Company lists · Data refreshed September 2026
Top 25 Cystic fibrosis gene therapy companies based in the United States
RevenueBase identified 54 cystic fibrosis gene therapy companies headquartered in the United States as of September 2026 and tracks 10,829 professional contacts across them, including 6,859 with verified work emails. Boston is the largest hub.
This list covers cystic fibrosis 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 cystic fibrosis gene therapy companies based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 8,099 contacts → | ||||
Indirect evidence “Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and has made significant…” 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 | 236 contacts → | ||||
Partial evidence “4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 5 | 189 contacts → | ||||
Partial evidence “Verve Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, is developing a new class of genetic medicines for cardiovascular disease with the potential to transform…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 6 | 160 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, 2026Thanks. The Data Team will review it. | |||||
| 7 | 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. | |||||
| 8 | 62 contacts → | ||||
Strong evidence “We are a life sciences company that is building on over a decade of extensive research on the genetic mutations associated with cystic fibrosis.” 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 “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, 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 | 35 contacts → | ||||
Strong 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 19, 2026Thanks. The Data Team will review it. | |||||
| 13 | 35 contacts → | ||||
Indirect 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. | |||||
| 14 | 29 contacts → | ||||
Strong evidence “Born from purpose and for patient access, Vector BioMed is a Public Benefit Corporation engineering the next frontier of lentivirus-based cell and gene therapy.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 15 | 26 contacts → | ||||
Indirect evidence “Anagram is a clinical stage company leveraging proprietary enzyme technologies and expertise in gastrointestinal diseases to solve complex problems and advance a pipeline of…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 16 | 22 contacts → | ||||
Strong evidence “Spirovant Sciences is developing novel gene therapies for cystic fibrosis and other pulmonary diseases.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 17 | 21 contacts → | ||||
Partial 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. | |||||
| 18 | 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. | |||||
| 19 | 16 contacts → | ||||
Strong evidence “Expanding Therapeutic Potential with Novel Parvovirus Gene Therapy” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 20 | 12 contacts → | ||||
Partial evidence “We develop first-in-class RNA-based therapies, with lead programs in Head & Neck Squamous Cell Carcinoma (HNSCC) and Facioscalpulohumeral Muscular Dystrophy (FSHD).” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 21 | 10 contacts → | ||||
Partial evidence “Optimeos Life Sciences is pioneering a novel targeted gene delivery approach to advance medical treatment and patient care.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 22 | 10 contacts → | ||||
Strong evidence “XyloCor Therapeutics is a development-stage, biotechnology company dedicated to developing first-in-class gene therapies for patients with unmet medical need in cardiovascular…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 23 | 10 contacts → | ||||
Strong evidence “StrideBio is a product-driven, fully integrated gene therapy company focused on creating and developing innovative genetic medicines with life-changing or curative potential for…” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 24 | 7 contacts → | ||||
Strong evidence “At Elpida Therapeutics our main goal is to deliver Gene Therapies to patients in need.” Source: RevenueBase Database · checked Sep 19, 2026Thanks. The Data Team will review it. | |||||
| 25 | 7 contacts → | ||||
Strong evidence “Aruvant’s mission is to bring hope to patients living with rare diseases by developing life changing and potentially curative gene therapies, with a near-term focus on sickle cell…” 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: cystic fibrosis 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 cystic fibrosis gene therapy companies.
- Partial evidence
- Its own public description names part of cystic fibrosis 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 cystic fibrosis 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 54 cystic fibrosis gene therapy companies based in the United States
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Cystic fibrosis 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 54 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 cystic fibrosis 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 “cystic fibrosis 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 cystic fibrosis 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 cystic fibrosis 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, 6,859 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 cystic fibrosis 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 cystic fibrosis gene therapy companies are there based in the United States?
RevenueBase identified 54 cystic fibrosis 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?
Vertex Pharmaceuticals, headquartered in Boston, tops this list with the largest team on record.
Which city has the most cystic fibrosis gene therapy companies?
Boston leads with 8 headquartered companies, followed by New York (4) and Cambridge (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 6,859 verified decision-maker contacts tracked across these 54 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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