Company lists · Data refreshed September 2026
Top 25 Lysosomal storage disease gene therapy companies based in the United States
RevenueBase identified 74 lysosomal storage disease gene therapy companies headquartered in the United States as of September 2026 and tracks 4,289 professional contacts across them, including 1,826 with verified work emails. Philadelphia is the largest hub.
This list covers lysosomal storage 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 20, 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 lysosomal storage 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 20, 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 20, 2026Thanks. The Data Team will review it. | |||||
| 3 | 236 contacts → | ||||
Strong 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 20, 2026Thanks. The Data Team will review it. | |||||
| 4 | 211 contacts → | ||||
Partial evidence “Our mission is to revolutionize medicine, with the ultimate goal of eliminating human suffering and enabling people to live without the burden of disease.” Source: RevenueBase Database · checked Sep 20, 2026Thanks. The Data Team will review it. | |||||
| 5 | 208 contacts → | ||||
Partial evidence “Voyager Therapeutics, Inc.(Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure –…” Source: RevenueBase Database · checked Sep 20, 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 20, 2026Thanks. The Data Team will review it. | |||||
| 7 | 155 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 20, 2026Thanks. The Data Team will review it. | |||||
| 8 | 121 contacts → | ||||
Indirect evidence “Ocugen, Inc.” Source: RevenueBase Database · checked Sep 20, 2026Thanks. The Data Team will review it. | |||||
| 9 | 109 contacts → | ||||
Partial evidence “Neurogene is accelerating development of new genetic medicines to people with devastating neurological diseases and their families.” Source: RevenueBase Database · checked Sep 20, 2026Thanks. The Data Team will review it. | |||||
| 10 | 88 contacts → | ||||
Strong evidence “ReCode Therapeutics is a clinical-stage genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics.” Source: RevenueBase Database · checked Sep 20, 2026Thanks. The Data Team will review it. | |||||
| 11 | 76 contacts → | ||||
Strong evidence “At Spark® Therapeutics, we believe in a world where no life is limited by disease.” Source: RevenueBase Database · checked Sep 20, 2026Thanks. The Data Team will review it. | |||||
| 12 | 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 20, 2026Thanks. The Data Team will review it. | |||||
| 13 | 61 contacts → | ||||
Partial evidence “GondolaBio is a clinical-stage biopharmaceutical company focused on developing novel medicines for patients living with genetic diseases.” Source: RevenueBase Database · checked Sep 20, 2026Thanks. The Data Team will review it. | |||||
| 14 | 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 20, 2026Thanks. The Data Team will review it. | |||||
| 15 | 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 20, 2026Thanks. The Data Team will review it. | |||||
| 16 | 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 20, 2026Thanks. The Data Team will review it. | |||||
| 17 | 39 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 20, 2026Thanks. The Data Team will review it. | |||||
| 18 | 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 20, 2026Thanks. The Data Team will review it. | |||||
| 19 | 35 contacts → | ||||
Partial evidence “Rampart Bioscience is evolving a new class of medicines for patients with genetically driven diseases.” Source: RevenueBase Database · checked Sep 20, 2026Thanks. The Data Team will review it. | |||||
| 20 | 34 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 20, 2026Thanks. The Data Team will review it. | |||||
| 21 | 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 20, 2026Thanks. The Data Team will review it. | |||||
| 22 | 29 contacts → | ||||
Strong evidence “At Prevail Therapeutics, we are developing novel gene therapies that target the root genetic causes of Parkinson’s disease and other neurodegenerative disorders.” Source: RevenueBase Database · checked Sep 20, 2026Thanks. The Data Team will review it. | |||||
| 23 | 23 contacts → | ||||
Indirect evidence Source: RevenueBase Database · checked Sep 20, 2026Thanks. The Data Team will review it. | |||||
| 24 | 22 contacts → | ||||
Strong evidence “Spirovant Sciences is developing novel gene therapies for cystic fibrosis and other pulmonary diseases.” Source: RevenueBase Database · checked Sep 20, 2026Thanks. The Data Team will review it. | |||||
| 25 | 20 contacts → | ||||
Strong evidence “Our vision is to bring personalized gene therapy to the world.” Source: RevenueBase Database · checked Sep 20, 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: lysosomal storage 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 lysosomal storage disease gene therapy companies.
- Partial evidence
- Its own public description names part of lysosomal storage 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 lysosomal storage 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 74 lysosomal storage disease gene therapy companies based in the United States
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Lysosomal storage disease gene therapy companies based in the United States at a glance
As of Sep 20, 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 74 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 lysosomal storage 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 “lysosomal storage 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 lysosomal storage 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 lysosomal storage 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, 1,826 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 20, 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 lysosomal storage 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 lysosomal storage disease gene therapy companies are there based in the United States?
RevenueBase identified 74 lysosomal storage 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 lysosomal storage disease gene therapy companies?
Philadelphia leads with 7 headquartered companies, followed by Cambridge (4) 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 1,826 verified decision-maker contacts tracked across these 74 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 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 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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