Organization (Industry)
Genethon (France)
ClinicalTrials.gov lists Genethon (France) as lead sponsor of 15 trials. It has 2 active development programs and 1 approved asset.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials started per year with this organization as sponsor or collaborator, 1990 onward. The series also counts trials where the organization is a collaborator; the lead-sponsor trial count does not.
2026 is partial: 1 registered with a 2026 start date. The dashed outline is the pace from the 1 registered with a start date through September, planned starts included, not adjusted; not a forecast. No forecast is made for this organization: it averages 0.6 trial starts a year over the five years to September, under the 5 a year the method needs.
This page covers Genethon (France): 15 lead-sponsored trials and 1 legal-entity registration.
Disease areas (17)
Shown in this order: most trials first.
| Disease area | Trials |
|---|---|
| Crigler-Najjar Syndrome | 3 |
| Granulomatous Disease, Chronic | 3 |
| Lymphopenia | 3 |
| Muscular Dystrophy, Duchenne | 3 |
| Primary Immunodeficiency Diseases | 3 |
Showing 5 of 17 disease areas.
Trials by start year as sponsor or collaborator (11)
Shown in this order: most recent first. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Show the table of trials by start year as sponsor or collaborator
| Year | Trials started |
|---|---|
| 2026 (partial year) | 1 |
| 2024 | 1 |
| 2021 | 2 |
| 2020 | 1 |
| 2019 | 1 |
| 2018 | 1 |
| 2015 | 1 |
| 2014 | 3 |
| 2013 | 2 |
| 2011 | 4 |
Showing 10 of 11 years.
Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
Active development programs (2)
Shown in this order: max phase, then active trials.
| Drug | Disease area | Max phase | Trials |
|---|---|---|---|
| GNT0003 | Crigler-Najjar Syndrome | Phase 2 | 2 |
| Imlifidase | Crigler-Najjar Syndrome | Phase 2 | 1 |
Approved assets (1)
| Drug | Trials |
|---|---|
| Imlifidase | 1 |
Sponsor delivery record (1)
Enrollment hit target: the share of this sponsor's lead-sponsored trials whose actual enrollment reached the planned enrollment. On time: the share whose actual primary completion date was no later than the planned date. Each share counts only trials where we observed an estimated figure before the actual one, and n is that number of trials. Below 5 trials the count is shown as a fraction, not a percentage.
| Enrollment | Timeline |
|---|---|
| 1 of 4 hit target (n=4) | 2 of 4 on time (n=4) |
Journals published in (9)
Shown in this order: most published in first.
| Journal | Publications |
|---|---|
| Neuromuscular disorders : NMD | 2 |
| Blood | 1 |
| Human gene therapy. Clinical development | 1 |
| Molecular therapy : the journal of the American Society of Gene Therapy | 1 |
| Nature medicine | 1 |
Showing 5 of 9 journals.
US granted patents (1)
Patents are not classified by disease area in the source, so this is the organization's overall portfolio, not a split by disease.
| US patents granted | Granted in the last 5 years | Most recent grant |
|---|---|---|
| 55 | 23 | 2025-11-11 |
Organization relationships (7)
Shown in this order: most evidence first.
| Related organization | Relationship | Evidence |
|---|---|---|
| Assistance Publique – Hôpitaux de Paris | Co sponsors trial with | 1 |
| University of California System | Co sponsors trial with | 1 |
| Institute of Child Health | Co sponsors trial with | 1 |
| Great Ormond Street Hospital for Children NHS Foundation Trust | Co sponsors trial with | 1 |
| Valerion Therapeutics (United States) | Co sponsors trial with | 1 |
Showing 5 of 7 relationships.
Latest dated events (73)
Shown in this order: newest first.
| Date | Event | Source |
|---|---|---|
| 2026-07-31 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2025-11-11 | Patent granted | Granted US patents (PatentsView / USPTO) |
| 2025-07-08 | Patent granted | Granted US patents (PatentsView / USPTO) |
| 2025-05-20 | Patent granted | Granted US patents (PatentsView / USPTO) |
| 2024-12-24 | Patent granted | Granted US patents (PatentsView / USPTO) |
Showing 5 of 73 events.
Biomarker capability evidence (51)
Shown in this order: tier desc, then gene symbol.
| Gene | Role | Evidence |
|---|---|---|
| DMD | Trial sponsor | Named in trial inclusion criteria |
| DMD | Trial sponsor | Named in trial inclusion criteria |
| DMD | Trial sponsor | Named in trial inclusion criteria |
| DMD | Trial sponsor | Primary endpoint in a Phase 3 trial |
| FKRP | Trial sponsor | Named in trial inclusion criteria |
Showing 5 of 51 evidence rows.
On Scientist.com
Genethon (France) is listed as a research supplier in the Scientist.com network.
Service areas
Genethon focuses their efforts in developing new vector and plasmid designs and contructing them in the application of gene therapy studies. Their expertise includes bioprocess development, bioanalytical development, and sample storage.
- Website
- www.genethon.fr
Not on this page
Checked, with nothing recorded for this organization: companies grouped under this page, related companies not grouped here, sec-reported annual financials, financing and grant events, fda warning letters, fda untitled letters, fda inspection record, fda recalls, fda import refusals, manufacturing and distribution sites, climate targets, as filed, workforce and board diversity, as filed, clia clinical laboratory certificates, and biomarkers this organization publishes on.
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