Company lists · Data refreshed September 2026
Top 25 Lysosomal storage disease gene therapy companies worldwide
RevenueBase identified 151 company matches for lysosomal storage disease gene therapy companies worldwide as of September 2026 and tracks 6,879 professional contacts across them, including 3,018 with verified work emails. The United States is the largest market and Philadelphia the largest hub.
This list covers company matches for lysosomal storage disease gene therapy companies worldwide, 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. Country and city lists for lysosomal storage disease gene therapy companies follow the table.
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 worldwide
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 | 398 contacts → | ||||
Exact match “MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicine company with a broad pipeline of late-stage clinical programs supported by end-to-end…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 4 | 349 contacts → | ||||
Exact match “uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 5 | 293 contacts → | ||||
Exact match “Généthon est un laboratoire à but non lucratif, créé et soutenu par l’AFM-Téléthon depuis 1990.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 6 | 270 contacts → | ||||
Exact match “For Sangamo Therapeutics, science is a means to develop new medicines with the potential to transform the lives of patients living with serious genetic diseases.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 7 | 257 contacts → | ||||
Exact match “TIGEM is a leading research institute dedicated to uncovering the molecular mechanisms of rare genetic diseases and translating these discoveries into innovative therapies, with a…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 8 | 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 | |||||
| 9 | 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 | |||||
| 10 | 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 | |||||
| 11 | 173 contacts → | ||||
Exact match “Founded in 1996, the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between Fondazione Telethon, a major Italian charity funding research on rare…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 12 | 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 | |||||
| 13 | 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 | |||||
| 14 | 121 contacts → | ||||
Exact match “Ocugen, Inc.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 15 | 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 | |||||
| 16 | 98 contacts → | ||||
Exact match “Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 17 | 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 | |||||
| 18 | 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 | |||||
| 19 | 75 contacts → | ||||
Exact match “Alpha Cognition is a clinical stage, biopharmaceutical company dedicated to developing novel treatments for under-served neurodegenerative diseases such as Alzheimer’s Dementia…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 20 | 63 contacts → | ||||
Exact match “At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 21 | 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 | |||||
| 22 | 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 | |||||
| 23 | 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 | |||||
| 24 | 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 | |||||
| 25 | 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 | |||||
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 151 lysosomal storage disease gene therapy companies worldwide
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Lysosomal storage disease gene therapy companies worldwide at a glance
As of Sep 20, 2026Companies by employee count
Email recency is reported on each country list linked below.
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.
- Coverage
- Every country. Country and city lists for lysosomal storage disease gene therapy companies are linked below.
- What qualifies
- Company records worldwide 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. 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 3,018 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.
Lysosomal storage disease gene therapy companies by location
1 lists · largest firstEvery Lysosomal storage disease gene therapy companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
Frequently asked
What is RevenueBase 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 worldwide?
RevenueBase identified 151 company matches for lysosomal storage disease gene therapy companies worldwide 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 country has the most lysosomal storage disease gene therapy companies?
The United States leads with 74 companies. Every lysosomal storage disease gene therapy companies list by country and city is linked on this page.
Which city has the most lysosomal storage disease gene therapy companies?
Philadelphia leads with 7 company records, followed by Amsterdam (4) and Cambridge (4).
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 3,018 professional contacts with verified work emails tracked across these 151 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 151 lysosomal storage disease gene therapy companies worldwide. 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.
More company lists
Company intelligence · public preview
Top 25 lysosomal storage disease gene therapy companies worldwide
Find companies in any niche.
Describe the businesses you want in your own words. RevenueBase SmartSearch matches company descriptions by meaning.
RevenueBase records 151 matching company records for lysosomal storage disease gene therapy companies worldwide, with 6,879 tracked contacts and 3,018 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 worldwide
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 |  Meiragtxmeiragtx.comHQ: London/new York, United KingdomReported employees: 51-200 | London/new York, United Kingdom | 51-200 | Exact match | 398 contacts → |
“MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicine company with a broad pipeline of late-stage clinical programs supported by end-to-end…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 |  Uniqureuniqure.comHQ: Amsterdam, United StatesReported employees: 51-200 | Amsterdam, United States | 51-200 | Exact match | 349 contacts → |
“uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 |  Genethongenethon.frHQ: Évry, FranceReported employees: 201-500 | Évry, France | 201-500 | Exact match | 293 contacts → |
“Généthon est un laboratoire à but non lucratif, créé et soutenu par l’AFM-Téléthon depuis 1990.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 |  Sangamo Therapeutics, INCsangamo.comHQ: Brisbane, CA, AustraliaReported employees: 201-500 | Brisbane, CA, Australia | 201-500 | Exact match | 270 contacts → |
“For Sangamo Therapeutics, science is a means to develop new medicines with the potential to transform the lives of patients living with serious genetic diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | HQ: Pozzuoli, ItalyReported employees: 201-500 | Pozzuoli, Italy | 201-500 | Exact match | 257 contacts → |
“TIGEM is a leading research institute dedicated to uncovering the molecular mechanisms of rare genetic diseases and translating these discoveries into innovative therapies, with a…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 |  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. | |||||
| 9 |  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. | |||||
| 10 |  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. | |||||
| 11 | SAN Raffaele Telethon Institute For Gene Therapy Sr-Tigetresearch.hsr.it HQ: Milan, ItalyReported employees: 201-500 | Milan, Italy | 201-500 | Exact match | 173 contacts → |
“Founded in 1996, the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between Fondazione Telethon, a major Italian charity funding research on rare…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 |  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. | |||||
| 13 |  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. | |||||
| 14 |  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. | |||||
| 15 |  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. | |||||
| 16 |  Spur Therapeuticsspurtherapeutics.comHQ: Stevenage, United KingdomReported employees: 11-50 | Stevenage, United Kingdom | 11-50 | Exact match | 98 contacts → |
“Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 |  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. | |||||
| 18 | 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. | |||||
| 19 |  Alpha Cognition, INCalphacognition.comHQ: Vancouver, CanadaReported employees: 11-50 | Vancouver, Canada | 11-50 | Exact match | 75 contacts → |
“Alpha Cognition is a clinical stage, biopharmaceutical company dedicated to developing novel treatments for under-served neurodegenerative diseases such as Alzheimer’s Dementia…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 |  Aviadobioaviadobio.comHQ: London, United KingdomReported employees: 11-50 | London, United Kingdom | 11-50 | Exact match | 63 contacts → |
“At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 |  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. | |||||
| 22 | 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. | |||||
| 23 |  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. | |||||
| 24 |  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. | |||||
| 25 |  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. | |||||
Showing 25 of 151 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
150 records in the displayed employee bands.
1 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 across the whole country list rather than for Worldwide, so it is reported there. Company counts and contact totals on this page are exact for Worldwide, over 151 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 ↗
Lysosomal storage disease gene therapy companies by location
1 lists · largest firstEvery Lysosomal storage disease gene therapy companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
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 worldwide?
The saved snapshot records 151 matching company records, 6,879 tracked contacts and 3,018 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.
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Cite this list
RevenueBase Data Team. Top 25 lysosomal storage disease gene therapy companies worldwide. Snapshot: September 20, 2026. https://revenuebase.ai/companies/browse/lysosomal-storage-disease-gene-therapy-companies.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
More company lists
Company intelligence · public preview
Top 25 lysosomal storage disease gene therapy companies worldwide
RevenueBase records 151 matching company records for lysosomal storage disease gene therapy companies worldwide, with 6,879 tracked contacts and 3,018 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 worldwide
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 | Meiragtxmeiragtx.com HQ: London/new York, United KingdomReported employees: 51-200 | London/new York, United Kingdom | 51-200 | Exact match | 398 contacts → |
“MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicine company with a broad pipeline of late-stage clinical programs supported by end-to-end…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | Uniqureuniqure.com HQ: Amsterdam, United StatesReported employees: 51-200 | Amsterdam, United States | 51-200 | Exact match | 349 contacts → |
“uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | Genethongenethon.fr HQ: Évry, FranceReported employees: 201-500 | Évry, France | 201-500 | Exact match | 293 contacts → |
“Généthon est un laboratoire à but non lucratif, créé et soutenu par l’AFM-Téléthon depuis 1990.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Sangamo Therapeutics, INCsangamo.com HQ: Brisbane, CA, AustraliaReported employees: 201-500 | Brisbane, CA, Australia | 201-500 | Exact match | 270 contacts → |
“For Sangamo Therapeutics, science is a means to develop new medicines with the potential to transform the lives of patients living with serious genetic diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | HQ: Pozzuoli, ItalyReported employees: 201-500 | Pozzuoli, Italy | 201-500 | Exact match | 257 contacts → |
“TIGEM is a leading research institute dedicated to uncovering the molecular mechanisms of rare genetic diseases and translating these discoveries into innovative therapies, with a…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | 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. | |||||
| 9 | 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. | |||||
| 10 | 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. | |||||
| 11 | SAN Raffaele Telethon Institute For Gene Therapy Sr-Tigetresearch.hsr.it HQ: Milan, ItalyReported employees: 201-500 | Milan, Italy | 201-500 | Exact match | 173 contacts → |
“Founded in 1996, the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between Fondazione Telethon, a major Italian charity funding research on rare…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | 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. | |||||
| 13 | 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. | |||||
| 14 | 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. | |||||
| 15 | 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. | |||||
| 16 | Spur Therapeuticsspurtherapeutics.com HQ: Stevenage, United KingdomReported employees: 11-50 | Stevenage, United Kingdom | 11-50 | Exact match | 98 contacts → |
“Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | 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. | |||||
| 18 | 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. | |||||
| 19 | Alpha Cognition, INCalphacognition.com HQ: Vancouver, CanadaReported employees: 11-50 | Vancouver, Canada | 11-50 | Exact match | 75 contacts → |
“Alpha Cognition is a clinical stage, biopharmaceutical company dedicated to developing novel treatments for under-served neurodegenerative diseases such as Alzheimer’s Dementia…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Aviadobioaviadobio.com HQ: London, United KingdomReported employees: 11-50 | London, United Kingdom | 11-50 | Exact match | 63 contacts → |
“At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | 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. | |||||
| 22 | 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. | |||||
| 23 | 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. | |||||
| 24 | 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. | |||||
| 25 | 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. | |||||
Showing 25 of 151 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
150 records in the displayed employee bands.
1 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 across the whole country list rather than for Worldwide, so it is reported there. Company counts and contact totals on this page are exact for Worldwide, over 151 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 ↗
Lysosomal storage disease gene therapy companies by location
1 lists · largest firstEvery Lysosomal storage disease gene therapy companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
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 worldwide?
The saved snapshot records 151 matching company records, 6,879 tracked contacts and 3,018 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.
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Cite this list
RevenueBase Data Team. Top 25 lysosomal storage disease gene therapy companies worldwide. Snapshot: September 20, 2026. https://revenuebase.ai/companies/browse/lysosomal-storage-disease-gene-therapy-companies.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗