IDUA-transduced autologous stem cell therapy (Fondazione Telethon)

N/A

Report date: 2025/07/09 20:00:48

Base Information

Drug NameIDUA-transduced autologous stem cell therapy (Fondazione Telethon)
Drug Alias NameOTL-203
ClassificationCell and gene therapy > Cell therapy > Donor > AutologousCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Lentiviral vectorCell and gene therapy > Cell therapy > Stem cell therapy > Hematopoietic stem cells therapy
Mechanisms of ActionAlpha-L-iduronidase stimulants
TargetsAlpha-L-iduronidase
Therapy Area/IndicationsMucopolysaccharidosis IMucopolysaccharidoses
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited StatesNetherlandsItaly +[4] 
CombinationsNo
Global Highest StatusPhase 3 Clinical
Highest Status in ChinaN/A
Originator CompaniesFondazione Telethon
China Registration ClassN/A

Develop Information

Active CompaniesOrchard Therapeutics PlcFondazione Telethon
Inactive CompaniesN/A
Active IndicationsMucopolysaccharidosis IMucopolysaccharidoses
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A