Company lists · Data refreshed September 2026
Top 25 Hematological disease gene therapy companies based in the United States
RevenueBase identified 79 hematological disease gene therapy companies headquartered in the United States as of September 2026 and tracks 3,872 professional contacts across them, including 1,945 with verified work emails. Cambridge is the largest hub.
This list covers hematological disease gene 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 19, 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.
The 25 largest hematological disease gene therapy companies based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 510 contacts → | ||||
Strong 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 19, 2026Thanks. The Data Team will review it. | |||||
| 2 | 456 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, 2026Thanks. The Data Team will review it. | |||||
| 3 | 287 contacts → | ||||
Indirect evidence “Sana Biotechnology is focused on utilizing engineered cells as medicines for patients.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 4 | 279 contacts → | ||||
Strong evidence “Orca Bio is a late-stage biotechnology company developing high-precision cell therapies for the treatment of blood cancer and autoimmune diseases.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 5 | 139 contacts → | ||||
Partial evidence “Disc Medicine is a biopharmaceutical company dedicated to transforming the lives of patients with hematologic disorders.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 6 | 101 contacts → | ||||
Indirect evidence “Ensoma /en’sōmə/ is derived from the Greek phrase “en soma,” meaning in the body.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 7 | 76 contacts → | ||||
Strong evidence “At Spark® Therapeutics, we believe in a world where no life is limited by disease.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 8 | 66 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, 2026Thanks. The Data Team will review it. | |||||
| 9 | 62 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, 2026Thanks. The Data Team will review it. | |||||
| 10 | 56 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, 2026Thanks. The Data Team will review it. | |||||
| 11 | 53 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, 2026Thanks. The Data Team will review it. | |||||
| 12 | 48 contacts → | ||||
Partial evidence “GenVivo was founded on the idea that we can effectively treat cancer patients with the latest gene delivery technologies.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 13 | 48 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, 2026Thanks. The Data Team will review it. | |||||
| 14 | 36 contacts → | ||||
Strong evidence “Emendo Biotherapeutics develops next generation gene editing tools for genetic disorders, addressing the current technological gaps for realizing the promise of gene therapy.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 15 | 36 contacts → | ||||
Partial evidence “Atavistik Bio is a clinical stage biotechnology company developing novel allosteric therapeutics, with an internal focus on rare hematology.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 16 | 35 contacts → | ||||
Partial evidence “Rampart Bioscience is evolving a new class of medicines for patients with genetically driven diseases.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 17 | 34 contacts → | ||||
Partial evidence “Syros is committed to developing new standards of care for the frontline treatment of patients with hematologic malignancies.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 18 | 33 contacts → | ||||
Strong evidence “American Gene Technologies® International Inc.(AGT™) is a private biotechnology company pursuing cures and treatments for infectious diseases, cancers, and monogenic disorders.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 19 | 24 contacts → | ||||
Strong evidence “EXUMA Biotech is a clinical-stage biotechnology company developing next-generation in vivo cell and gene therapies.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 20 | 23 contacts → | ||||
Indirect evidence Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 21 | 21 contacts → | ||||
Strong evidence “ASC Therapeutics focuses on the development of curative therapies for rare diseases.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 22 | 20 contacts → | ||||
Strong evidence “Our vision is to bring personalized gene therapy to the world.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 23 | 19 contacts → | ||||
Indirect evidence Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 24 | 17 contacts → | ||||
Strong evidence “Homology Medicines is a genetic medicines company translating proprietary gene editing and gene therapy technologies into novel treatments for patients with rare diseases.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 25 | 17 contacts → | ||||
Strong evidence “Kamau Therapeutics is a clinical-stage, next-generation gene correction company harnessing high efficiency targeted gene integration to develop a new class of therapies with the…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
RevenueBase Smart Search
RevenueBase Smart Search assesses information from a company’s LinkedIn profile, information supplied directly by the company, its website, and other publicly available sources. It uses that information to rate how closely the company relates to this list’s category: hematological disease gene therapy companies. Every row carries its rating and the description the rating was read from, so each one can be checked rather than taken on trust.
- Strong evidence
- The company's own public description of its business names hematological disease gene therapy companies.
- Partial evidence
- Its own public description names part of hematological disease gene therapy companies, so it works in or next to the category without describing itself that way.
- Indirect evidence
- The description shown does not name hematological disease gene therapy companies. The match rests on the rest of the company's public profile rather than the one sentence quoted here, so this is the rating to check first.
RevenueBase Smart Search is how these lists are built: RevenueBase's data engine and AI assemble the companies that match a category defined in a user's own words, so a list can be about what companies actually do rather than about whichever industry code they were filed under.
Showing 25 of 79 hematological disease gene therapy companies based in the United States
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Hematological disease gene therapy companies based in the United States at a glance
As of Sep 19, 2026Companies by employee count
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 79 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 hematological disease 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.
- What qualifies
- Companies headquartered in the United States whose own description of what they do matches “hematological disease 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 hematological disease 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 hematological disease gene 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, 1,945 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.
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 hematological disease 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 hematological disease gene therapy companies are there based in the United States?
RevenueBase identified 79 hematological disease gene 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?
Askbio INC, headquartered in Research Triangle Park, tops this list with the largest team on record.
Which city has the most hematological disease gene therapy companies?
Cambridge leads with 7 headquartered companies, followed by Baltimore (3) and Boston (3).
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 1,945 verified decision-maker contacts tracked across these 79 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.
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