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Highest phase reached
Phase 3
First clinical trial
Mar 1, 1992Last known activity
Dec 9, 2025
Timeline
1990s
- Jan 1, 1990
Fondazione Telethon— Founded
Drugs by trial phase (12 distinct, 10 trials)
Phase 3 (1 own, 0 via acquisition)
Phase 2 (11 own, 0 via acquisition)
- plerixafor
- doxycycline
- ibuprofen
- eltrombopag
- sodium phenylbutyrate
- aav2/8.tbg.harsb
- autologous cd34+ cells
- autologous hematopoietic stem cells genetically modified with globe lentiviral vector encoding for the human beta-globin gene
- sargramostim
- ada-transduced cd34+ cells
- frozen autologous cd34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector idua lv, encoding for the α-l-iduronidase cdna
Indications
Studied for
- beta-Thalassemia
- Blood Platelet Disorders
- Cystic Fibrosis
- Fibrosis
- Granulomatous Disease, Chronic
- Immunologic Deficiency Syndromes
- Leukodystrophy, Metachromatic
- Lysosomal Storage Diseases
- Mucopolysaccharidoses
- Mucopolysaccharidosis I
- Mucopolysaccharidosis VI
- Osteoporosis
- Pyruvate Dehydrogenase Complex Deficiency Disease
- Severe Combined Immunodeficiency
- Thalassemia
- Thrombocytopenia
Recent trials (13)
- NCT03734263Lead sponsorPhase 1/Phase 2Oct 1, 2018
- NCT03734263Reponsible party - principal investigatorPhase 1/Phase 2Oct 1, 2018
- NCT03488394CollaboratorPhase 1/Phase 2May 11, 2018
- NCT03173521Overall official - principal investigatorPhase 1/Phase 2Jul 1, 2017
- NCT03173521Reponsible party - principal investigatorPhase 1/Phase 2Jul 1, 2017
- NCT03173521Lead sponsorPhase 1/Phase 2Jul 1, 2017
- NCT03055247CollaboratorPhase 2Nov 1, 2015
- NCT02453477CollaboratorPhase 1/Phase 2May 1, 2015
- NCT04846335CollaboratorPhase 2Apr 28, 2011
- NCT01560182CollaboratorPhase 2May 1, 2010
- NCT01133860CollaboratorPhase 2Jan 1, 2009
- NCT01812551CollaboratorPhase 3Oct 1, 2002
- NCT00599781CollaboratorPhase 1/Phase 2Mar 1, 1992
Details
- Founded
- Milano, IT Jan 1, 1990
- Last known activity
- Dec 9, 2025