Company lists · Data refreshed September 2026

Top 25 Muscular dystrophy gene therapy companies based in the United States

RevenueBase identified 57 company matches for muscular dystrophy gene therapy companies associated with the United States as of September 2026 and tracks 3,595 professional contacts across them, including 2,602 with verified work emails. Cambridge is the largest hub.

This list covers company matches for muscular dystrophy 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
57Companies
3,595Contacts tracked
2,602Verified emails
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The 25 largest muscular dystrophy gene therapy companies based in the United States

Ranked by team size
Top 25 of 57 company matches for muscular dystrophy 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 matchWaltham, MA, United StatesHQ recorded Sep 20, 202651-200256 contacts →

Exact match

“Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
4Exact matchBoston, MA, United StatesHQ recorded Sep 20, 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 20, 2026 · View company records in RevenueBase
5Exact matchPittsburgh, PA, United StatesHQ recorded Sep 20, 2026201-500205 contacts →

Exact match

“Cook MyoSite is on a mission to make regenerative medicine a part of everyday medicine.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
6Exact 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
7Exact matchParamus, NJ, United StatesHQ recorded Sep 20, 202651-200155 contacts →

Exact match

“NS Pharma, Inc.(NSP) is a biopharmaceutical leader in delivering life-changing care to people living with complex, rare diseases, backed by over 100 years of Nippon Shinyaku Co.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
8Exact matchBoulder, CO, United StatesHQ recorded Sep 20, 202651-200142 contacts →

Exact match

“Edgewise Therapeutics, Inc.(Nasdaq: EWTX) is a leading clinical-stage biopharmaceutical company focused on cardiovascular diseases.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
9Exact matchWaltham, MA, United StatesHQ recorded Sep 20, 202651-20062 contacts →

Exact match

“We are a team with decades of proven success in translating science from bench to bedside across many companies and medicines, in gene therapy and beyond.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
10Exact matchCarlsbad, CA, United StatesHQ recorded Sep 20, 202651-20057 contacts →

Exact match

“We are a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC™ gene targeted chimera small molecules.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
11Exact matchPhiladelphia, PA, United StatesHQ recorded Sep 20, 202651-20056 contacts →

Exact match

“GEMMABio, a new therapeutics company, will serve as the research and innovation arm to speed the research of and global access to life-changing advanced therapies for those living…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
12Exact matchWashington Dc, United StatesHQ recorded Sep 20, 202611-5052 contacts →

Exact match

“Parent Project Muscular Dystrophy fights to end Duchenne.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
13Exact matchNewport Beach, CA, United StatesHQ recorded Sep 20, 202611-5035 contacts →

Exact match

“CureDuchenne is the global leader in Duchenne research, patient care and innovation.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
14Exact matchUnited StatesHQ recorded Sep 20, 20261,001-5,00035 contacts →

Exact match

“Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
15Exact matchMonrovia, CA, United StatesHQ recorded Sep 20, 2026n/a35 contacts →

Exact match

“Rampart Bioscience is evolving a new class of medicines for patients with genetically driven diseases.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
16Exact matchNY, United StatesHQ recorded Sep 20, 202651-20029 contacts →

Exact match

“At Prevail Therapeutics, we are developing novel gene therapies that target the root genetic causes of Parkinson’s disease and other neurodegenerative disorders.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
17Exact matchCambridge, MA, United StatesHQ recorded Sep 20, 202651-20020 contacts →

Exact match

“Our vision is to bring personalized gene therapy to the world.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
18Exact matchSeattle, WA, United StatesHQ recorded Sep 20, 20261-1018 contacts →

Exact match

“Our mission is to orchestrate a cure for muscular dystrophies caused by dysferlin protein deficiency.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
19Exact matchAlachua, FL, United StatesHQ recorded Sep 20, 202651-20015 contacts →

Exact match

Recorded description not available.

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
20Exact matchPlymouth, MA, United StatesHQ recorded Sep 20, 202611-5014 contacts →

Exact match

“In 2001, Christine and Stephen McSherry started Jett Foundation after learning the devastating news that their then five-year-old son, Jett, had Duchenne muscular dystrophy.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
21Exact matchGlendale, CA, United StatesHQ recorded Sep 20, 20261-109 contacts →

Exact match

“It is simple, the cure is the goal, we don’t just cover symptoms.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
22Exact matchNeedham, MA, United StatesHQ recorded Sep 20, 20261-109 contacts →

Exact match

“MLAB Biosciences is a biotechnology company that leverages its proprietary DIRECT™ platform to discover and develop novel antibody-based therapies for muscular dystrophies…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
23Exact matchBala Cynwyd, PA, United StatesHQ recorded Sep 20, 202611-508 contacts →

Exact match

“With offices in Philadelphia and Boston, SwanBio Therapeutics is developing leading-edge therapies to deliver dramatic clinical efficacy for the treatment of neurological diseases.”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
24Exact matchSan Diego, CA, United StatesHQ recorded Sep 20, 202611-507 contacts →

Exact match

“Kate Therapeutics (KateTx) is a patient-focused biotechnology company developing adeno-associated virus (AAV)-based gene therapies to treat genetically defined muscle and heart…”

Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase
25Exact matchColumbus, OH, United StatesHQ recorded Sep 20, 20261-106 contacts →

Exact match

Recorded description not available.

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

Showing 25 of 57 muscular dystrophy gene therapy companies based in the United States

See the full list, filter by size, metro and specialty, and access 2,602 tracked professional contacts with verified work emails. Direct dials are available where recorded.

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Muscular dystrophy gene therapy companies based in the United States at a glance

As of Sep 20, 2026
72.4%Verified-email coverage
63.1Average contacts per company
1-10Median size (employees)
8.8%Largest-city concentration · Cambridge
Sep 20, 2026Page data refreshed

Companies by employee count

1-10
31
11-50
9
51-200
12
201-500
1
501-1,000
2
1,001-5,000
2

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 57 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 “muscular dystrophy 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 2,602 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 “muscular dystrophy gene therapy companies”; each Exact match badge shows search membership, not an individual similarity or quality score.

How many muscular dystrophy gene therapy companies are there based in the United States?

RevenueBase identified 57 company matches for muscular dystrophy 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 muscular dystrophy gene therapy companies?

Cambridge leads with 5 company records, followed by Boston (3) and Reno (3).

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 2,602 professional contacts with verified work emails tracked across these 57 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.