 and tracks 16,992 professional contacts across them, including 8,822 with verified work emails and 0 with mobile numbers. The United States is the largest market and Cambridge the largest hub.
This list covers neurodegenerative disorder gene therapy 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 neurodegenerative disorder gene therapy 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 neurodegenerative disorder gene therapy companies worldwide
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 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. | |||||
| 2 | 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. | |||||
| 3 | 499 contacts → | ||||
Partial evidence “BlueRock Therapeutics is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 4 | 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. | |||||
| 5 | 398 contacts → | ||||
Indirect 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. | |||||
| 6 | 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. | |||||
| 7 | 346 contacts → | ||||
Strong evidence “Sage Therapeutics is a biopharmaceutical company committed to developing novel therapies with the potential to transform the lives of people with debilitating disorders of the…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 8 | 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. | |||||
| 9 | 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. | |||||
| 10 | 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. | |||||
| 11 | 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. | |||||
| 12 | 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. | |||||
| 13 | 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. | |||||
| 14 | 229 contacts → | ||||
Partial evidence “Acorda Therapeutics is a publicly traded (NASDAQ: ACOR) biotechnology company whose mission is to develop therapies that restore neurological function and improve the lives of…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 15 | 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 19, 2026Thanks. The Data Team will review it. | |||||
| 16 | 211 contacts → | ||||
Indirect 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. | |||||
| 17 | 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. | |||||
| 18 | 207 contacts → | ||||
Partial evidence “Abeona Therapeutics Inc.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 19 | 203 contacts → | ||||
Indirect evidence “Praxis Precision Medicines is a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, translating insights from genetic…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 20 | 192 contacts → | ||||
Partial evidence “We are a late-stage clinical biotechnology company with a mission to make degenerative brain disorders history.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 21 | 183 contacts → | ||||
Partial evidence “Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 22 | 177 contacts → | ||||
Partial evidence “Neurelis, Inc, is dedicated to innovating life-changing therapeutics that help people with neurological conditions gain greater control over their journeys.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 23 | 177 contacts → | ||||
Strong evidence “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: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 24 | 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. | |||||
| 25 | 155 contacts → | ||||
Strong evidence “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: 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: neurodegenerative disorder 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 neurodegenerative disorder gene therapy companies.
- Partial evidence
- Its own public description names part of neurodegenerative disorder 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 neurodegenerative disorder 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 513 neurodegenerative disorder gene therapy companies worldwide
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Neurodegenerative disorder gene therapy companies worldwide at a glance
As of Sep 19, 2026Companies by employee count
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 neurodegenerative disorder 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.
- Coverage
- 4 countries, largest first: the United States, France, the United Kingdom, Canada. Country and city lists for neurodegenerative disorder gene therapy companies are linked below.
- What qualifies
- Companies worldwide whose own description of what they do matches “neurodegenerative disorder 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 neurodegenerative disorder 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 neurodegenerative disorder gene therapy 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, 8,822 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.
Neurodegenerative disorder gene therapy companies by location
4 lists · largest firstEvery Neurodegenerative disorder gene therapy 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 neurodegenerative disorder 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 neurodegenerative disorder gene therapy companies are there worldwide?
RevenueBase identified 513 neurodegenerative disorder gene therapy 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?
Sarepta Therapeutics, headquartered in Cambridge, tops this list with the largest team on record.
Which country has the most neurodegenerative disorder gene therapy companies?
The United States leads with 405 companies, followed by France (44) and the United Kingdom (36). Every neurodegenerative disorder gene therapy companies list by country and city is linked on this page.
Which city has the most neurodegenerative disorder gene therapy companies?
Cambridge leads with 37 headquartered companies, followed by Boston (29) and San Diego (24).
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 8,822 verified decision-maker contacts tracked across these 513 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.