Company lists · Data refreshed September 2026

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

RevenueBase identified 405 company matches for neurodegenerative disorder gene therapy companies associated with the United States as of September 2026 and tracks 14,836 professional contacts across them, including 7,612 with verified work emails. Cambridge is the largest hub.

This list covers company matches for neurodegenerative 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 19, 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 19, 2026 How this list was built
405Companies
14,836Contacts tracked
7,612Verified emails
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The 25 largest neurodegenerative disorder gene therapy companies based in the United States

Ranked by team size
Top 25 of 405 company matches for neurodegenerative disorder gene therapy companies based in the United States, ordered by professional contacts tracked in RevenueBase, largest first. Recorded Sep 19, 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 19, 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 19, 2026 · View company records in RevenueBase
2Exact matchResearch Triangle Park, NC, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
3Exact matchCambridge, MA, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
4Exact matchColumbus, OH, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
5Exact matchRockville, MD, United StatesHQ recorded Sep 19, 2026201-500393 contacts →

Exact match

“REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.”

Recorded in the RevenueBase company graph · Sep 19, 2026 · View company records in RevenueBase
6Exact matchCambridge, MA, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
7Exact matchSeattle, WA, United StatesHQ recorded Sep 19, 2026201-500287 contacts →

Exact match

“Sana Biotechnology is focused on utilizing engineered cells as medicines for patients.”

Recorded in the RevenueBase company graph · Sep 19, 2026 · View company records in RevenueBase
8Exact matchCranbury, NJ, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
9Exact matchBoston, MA, United StatesHQ recorded Sep 19, 202651-200239 contacts →

Exact match

“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…”

Recorded in the RevenueBase company graph · Sep 19, 2026 · View company records in RevenueBase
10Exact matchEmeryville, CA, United StatesHQ recorded Sep 19, 202651-200236 contacts →

Exact match

“4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…”

Recorded in the RevenueBase company graph · Sep 19, 2026 · View company records in RevenueBase
11Exact matchPearl River, NY, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
12Exact matchMorrisville, NC, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
13Exact matchLexington, MA, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
14Exact matchCleveland, OH, United StatesHQ recorded Sep 19, 2026201-500207 contacts →

Exact match

“Abeona Therapeutics Inc.”

Recorded in the RevenueBase company graph · Sep 19, 2026 · View company records in RevenueBase
15Exact matchBoston, MA, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
16Exact matchSouth San Francisco, CA, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
17Exact matchBedford, MA, United StatesHQ recorded Sep 19, 2026201-500183 contacts →

Exact match

“Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine.”

Recorded in the RevenueBase company graph · Sep 19, 2026 · View company records in RevenueBase
18Exact matchSan Diego, CA, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
19Exact matchRedwood City, CA, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
20Exact matchBoston, MA, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
21Exact matchSouth San Francisco, CA, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
22Exact matchDallas, TX, United StatesHQ recorded Sep 19, 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 19, 2026 · View company records in RevenueBase
23Exact matchMalvern, PA, United StatesHQ recorded Sep 19, 202651-200121 contacts →

Exact match

“Ocugen, Inc.”

Recorded in the RevenueBase company graph · Sep 19, 2026 · View company records in RevenueBase
24Exact matchRedwood City, CA, United StatesHQ recorded Sep 19, 202651-200116 contacts →

Exact match

“Maplight is a biopharmaceutical company discovering and developing novel therapeutics for patients with disorders of the central nervous system (CNS).”

Recorded in the RevenueBase company graph · Sep 19, 2026 · View company records in RevenueBase
25Exact matchCambridge, MA, United StatesHQ recorded Sep 19, 202651-200115 contacts →

Exact match

“Obsidian Therapeutics is pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases.”

Recorded in the RevenueBase company graph · Sep 19, 2026 · View company records in RevenueBase

Showing 25 of 405 neurodegenerative disorder gene therapy companies based in the United States

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

As of Sep 19, 2026
51.3%Verified-email coverage
36.6Average contacts per company
11-50Median size (employees)
7.9%Largest-city concentration · Cambridge
Sep 19, 2026Page data refreshed

Companies by employee count

1-10
198
11-50
121
51-200
62
201-500
16
501-1,000
5
1,001-5,000
2
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 405 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 “neurodegenerative 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 7,612 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 19, 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 “neurodegenerative disorder gene therapy companies”; each Exact match badge shows search membership, not an individual similarity or quality score.

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

RevenueBase identified 405 company matches for neurodegenerative 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 neurodegenerative disorder gene therapy companies?

Cambridge leads with 32 company records, 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 limited free results with 500 credits to start. Upgrading provides access to the 7,612 professional contacts with verified work emails tracked across these 405 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 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 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.