Company lists · Data refreshed September 2026
Top 25 Lysosomal storage disease gene therapy companies based in the United States
RevenueBase identified 74 company matches for lysosomal storage disease gene therapy companies associated with 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 company matches for lysosomal storage disease gene therapy companies associated with the United States, found by RevenueBase SmartSearch using the meaning of business descriptions in its company graph at Exact. Ranked by professionals tracked, largest first. Rows show recorded descriptions where available, Exact search membership and the snapshot date. Data refreshed September 20, 2026.
Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase SmartSearch, 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 → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 2 | 456 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 3 | 236 contacts → | ||||
Exact match “4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 4 | 211 contacts → | ||||
Exact match “Our mission is to revolutionize medicine, with the ultimate goal of eliminating human suffering and enabling people to live without the burden of disease.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 5 | 208 contacts → | ||||
Exact match “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 –…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 6 | 160 contacts → | ||||
Exact match “Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 7 | 155 contacts → | ||||
Exact match “Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 8 | 121 contacts → | ||||
Exact match “Ocugen, Inc.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 9 | 109 contacts → | ||||
Exact match “Neurogene is accelerating development of new genetic medicines to people with devastating neurological diseases and their families.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 10 | 88 contacts → | ||||
Exact match “ReCode Therapeutics is a clinical-stage genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 11 | 76 contacts → | ||||
Exact match “At Spark® Therapeutics, we believe in a world where no life is limited by disease.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 12 | 62 contacts → | ||||
Exact match “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.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 13 | 61 contacts → | ||||
Exact match “GondolaBio is a clinical-stage biopharmaceutical company focused on developing novel medicines for patients living with genetic diseases.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 14 | 56 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 15 | 53 contacts → | ||||
Exact match “Jaguar Gene Therapy, LLC is dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 16 | 48 contacts → | ||||
Exact match “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…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 17 | 39 contacts → | ||||
Exact match “iECURE is a clinical-stage genome editing company dedicated to transforming the lives of patients with devastating neurometabolic disorders by harnessing new advancements in…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 18 | 35 contacts → | ||||
Exact match “Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 19 | 35 contacts → | ||||
Exact match “Rampart Bioscience is evolving a new class of medicines for patients with genetically driven diseases.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 20 | 34 contacts → | ||||
Exact match “Bamboo represents a community of researchers, clinicians, parents, regulatory scientists, and business professionals committed to providing hope to patients suffering from…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 21 | 33 contacts → | ||||
Exact match “American Gene Technologies® International Inc.(AGT™) is a private biotechnology company pursuing cures and treatments for infectious diseases, cancers, and monogenic disorders.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 22 | 29 contacts → | ||||
Exact match “At Prevail Therapeutics, we are developing novel gene therapies that target the root genetic causes of Parkinson’s disease and other neurodegenerative disorders.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 23 | 23 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 24 | 22 contacts → | ||||
Exact match “Spirovant Sciences is developing novel gene therapies for cystic fibrosis and other pulmonary diseases.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 25 | 20 contacts → | ||||
Exact match “Our vision is to bring personalized gene therapy to the world.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
RevenueBase SmartSearch
Describe a niche business in your own words. RevenueBase SmartSearch compares the meaning of your description with company business descriptions compiled in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit.
This list uses Exact for “lysosomal storage disease gene therapy companies”. Each Exact match badge shows membership in that saved search; it is not an individual similarity score or a service-quality rating. Recorded descriptions are shown where available.
Showing 25 of 74 lysosomal storage disease gene therapy companies based in the United States
See the full list, filter by size, metro and specialty, and access 1,826 tracked professional contacts with verified work emails. Direct dials are available where recorded.
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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
The recorded descriptions help you assess these company matches. Check a company’s website for its current services and locations.
- Source
- The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase SmartSearch, the natural-language company search available to every RevenueBase user.
- What qualifies
- Company records associated with the United States whose business descriptions match “lysosomal storage disease gene therapy companies” by meaning at the Exact setting. Descriptions are compiled in the RevenueBase company graph from public websites, profiles and company information where available; they are not guaranteed quotations from the company's website.
- RevenueBase SmartSearch
- Describe a niche company in your own words to find businesses by meaning. Exact, Similar, Related and Broad control the breadth of RevenueBase SmartSearch. This saved list uses Exact. Each row's Exact match badge shows inclusion in that search, not an individual score or a service-quality judgment.
- Public preview
- The public preview omits records without a verified work email. Where headquarters information is recorded, preview rows are also filtered to this page’s location. Dataset totals and geographic summaries describe the full matched records before these preview filters.
- Match quality
- RevenueBase's August 25, 2026 study assessed 4,198 description/company pairs across matching settings. The publisher reports 86% good or better at Exact. This evaluates the method across queries; it does not audit this individual list. Recorded company descriptions, where available, help you judge each match yourself. Read the full methodology: how the lists are built and what the study measures.
- 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 displayed company carries at least one verified work email. RevenueBase tracks 1,826 professional contacts with verified work emails across the full matched dataset. 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.
- Record context
- Company records and descriptions can change. Check the company's website for its current services and locations.
Frequently asked
What is RevenueBase SmartSearch?
RevenueBase SmartSearch finds niche companies by the meaning of their business descriptions in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit your description. This list uses Exact for “lysosomal storage disease gene therapy companies”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many lysosomal storage disease gene therapy companies are there based in the United States?
RevenueBase identified 74 company matches for lysosomal storage disease gene therapy companies associated with the United States as of September 2026, using the meaning of business descriptions at Exact. This is the recorded matched set in the RevenueBase company graph.
What is the largest company on this list?
Askbio INC tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.
Which city has the most lysosomal storage disease gene therapy companies?
Philadelphia leads with 7 company records, 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 limited free results with 500 credits to start. Upgrading provides access to the 1,826 professional contacts with verified work emails tracked across these 74 companies; direct dials are available where recorded.
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.
Ready to reach these companies? Verified emails and direct dials for all 74 lysosomal storage disease gene therapy companies based in the United States. Start with a free account, no credit card.
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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 SmartSearch, 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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Company intelligence · public preview
Top 25 lysosomal storage disease gene therapy companies based in the United States
Find companies in any niche.
Describe the businesses you want in your own words. RevenueBase SmartSearch matches company descriptions by meaning.
RevenueBase records 74 matching company records for lysosomal storage disease gene therapy companies based in the United States, with 4,289 tracked contacts and 1,826 verified work emails.
This public preview shows 25 selected records ordered by tracked contacts. RevenueBase SmartSearch finds company descriptions that match by meaning at Exact. Snapshot: September 20, 2026.
RevenueBase compiles business descriptions for niche-company discovery; each row shows a recorded excerpt where available. Adapt this search to find the companies and contacts you want.
Top 25 lysosomal storage disease gene therapy companies based in the United States
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 |  Askbio INCaskbio.comHQ: Research Triangle Park, NC, United StatesReported employees: 501-1,000 | Research Triangle Park, NC, United States | 501-1,000 | Exact match | 510 contacts → |
“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…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 |  Forge Biologicsforgebiologics.comHQ: Columbus, OH, United StatesReported employees: 201-500 | Columbus, OH, United States | 201-500 | Exact match | 456 contacts → |
“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…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 |  4d Molecular Therapeutics4dmoleculartherapeutics.comHQ: Emeryville, CA, United StatesReported employees: 51-200 | Emeryville, CA, United States | 51-200 | Exact match | 236 contacts → |
“4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 |  Kriya Therapeutics, INCkriyatx.comHQ: Morrisville, NC, United StatesReported employees: 51-200 | Morrisville, NC, United States | 51-200 | Exact match | 211 contacts → |
“Our mission is to revolutionize medicine, with the ultimate goal of eliminating human suffering and enabling people to live without the burden of disease.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 |  Voyager Therapeutics, INCvoyagertherapeutics.comHQ: Lexington, MA, United StatesReported employees: 51-200 | Lexington, MA, United States | 51-200 | Exact match | 208 contacts → |
“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 –…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 |  Solid Biosciencessolidbio.comHQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 160 contacts → |
“Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 |  Encoded Therapeutics INCencoded.comHQ: South San Francisco, CA, United StatesReported employees: 51-200 | South San Francisco, CA, United States | 51-200 | Exact match | 155 contacts → |
“Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 |  Ocugenocugen.comHQ: Malvern, PA, United StatesReported employees: 51-200 | Malvern, PA, United States | 51-200 | Exact match | 121 contacts → |
“Ocugen, Inc.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 |  Neurogene INCneurogene.comHQ: New York, NY, United StatesReported employees: 51-200 | New York, NY, United States | 51-200 | Exact match | 109 contacts → |
“Neurogene is accelerating development of new genetic medicines to people with devastating neurological diseases and their families.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 |  Recode Therapeuticsrecodetx.comHQ: Menlo Park, NC, United StatesReported employees: 11-50 | Menlo Park, NC, United States | 11-50 | Exact match | 88 contacts → |
“ReCode Therapeutics is a clinical-stage genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | Spark Therapeutics, INCsparktx.com HQ: Philadelphia, PA, United StatesReported employees: 501-1,000 | Philadelphia, PA, United States | 501-1,000 | Exact match | 76 contacts → |
“At Spark® Therapeutics, we believe in a world where no life is limited by disease.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 |  Affinia Therapeuticsaffiniatx.comHQ: Waltham, MA, United StatesReported employees: 51-200 | Waltham, MA, United States | 51-200 | Exact match | 62 contacts → |
“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.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | Gondolabiogondolabio.com HQ: Palo Alto, CA, United StatesReported employees: 11-50 | Palo Alto, CA, United States | 11-50 | Exact match | 61 contacts → |
“GondolaBio is a clinical-stage biopharmaceutical company focused on developing novel medicines for patients living with genetic diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 |  Gemma Biotherapeuticsgemmabiotx.comHQ: Philadelphia, PA, United StatesReported employees: 51-200 | Philadelphia, PA, United States | 51-200 | Exact match | 56 contacts → |
“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…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 |  Jaguar Gene Therapyjaguargenetherapy.comHQ: Lake Forest, IL, United StatesReported employees: 51-200 | Lake Forest, IL, United States | 51-200 | Exact match | 53 contacts → |
“Jaguar Gene Therapy, LLC is dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 |  AGTCagtc.comHQ: Alachua, FL, United StatesReported employees: 51-200 | Alachua, FL, United States | 51-200 | Exact match | 48 contacts → |
“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…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Iecure, INCiecure.com HQ: Philadelphia, PA, United StatesReported employees: 11-50 | Philadelphia, PA, United States | 11-50 | Exact match | 39 contacts → |
“iECURE is a clinical-stage genome editing company dedicated to transforming the lives of patients with devastating neurometabolic disorders by harnessing new advancements in…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 |  Novartis Gene Therapiesnovartis.comHQ: United StatesReported employees: 1,001-5,000 | United States | 1,001-5,000 | Exact match | 35 contacts → |
“Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Rampart Biosciencerampartbio.com HQ: Monrovia, CA, United StatesReported employees: Not recorded | Monrovia, CA, United States | Not recorded | Exact match | 35 contacts → |
“Rampart Bioscience is evolving a new class of medicines for patients with genetically driven diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Bamboo Therapeutics, Inc. A Wholly Owned Subsidiary Of Pfizer INCbambootherapeutics.com HQ: Chapel Hill, NC, United StatesReported employees: 11-50 | Chapel Hill, NC, United States | 11-50 | Exact match | 34 contacts → |
“Bamboo represents a community of researchers, clinicians, parents, regulatory scientists, and business professionals committed to providing hope to patients suffering from…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 |  American Gene Technologies International INCamericangene.comHQ: Rockville, MD, United StatesReported employees: 11-50 | Rockville, MD, United States | 11-50 | Exact match | 33 contacts → |
“American Gene Technologies® International Inc.(AGT™) is a private biotechnology company pursuing cures and treatments for infectious diseases, cancers, and monogenic disorders.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | Prevail Therapeuticsprevailtherapeutics.com HQ: NY, United StatesReported employees: 51-200 | NY, United States | 51-200 | Exact match | 29 contacts → |
“At Prevail Therapeutics, we are developing novel gene therapies that target the root genetic causes of Parkinson’s disease and other neurodegenerative disorders.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Genprex, INCgenprex.com HQ: Austin, TX, United StatesReported employees: 11-50 | Austin, TX, United States | 11-50 | Exact match | 23 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 24 | Spirovant Sciences, INCspirovant.com HQ: Philadelphia, PA, United StatesReported employees: 11-50 | Philadelphia, PA, United States | 11-50 | Exact match | 22 contacts → |
“Spirovant Sciences is developing novel gene therapies for cystic fibrosis and other pulmonary diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 |  Avrobioavrobio.comHQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 20 contacts → |
“Our vision is to bring personalized gene therapy to the world.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 74 company matches
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Lysosomal storage disease gene therapy companies: dataset insights
September 20, 2026These summaries describe recorded coverage in this saved dataset. This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Reported company sizes
74 records in the displayed employee bands.
0 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
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 company list was built
Describe a niche in your own words: companies installing commercial solar, or manufacturers of sustainable packaging. RevenueBase SmartSearch finds companies whose compiled business descriptions match by meaning. This list is one saved example to adapt.
Where the company descriptions come from
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
RevenueBase SmartSearch matching settings
- Exact
- Closest fits to the business described.
- Similar
- Similar businesses with a wider range of fits.
- Related
- Related businesses in and around the category.
- Broad
- A broader set of businesses connected to the description.
RevenueBase SmartSearch · Exact match identifies inclusion in this search's closest semantic matching setting. It is not a per-company confidence score, a literal keyword match, or a quality endorsement.
Selection, ordering and scope
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Recorded company descriptions support research; check company websites for current services. The selected preview is ordered by tracked contacts and is not a list of the best service providers.
Snapshot: September 20, 2026.
RevenueBase is a B2B data infrastructure company. Its September 3, 2026 published methodology describes a graph of 60 million companies and 398.9 million professional contacts; these platform figures differ from this page's totals. Published methodology ↗
Questions about this company list
What does Exact match mean for lysosomal storage disease gene therapy companies?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many lysosomal storage disease gene therapy companies did RevenueBase find based in the United States?
The saved snapshot records 74 matching company records, 4,289 tracked contacts and 1,826 verified emails. The public preview displays 25 selected records.
Where do the company descriptions come from?
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
How are the public companies ordered?
The selected public records are ordered by contacts tracked in RevenueBase, highest first. Contact coverage is a prospecting measure, not an assessment of the company's services or quality.
How can I get contact information?
Select a company's contact count to open its filtered contacts in RevenueBase. Free signup is required and includes 500 credits and limited free results. Verified emails, direct dials and mobile numbers depend on available coverage.
How current is this list?
Snapshot: September 20, 2026. Review each company's website for current services and locations. A recorded business description does not establish that every company offers every service named in the search.
What do headquarters and employee bands mean?
These are recorded company-level fields, rather than a map of every branch or a count of specialists. Missing headquarters or employee bands are labeled; older records may show a location separately from recorded headquarters.
Build your next target company list
Adapt this niche search and work with available contacts in RevenueBase.
Related lists
Cite this list
RevenueBase Data Team. Top 25 lysosomal storage disease gene therapy companies based in the United States. Snapshot: September 20, 2026. https://revenuebase.ai/companies/lysosomal-storage-disease-gene-therapy-companies/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
More company lists
- All company lists
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- Lists A-Z
- New lists
- AI companies based in the United States
- Cybersecurity companies based in the United States
- Manufacturing companies based in the United States
- Media companies based in the United States
- Oil and gas companies based in the United States
- Real estate companies based in the United States
Company intelligence · public preview
Top 25 lysosomal storage disease gene therapy companies based in the United States
RevenueBase records 74 matching company records for lysosomal storage disease gene therapy companies based in the United States, with 4,289 tracked contacts and 1,826 verified work emails.
This public preview shows 25 selected records ordered by tracked contacts. RevenueBase SmartSearch finds company descriptions that match by meaning at Exact. Snapshot: September 20, 2026.
RevenueBase compiles business descriptions for niche-company discovery; each row shows a recorded excerpt where available. Adapt this search to find the companies and contacts you want.
Top 25 lysosomal storage disease gene therapy companies based in the United States
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 | Askbio INCaskbio.com HQ: Research Triangle Park, NC, United StatesReported employees: 501-1,000 | Research Triangle Park, NC, United States | 501-1,000 | Exact match | 510 contacts → |
“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…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | Forge Biologicsforgebiologics.com HQ: Columbus, OH, United StatesReported employees: 201-500 | Columbus, OH, United States | 201-500 | Exact match | 456 contacts → |
“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…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 | 4d Molecular Therapeutics4dmoleculartherapeutics.com HQ: Emeryville, CA, United StatesReported employees: 51-200 | Emeryville, CA, United States | 51-200 | Exact match | 236 contacts → |
“4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | Kriya Therapeutics, INCkriyatx.com HQ: Morrisville, NC, United StatesReported employees: 51-200 | Morrisville, NC, United States | 51-200 | Exact match | 211 contacts → |
“Our mission is to revolutionize medicine, with the ultimate goal of eliminating human suffering and enabling people to live without the burden of disease.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | Voyager Therapeutics, INCvoyagertherapeutics.com HQ: Lexington, MA, United StatesReported employees: 51-200 | Lexington, MA, United States | 51-200 | Exact match | 208 contacts → |
“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 –…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Solid Biosciencessolidbio.com HQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 160 contacts → |
“Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | Encoded Therapeutics INCencoded.com HQ: South San Francisco, CA, United StatesReported employees: 51-200 | South San Francisco, CA, United States | 51-200 | Exact match | 155 contacts → |
“Encoded Therapeutics is creating one-time, disease-modifying gene therapies for pediatric central nervous system (CNS) disorders with its cell-selective targeting and regulation…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | Ocugenocugen.com HQ: Malvern, PA, United StatesReported employees: 51-200 | Malvern, PA, United States | 51-200 | Exact match | 121 contacts → |
“Ocugen, Inc.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | Neurogene INCneurogene.com HQ: New York, NY, United StatesReported employees: 51-200 | New York, NY, United States | 51-200 | Exact match | 109 contacts → |
“Neurogene is accelerating development of new genetic medicines to people with devastating neurological diseases and their families.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | Recode Therapeuticsrecodetx.com HQ: Menlo Park, NC, United StatesReported employees: 11-50 | Menlo Park, NC, United States | 11-50 | Exact match | 88 contacts → |
“ReCode Therapeutics is a clinical-stage genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | Spark Therapeutics, INCsparktx.com HQ: Philadelphia, PA, United StatesReported employees: 501-1,000 | Philadelphia, PA, United States | 501-1,000 | Exact match | 76 contacts → |
“At Spark® Therapeutics, we believe in a world where no life is limited by disease.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | Affinia Therapeuticsaffiniatx.com HQ: Waltham, MA, United StatesReported employees: 51-200 | Waltham, MA, United States | 51-200 | Exact match | 62 contacts → |
“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.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | Gondolabiogondolabio.com HQ: Palo Alto, CA, United StatesReported employees: 11-50 | Palo Alto, CA, United States | 11-50 | Exact match | 61 contacts → |
“GondolaBio is a clinical-stage biopharmaceutical company focused on developing novel medicines for patients living with genetic diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Gemma Biotherapeuticsgemmabiotx.com HQ: Philadelphia, PA, United StatesReported employees: 51-200 | Philadelphia, PA, United States | 51-200 | Exact match | 56 contacts → |
“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…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | Jaguar Gene Therapyjaguargenetherapy.com HQ: Lake Forest, IL, United StatesReported employees: 51-200 | Lake Forest, IL, United States | 51-200 | Exact match | 53 contacts → |
“Jaguar Gene Therapy, LLC is dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | AGTCagtc.com HQ: Alachua, FL, United StatesReported employees: 51-200 | Alachua, FL, United States | 51-200 | Exact match | 48 contacts → |
“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…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Iecure, INCiecure.com HQ: Philadelphia, PA, United StatesReported employees: 11-50 | Philadelphia, PA, United States | 11-50 | Exact match | 39 contacts → |
“iECURE is a clinical-stage genome editing company dedicated to transforming the lives of patients with devastating neurometabolic disorders by harnessing new advancements in…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Novartis Gene Therapiesnovartis.com HQ: United StatesReported employees: 1,001-5,000 | United States | 1,001-5,000 | Exact match | 35 contacts → |
“Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Rampart Biosciencerampartbio.com HQ: Monrovia, CA, United StatesReported employees: Not recorded | Monrovia, CA, United States | Not recorded | Exact match | 35 contacts → |
“Rampart Bioscience is evolving a new class of medicines for patients with genetically driven diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Bamboo Therapeutics, Inc. A Wholly Owned Subsidiary Of Pfizer INCbambootherapeutics.com HQ: Chapel Hill, NC, United StatesReported employees: 11-50 | Chapel Hill, NC, United States | 11-50 | Exact match | 34 contacts → |
“Bamboo represents a community of researchers, clinicians, parents, regulatory scientists, and business professionals committed to providing hope to patients suffering from…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | American Gene Technologies International INCamericangene.com HQ: Rockville, MD, United StatesReported employees: 11-50 | Rockville, MD, United States | 11-50 | Exact match | 33 contacts → |
“American Gene Technologies® International Inc.(AGT™) is a private biotechnology company pursuing cures and treatments for infectious diseases, cancers, and monogenic disorders.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | Prevail Therapeuticsprevailtherapeutics.com HQ: NY, United StatesReported employees: 51-200 | NY, United States | 51-200 | Exact match | 29 contacts → |
“At Prevail Therapeutics, we are developing novel gene therapies that target the root genetic causes of Parkinson’s disease and other neurodegenerative disorders.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Genprex, INCgenprex.com HQ: Austin, TX, United StatesReported employees: 11-50 | Austin, TX, United States | 11-50 | Exact match | 23 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 24 | Spirovant Sciences, INCspirovant.com HQ: Philadelphia, PA, United StatesReported employees: 11-50 | Philadelphia, PA, United States | 11-50 | Exact match | 22 contacts → |
“Spirovant Sciences is developing novel gene therapies for cystic fibrosis and other pulmonary diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Avrobioavrobio.com HQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 20 contacts → |
“Our vision is to bring personalized gene therapy to the world.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 74 company matches
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Lysosomal storage disease gene therapy companies: dataset insights
September 20, 2026These summaries describe recorded coverage in this saved dataset. This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Reported company sizes
74 records in the displayed employee bands.
0 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
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 company list was built
Describe a niche in your own words: companies installing commercial solar, or manufacturers of sustainable packaging. RevenueBase SmartSearch finds companies whose compiled business descriptions match by meaning. This list is one saved example to adapt.
Where the company descriptions come from
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
RevenueBase SmartSearch matching settings
- Exact
- Closest fits to the business described.
- Similar
- Similar businesses with a wider range of fits.
- Related
- Related businesses in and around the category.
- Broad
- A broader set of businesses connected to the description.
RevenueBase SmartSearch · Exact match identifies inclusion in this search's closest semantic matching setting. It is not a per-company confidence score, a literal keyword match, or a quality endorsement.
Selection, ordering and scope
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Recorded company descriptions support research; check company websites for current services. The selected preview is ordered by tracked contacts and is not a list of the best service providers.
Snapshot: September 20, 2026.
RevenueBase is a B2B data infrastructure company. Its September 3, 2026 published methodology describes a graph of 60 million companies and 398.9 million professional contacts; these platform figures differ from this page's totals. Published methodology ↗
Questions about this company list
What does Exact match mean for lysosomal storage disease gene therapy companies?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many lysosomal storage disease gene therapy companies did RevenueBase find based in the United States?
The saved snapshot records 74 matching company records, 4,289 tracked contacts and 1,826 verified emails. The public preview displays 25 selected records.
Where do the company descriptions come from?
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
How are the public companies ordered?
The selected public records are ordered by contacts tracked in RevenueBase, highest first. Contact coverage is a prospecting measure, not an assessment of the company's services or quality.
How can I get contact information?
Select a company's contact count to open its filtered contacts in RevenueBase. Free signup is required and includes 500 credits and limited free results. Verified emails, direct dials and mobile numbers depend on available coverage.
How current is this list?
Snapshot: September 20, 2026. Review each company's website for current services and locations. A recorded business description does not establish that every company offers every service named in the search.
What do headquarters and employee bands mean?
These are recorded company-level fields, rather than a map of every branch or a count of specialists. Missing headquarters or employee bands are labeled; older records may show a location separately from recorded headquarters.
Build your next target company list
Adapt this niche search and work with available contacts in RevenueBase.
Related lists
Cite this list
RevenueBase Data Team. Top 25 lysosomal storage disease gene therapy companies based in the United States. Snapshot: September 20, 2026. https://revenuebase.ai/companies/lysosomal-storage-disease-gene-therapy-companies/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
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