Organization (Nonprofit)
FONDAZIONE TELETHON
ClinicalTrials.gov lists FONDAZIONE TELETHON as lead sponsor of 12 trials. It has 1 active development program and 3 approved assets.
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: 3 registered with a 2026 start date. The dashed outline is the pace from the 3 registered with a start date through September, planned starts included, not adjusted; not a forecast. No forecast is made for this organization: it averages 1.0 trial starts a year over the five years to September, under the 5 a year the method needs.
Logo Fondazione Telethon, via Wikimedia Commons, CC BY-SA 4.0. Shown to identify the organization; it may be a registered trademark.
This page covers FONDAZIONE TELETHON: 12 lead-sponsored trials and 2 legal-entity registrations.
Disease areas (26)
Shown in this order: most trials first.
| Disease area | Trials |
|---|---|
| Primary Immunodeficiency Diseases | 4 |
| Lymphopenia | 2 |
| Severe Combined Immunodeficiency | 2 |
| Wiskott-Aldrich Syndrome | 2 |
| Anemia | 1 |
Showing 5 of 26 disease areas.
Trials by start year as sponsor or collaborator (13)
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) | 3 |
| 2024 | 2 |
| 2021 | 1 |
| 2020 | 1 |
| 2019 | 1 |
| 2018 | 4 |
| 2017 | 2 |
| 2015 | 2 |
| 2011 | 1 |
| 2010 | 1 |
Showing 10 of 13 years.
Recruitment duration, a model prediction: none of the 12 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
Active development programs (1)
| Drug | Disease area | Max phase | Trials |
|---|---|---|---|
| AAV2/8.TBG.hARSB | Mucopolysaccharidosis VI | Phase 1/2 | 1 |
Approved assets (3)
Shown in this order: max phase first.
| Drug | Trials |
|---|---|
| STRIMVELIS | 4 |
| Busulfan | 1 |
| Phenylbutyrate | 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 |
|---|---|
| 57% hit target (n=7) | 43% on time (n=7) |
Journals published in (53)
Shown in this order: most published in first.
| Journal | Publications |
|---|---|
| Blood | 10 |
| Molecular therapy : the journal of the American Society of Gene Therapy | 8 |
| The New England journal of medicine | 5 |
| Human gene therapy | 3 |
| Nature medicine | 3 |
Showing 5 of 53 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 |
|---|---|---|
| 36 | 11 | 2025-12-02 |
Organization relationships (8)
Shown in this order: most evidence first.
| Related organization | Relationship | Evidence |
|---|---|---|
| Orchard Therapeutics (United Kingdom) | Co sponsors trial with | 2 |
| Istituti di Ricovero e Cura a Carattere Scientifico | Co sponsors trial with | 1 |
| University of Messina | Co sponsors trial with | 1 |
| IRCCS Fondazione Stella Maris | Co sponsors trial with | 1 |
| Kolfarma s.r.l. - Italy | Co sponsors trial with | 1 |
Showing 5 of 8 relationships.
Latest dated events (58)
Shown in this order: newest first.
| Date | Event | Source |
|---|---|---|
| 2026-07-06 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2026-06-20 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2026-06-10 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2025-12-02 | Patent granted | Granted US patents (PatentsView / USPTO) |
| 2025-09-09 | Patent granted | Granted US patents (PatentsView / USPTO) |
Showing 5 of 58 events.
Biomarker capability evidence (35)
Shown in this order: tier desc, then gene symbol.
| Gene | Role | Evidence |
|---|---|---|
| ARSB | Trial sponsor | Named in trial inclusion criteria |
| CDA | Trial collaborator | Named in trial inclusion criteria |
| CDKL5 | Trial collaborator | Named in trial inclusion criteria |
| IDUA | Trial collaborator | Named in trial inclusion criteria |
| MYH9 | Trial collaborator | Named in trial inclusion criteria |
Showing 5 of 35 evidence rows.
Biomarkers this organization publishes on (14)
Shown in this order: most publications first.
Showing 5 of 14 biomarkers.
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, and clia clinical laboratory certificates.