Company lists · Data refreshed September 2026

Top 19 Spinal muscular atrophy therapy companies based in the United States

RevenueBase identified 35 spinal muscular atrophy therapy companies headquartered in the United States as of September 2026 and tracks 3,079 professional contacts across them, including 2,337 with verified work emails. Cambridge is the largest hub.

This list covers spinal muscular atrophy therapy companies headquartered in the United States, 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 22, 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.

How to read this list HQ checked on every row RevenueBase Smart Search rating on every row Refreshed Sep 22, 2026 86% judged on-target in blind review
35Companies
3,079Contacts tracked
2,337Verified emails
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The 19 largest spinal muscular atrophy therapy companies based in the United States

Ranked by team size
Top 19 of 35 spinal muscular atrophy therapy companies based in the United States, ranked by team size, largest first. Data refreshed Sep 22, 2026. RevenueBase Smart Search rates how each company's information relates to spinal muscular atrophy therapy companies: strong evidence (its own description names it), partial (names part of it), indirect (matched on the rest of its profile rather than the description shown). Click a contact count to view that company's people.
#CompanyRevenueBase Smart SearchHeadquartersEmployeesContacts
1Partial evidenceCambridge, MA, United StatesHQ checked Sep 22, 2026501-1,0001,359 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 22, 2026
2Partial evidenceResearch Triangle Park, NC, United StatesHQ checked Sep 22, 2026501-1,000510 contacts →

Partial 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 22, 2026
3Partial evidenceCambridge, MA, United StatesHQ checked Sep 22, 202651-200325 contacts →

Partial evidence

“Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need.”

Source: RevenueBase Database · checked Sep 22, 2026
4Partial evidenceWaltham, MA, United StatesHQ checked Sep 22, 202651-200256 contacts →

Partial evidence

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

Source: RevenueBase Database · checked Sep 22, 2026
5Partial evidenceBoston, MA, United StatesHQ checked Sep 22, 202651-200239 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 22, 2026
6Partial evidenceBoulder, CO, United StatesHQ checked Sep 22, 202651-200142 contacts →

Partial evidence

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

Source: RevenueBase Database · checked Sep 22, 2026
7Strong evidenceSchaumburg, IL, United StatesHQ checked Sep 22, 202611-5077 contacts →

Strong evidence

“Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA).”

Source: RevenueBase Database · checked Sep 22, 2026
8Partial evidenceUnited StatesHQ checked Sep 22, 20261,001-5,00035 contacts →

Partial evidence

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

Source: RevenueBase Database · checked Sep 22, 2026
9Indirect evidenceChapel Hill, NC, United StatesHQ checked Sep 22, 202611-5034 contacts →

Indirect evidence

“Bamboo represents a community of researchers, clinicians, parents, regulatory scientists, and business professionals committed to providing hope to patients suffering from…”

Source: RevenueBase Database · checked Sep 22, 2026
10Indirect evidenceSan Diego, CA, United StatesHQ checked Sep 22, 202611-5012 contacts →

Indirect evidence

“Epirium, a biopharmaceutical company based in San Diego, California, has identified and established an IP-protected platform of orally bioavailable small molecules that constitute…”

Source: RevenueBase Database · checked Sep 22, 2026
11Indirect evidenceNew York, NY, United StatesHQ checked Sep 22, 20261-1010 contacts →

Indirect evidence

“The mission of the SMA Foundation is to accelerate the development of a treatment for SMA, the number one genetic killer of infants and toddlers.”

Source: RevenueBase Database · checked Sep 22, 2026
12Indirect evidenceColumbus, OH, United StatesHQ checked Sep 22, 20261-106 contacts →

Indirect evidence

Source: RevenueBase Database · checked Sep 22, 2026
13Indirect evidenceReno, NV, United StatesHQ checked Sep 22, 20261-105 contacts →

Indirect evidence

“Sarcomatrix, Inc.”

Source: RevenueBase Database · checked Sep 22, 2026
14Indirect evidenceSanta Barbara, CA, United StatesHQ checked Sep 22, 20261-105 contacts →

Indirect evidence

“Mission: Transforming lives and building a kinder and more inclusive future through advocacy, research, and support.”

Source: RevenueBase Database · checked Sep 22, 2026
15Indirect evidenceCalabasas, CA, United StatesHQ checked Sep 22, 20261-102 contacts →

Indirect evidence

“Orphina is a pre-startup biotechnology company focused on preserving and prolonging muscle health.”

Source: RevenueBase Database · checked Sep 22, 2026
16Indirect evidenceLa Jolla, CA, United StatesHQ checked Sep 22, 202611-502 contacts →

Indirect evidence

“Genea Biocells is a neuromuscular disease-focused company based on a proprietary technology platform for the accelerated development of drugs to treat neuromuscular diseases -…”

Source: RevenueBase Database · checked Sep 22, 2026
17Partial evidenceOverland Park, KS, United StatesHQ checked Sep 22, 20261-102 contacts →

Partial evidence

“Shift Pharmaceuticals is developing Antisense Oligonucleotides as drug candidates for a variety of genetic disorders, including Charcot Marie Tooth Syndrome (CMT1A) and Spinal…”

Source: RevenueBase Database · checked Sep 22, 2026
18Partial evidenceChicago, United StatesHQ checked Sep 22, 20261-102 contacts →

Partial evidence

“Dystrogen Therapeutics is a clinical stage chimeric cell therapy company focusing on rare diseases.”

Source: RevenueBase Database · checked Sep 22, 2026
19Partial evidenceSan Diego, CA, United StatesHQ checked Sep 22, 20261-101 contacts →

Partial evidence

“Papillon Therapeutics is a biotechnology company advancing genetic medicines for life threatening conditions with neurologic disease, including Friedreich's ataxia, Danon disease…”

Source: RevenueBase Database · checked Sep 22, 2026

Showing 19 of 35 spinal muscular atrophy therapy companies based in the United States

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Spinal muscular atrophy therapy companies based in the United States at a glance

As of Sep 22, 2026
75.9%Verified-email coverage
88.0Average contacts per company
1-10Median size (employees)
11.4%Largest-city concentration · Cambridge
Sep 22, 2026Page data refreshed

Companies by employee count

1-10
19
11-50
7
51-200
5
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 35 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 spinal muscular atrophy 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.
What qualifies
Companies headquartered in the United States whose own description of what they do matches “spinal muscular atrophy 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 spinal muscular atrophy 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 spinal muscular atrophy therapy companies, and records with no verified work email. Companies headquartered outside United States 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, 2,337 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 22, 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 spinal muscular atrophy 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 spinal muscular atrophy therapy companies are there based in the United States?

RevenueBase identified 35 spinal muscular atrophy therapy companies headquartered in the United States 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 city has the most spinal muscular atrophy therapy companies?

Cambridge leads with 4 headquartered companies, followed by New York (2) and San Diego (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 2,337 verified decision-maker contacts tracked across these 35 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 22, 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.