Company lists · Data refreshed September 2026

Top 25 Beta thalassemia gene therapy companies worldwide

RevenueBase identified 175 beta thalassemia gene therapy companies worldwide as of September 2026 and tracks 6,164 professional contacts across them, including 3,150 with verified work emails. The United States is the largest market and Cambridge the largest hub.

This list covers beta thalassemia 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 beta thalassemia 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.

How to read this list HQ checked on every row RevenueBase Smart Search rating on every row Refreshed Sep 19, 2026 86% judged on-target in blind review
175Companies
6,164Contacts tracked
3,150Verified emails
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The 25 largest beta thalassemia gene therapy companies worldwide

Ranked by team size
Top 25 of 175 beta thalassemia gene therapy companies worldwide, ranked by team size, largest first. Data refreshed Sep 19, 2026. RevenueBase Smart Search rates how each company's information relates to beta thalassemia gene 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
1Strong evidenceColumbus, OH, United StatesHQ checked Sep 19, 2026201-500456 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, 2026
2Indirect evidenceLondon/new York, United KingdomHQ checked Sep 19, 202651-200398 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, 2026
3Strong evidenceRockville, MD, United StatesHQ checked Sep 19, 2026201-500393 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, 2026
4Strong evidenceAmsterdam, United StatesHQ checked Sep 19, 202651-200349 contacts →

Strong evidence

“uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results.”

Source: RevenueBase Database · checked Sep 19, 2026
5Indirect evidenceSeattle, WA, United StatesHQ checked Sep 19, 2026201-500287 contacts →

Indirect evidence

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

Source: RevenueBase Database · checked Sep 19, 2026
6Partial evidenceBrisbane, CA, AustraliaHQ checked Sep 19, 2026201-500270 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, 2026
7Indirect evidenceParis, United StatesHQ checked Sep 19, 202651-200236 contacts →

Indirect evidence

“Cellectis is a global, clinical-stage biopharmaceutical company.”

Source: RevenueBase Database · checked Sep 19, 2026
8Strong evidenceLondon, United KingdomHQ checked Sep 19, 202651-200221 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, 2026
9Partial evidenceCleveland, OH, United StatesHQ checked Sep 19, 2026201-500207 contacts →

Partial evidence

“Abeona Therapeutics Inc.”

Source: RevenueBase Database · checked Sep 19, 2026
10Strong evidenceBoston, MA, United StatesHQ checked Sep 19, 202651-200160 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, 2026
11Strong evidenceSouth San Francisco, CA, United StatesHQ checked Sep 19, 202651-200155 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, 2026
12Indirect evidenceMalvern, PA, United StatesHQ checked Sep 19, 202651-200121 contacts →

Indirect evidence

“Ocugen, Inc.”

Source: RevenueBase Database · checked Sep 19, 2026
13Indirect evidenceNewbury Park, CA, United StatesHQ checked Sep 19, 202651-200109 contacts →

Indirect evidence

“Capsida Biotherapeutics Inc.”

Source: RevenueBase Database · checked Sep 19, 2026
14Strong evidenceStevenage, United KingdomHQ checked Sep 19, 202611-5098 contacts →

Strong evidence

“Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions.”

Source: RevenueBase Database · checked Sep 19, 2026
15Partial evidencePhiladelphia, PA, United StatesHQ checked Sep 19, 2026501-1,00076 contacts →

Partial evidence

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

Source: RevenueBase Database · checked Sep 19, 2026
16Partial evidenceBoston, MA, United StatesHQ checked Sep 19, 202611-5066 contacts →

Partial evidence

“Kelonia is pioneering a new wave of genetic medicines using its next generation gene delivery platform.”

Source: RevenueBase Database · checked Sep 19, 2026
17Indirect evidenceLondon, United KingdomHQ checked Sep 19, 202611-5063 contacts →

Indirect evidence

“At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal…”

Source: RevenueBase Database · checked Sep 19, 2026
18Strong evidenceWaltham, MA, United StatesHQ checked Sep 19, 202651-20062 contacts →

Strong evidence

“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.”

Source: RevenueBase Database · checked Sep 19, 2026
19Partial evidencePhiladelphia, PA, United StatesHQ checked Sep 19, 202651-20056 contacts →

Partial evidence

“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…”

Source: RevenueBase Database · checked Sep 19, 2026
20Partial evidenceRedwood City, CA, United StatesHQ checked Sep 19, 202651-20056 contacts →

Partial evidence

“Jasper Therapeutics is a biotechnology company on a mission to make safer and potentially curative therapy possible for more patients in need.”

Source: RevenueBase Database · checked Sep 19, 2026
21Strong evidenceLake Forest, IL, United StatesHQ checked Sep 19, 202651-20053 contacts →

Strong evidence

“Jaguar Gene Therapy, LLC is dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable…”

Source: RevenueBase Database · checked Sep 19, 2026
22Strong evidenceAlachua, FL, United StatesHQ checked Sep 19, 202651-20048 contacts →

Strong evidence

“AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe…”

Source: RevenueBase Database · checked Sep 19, 2026
23Indirect evidenceNantes, FranceHQ checked Sep 19, 202651-20041 contacts →

Indirect evidence

“Laboratoire de Thérapie génique de Nantes”

Source: RevenueBase Database · checked Sep 19, 2026
24Indirect evidencePhiladelphia, PA, United StatesHQ checked Sep 19, 202611-5039 contacts →

Indirect evidence

“iECURE is a clinical-stage genome editing company dedicated to transforming the lives of patients with devastating neurometabolic disorders by harnessing new advancements in…”

Source: RevenueBase Database · checked Sep 19, 2026
25Strong evidenceDresden, GermanyHQ checked Sep 19, 202611-5035 contacts →

Strong evidence

“Seamless Therapeutics is changing the paradigm of gene editing through a pioneering approach to restore health in patients with severe conditions in a safe and precise manner.”

Source: RevenueBase Database · checked Sep 19, 2026

Showing 25 of 175 beta thalassemia gene therapy companies worldwide

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Beta thalassemia gene therapy companies worldwide at a glance

As of Sep 19, 2026
51.1%Verified-email coverage
35.2Average contacts per company
11-50Median size (employees)
4.0%Largest-city concentration · Cambridge
Sep 19, 2026Page data refreshed

Companies by employee count

1-10
78
11-50
46
51-200
33
201-500
14
501-1,000
2
1,001-5,000
1

Email recency is reported on each country list linked below.

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 beta thalassemia 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
Every country. Country and city lists for beta thalassemia gene therapy companies are linked below.
What qualifies
Companies worldwide whose own description of what they do matches “beta thalassemia 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 beta thalassemia 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 beta thalassemia 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, 3,150 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.

Beta thalassemia gene therapy companies by location

1 lists · largest first

Every Beta thalassemia 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 beta thalassemia 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 beta thalassemia gene therapy companies are there worldwide?

RevenueBase identified 175 beta thalassemia 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?

Forge Biologics, headquartered in Columbus, tops this list with the largest team on record.

Which country has the most beta thalassemia gene therapy companies?

The United States leads with 85 companies. Every beta thalassemia gene therapy companies list by country and city is linked on this page.

Which city has the most beta thalassemia gene therapy companies?

Cambridge leads with 7 headquartered companies, followed by London (5) and New York (5).

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 3,150 verified decision-maker contacts tracked across these 175 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.