Company lists · Data refreshed September 2026

Top 25 Neurological disorder gene therapy companies based in the United States

RevenueBase identified 234 company matches for neurological disorder gene therapy companies associated with the United States as of September 2026 and tracks 9,370 professional contacts across them, including 5,445 with verified work emails. Boston is the largest hub.

This list covers company matches for neurological disorder gene therapy companies associated with the United States, found by RevenueBase SmartSearch using the meaning of business descriptions in its company graph at Exact. Ranked by professionals tracked, largest first. Rows show recorded descriptions where available, Exact search membership and the snapshot date. Data refreshed September 20, 2026.

Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase SmartSearch, 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.

How to read this list RevenueBase SmartSearch · Exact Recorded Sep 20, 2026 How this list was built
234Companies
9,370Contacts tracked
5,445Verified emails
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The 25 largest neurological disorder gene therapy companies based in the United States

Ranked by team size
Top 25 of 234 company matches for neurological disorder gene therapy companies based in the United States, ordered by professional contacts tracked in RevenueBase, largest first. Recorded Sep 20, 2026. The RevenueBase SmartSearch column shows Exact match membership in this saved search. Click a contact count to view that company's people. Totals use company locations recorded by RevenueBase; the displayed preview is a smaller selection.
#CompanyRevenueBase SmartSearchHeadquartersEmployeesContacts
1Exact matchCambridge, MA, United StatesHQ recorded Sep 20, 2026501-1,0001,359 contacts →

Exact match

“Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
2Exact matchResearch Triangle Park, NC, United StatesHQ recorded Sep 20, 2026501-1,000510 contacts →

Exact match

“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…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
3Exact matchCambridge, MA, United StatesHQ recorded Sep 20, 2026201-500499 contacts →

Exact match

“BlueRock Therapeutics is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
4Exact matchColumbus, OH, United StatesHQ recorded Sep 20, 2026201-500456 contacts →

Exact match

“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…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
5Exact matchCambridge, MA, United StatesHQ recorded Sep 20, 2026n/a346 contacts →

Exact match

“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…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
6Exact matchCranbury, NJ, United StatesHQ recorded Sep 20, 2026201-500258 contacts →

Exact match

“Rocket Pharma (NASDAQ: RCKT) is a fully-integrated, clinical-stage company seeking gene therapy cures.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
7Exact matchPearl River, NY, United StatesHQ recorded Sep 20, 2026201-500229 contacts →

Exact match

“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…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
8Exact matchMorrisville, NC, United StatesHQ recorded Sep 20, 202651-200211 contacts →

Exact match

“Our mission is to revolutionize medicine, with the ultimate goal of eliminating human suffering and enabling people to live without the burden of disease.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
9Exact matchLexington, MA, United StatesHQ recorded Sep 20, 202651-200208 contacts →

Exact match

“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 –…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
10Exact matchBoston, MA, United StatesHQ recorded Sep 20, 2026201-500203 contacts →

Exact match

“Praxis Precision Medicines is a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, translating insights from genetic…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
11Exact matchSouth San Francisco, CA, United StatesHQ recorded Sep 20, 202651-200192 contacts →

Exact match

“We are a late-stage clinical biotechnology company with a mission to make degenerative brain disorders history.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
12Exact matchSan Diego, CA, United StatesHQ recorded Sep 20, 202651-200177 contacts →

Exact match

“Neurelis, Inc, is dedicated to innovating life-changing therapeutics that help people with neurological conditions gain greater control over their journeys.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
13Exact matchRedwood City, CA, United StatesHQ recorded Sep 20, 202651-200177 contacts →

Exact match

“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…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
14Exact matchBoston, MA, United StatesHQ recorded Sep 20, 202651-200160 contacts →

Exact match

“Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
15Exact matchSouth San Francisco, CA, United StatesHQ recorded Sep 20, 202651-200155 contacts →

Exact match

“Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
16Exact matchSolana Beach, CA, United StatesHQ recorded Sep 20, 202651-200132 contacts →

Exact match

“ClearPoint Neuro is a device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
17Exact matchDallas, TX, United StatesHQ recorded Sep 20, 202651-200122 contacts →

Exact match

“Taysha Gene Therapies is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
18Exact matchMalvern, PA, United StatesHQ recorded Sep 20, 202651-200121 contacts →

Exact match

“Ocugen, Inc.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
19Exact matchNew York, NY, United StatesHQ recorded Sep 20, 202651-200109 contacts →

Exact match

“Neurogene is accelerating development of new genetic medicines to people with devastating neurological diseases and their families.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
20Exact matchNewbury Park, CA, United StatesHQ recorded Sep 20, 202651-200109 contacts →

Exact match

“Capsida Biotherapeutics Inc.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
21Exact matchMenlo Park, NC, United StatesHQ recorded Sep 20, 202611-5088 contacts →

Exact match

“ReCode Therapeutics is a clinical-stage genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
22Exact matchBoston, MA, United StatesHQ recorded Sep 20, 202651-20087 contacts →

Exact match

“Rapport Therapeutics aims to transform the treatment of neurological disorders through a precision medicine approach, precisely targeting disease-driving neurocircuits by…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
23Exact matchNew York, NY, United StatesHQ recorded Sep 20, 202611-5085 contacts →

Exact match

“Based in New York City, Lexeo Therapeutics is a clinical-stage genetic medicines company dedicated to transforming healthcare by applying pioneering science to fundamentally…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
24Exact matchWatertown, MA, United StatesHQ recorded Sep 20, 202651-20083 contacts →

Exact match

“Brain diseases collectively represent one of the largest areas of unmet medical need, affecting upwards of 1.5 billion patients globally.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
25Exact matchPhiladelphia, PA, United StatesHQ recorded Sep 20, 2026501-1,00076 contacts →

Exact match

“At Spark® Therapeutics, we believe in a world where no life is limited by disease.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase

Showing 25 of 234 neurological disorder gene therapy companies based in the United States

See the full list, filter by size, metro and specialty, and access 5,445 tracked professional contacts with verified work emails. Direct dials are available where recorded.

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Neurological disorder gene therapy companies based in the United States at a glance

As of Sep 20, 2026
58.1%Verified-email coverage
40.0Average contacts per company
11-50Median size (employees)
9.0%Largest-city concentration · Boston
Sep 20, 2026Page data refreshed

Companies by employee count

1-10
109
11-50
69
51-200
41
201-500
9
501-1,000
4
1,001-5,000
1
10001+
1

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 234 companies.

How this list was built

The recorded descriptions help you assess these company matches. Check a company’s website for its current services and locations.

Source
The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase SmartSearch, the natural-language company search available to every RevenueBase user.
What qualifies
Company records associated with the United States whose business descriptions match “neurological disorder gene therapy companies” by meaning at the Exact setting. Descriptions are compiled in the RevenueBase company graph from public websites, profiles and company information where available; they are not guaranteed quotations from the company's website.
RevenueBase SmartSearch
Describe a niche company in your own words to find businesses by meaning. Exact, Similar, Related and Broad control the breadth of RevenueBase SmartSearch. This saved list uses Exact. Each row's Exact match badge shows inclusion in that search, not an individual score or a service-quality judgment.
Public preview
The public preview omits records without a verified work email. Where headquarters information is recorded, preview rows are also filtered to this page’s location. Dataset totals and geographic summaries describe the full matched records before these preview filters.
Match quality
RevenueBase's August 25, 2026 study assessed 4,198 description/company pairs across matching settings. The publisher reports 86% good or better at Exact. This evaluates the method across queries; it does not audit this individual list. Recorded company descriptions, where available, help you judge each match yourself. Read the full methodology: how the lists are built and what the study measures.
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 displayed company carries at least one verified work email. RevenueBase tracks 5,445 professional contacts with verified work emails across the full matched dataset. 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 20, 2026. Lists refresh periodically as the graph is re-checked.
Record context
Company records and descriptions can change. Check the company's website for its current services and locations.

Frequently asked

What is RevenueBase SmartSearch?

RevenueBase SmartSearch finds niche companies by the meaning of their business descriptions in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit your description. This list uses Exact for “neurological disorder gene therapy companies”; each Exact match badge shows search membership, not an individual similarity or quality score.

How many neurological disorder gene therapy companies are there based in the United States?

RevenueBase identified 234 company matches for neurological disorder gene therapy companies associated with the United States as of September 2026, using the meaning of business descriptions at Exact. This is the recorded matched set in the RevenueBase company graph.

What is the largest company on this list?

Sarepta Therapeutics tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.

Which city has the most neurological disorder gene therapy companies?

Boston leads with 21 company records, followed by Cambridge (15) and San Diego (11).

How do I get contact information for these companies?

A free RevenueBase account lets you run this exact search and work limited free results with 500 credits to start. Upgrading provides access to the 5,445 professional contacts with verified work emails tracked across these 234 companies; direct dials are available where recorded.

How current is this list?

The list refreshes periodically as the company graph is re-checked. This page's figures were pulled on September 20, 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 SmartSearch, 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.