Atidarsagene autotemcel

阿替达基奥干赛

Report date: 2024/03/22 11:34:47

Base Information

Drug NameAtidarsagene autotemcel
Drug Alias NameARSA gene therapy (GlaxoSmithKline/Fondazione Telethon/Ospedale San Raffaele)Arylsulfatase A gene therapy (GlaxoSmithKline/Fondazione Telethon/Ospedale San Raffaele)Ex vivo stem cell gene therapy (GlaxoSmithKline/Fondazione Telethon/Ospedale San Raffaele)ARSA transduced autologous CD34+ stem cells (GlaxoSmithKline/Fondazione Telethon/Ospedale San Raffaele)Libmeldy +[9] 
ClassificationCell and gene therapy > Cell therapy > Stem cell therapy > Hematopoietic stem cells therapy
Mechanisms of ActionARSA gene transference
TargetsCerebroside-sulfatase
Therapy Area/IndicationsLysosomal Storage DiseasesLeukodystrophy, Metachromatic
Global Approved IndicationsLeukodystrophy, Metachromatic
Approved Indications in ChinaN/A
Original Approved Country/TerritoryEU
Original Approved Date2020/12/17
Country/TerritoryUnited StatesItalyEU
CombinationsNo
Global Highest StatusApproved
Highest Status in ChinaN/A
Originator CompaniesGlaxosmithkline PlcOspedale San RaffaeleFondazione Telethon
China Registration ClassN/A

Develop Information

Active CompaniesOrchard Therapeutics PlcOspedale San RaffaeleOrchard Therapeutics (Netherlands) Bv
Inactive CompaniesN/A
Active IndicationsLysosomal Storage DiseasesLeukodystrophy, Metachromatic
Inactive IndicationsN/A

Other Information

ATC CodeA16AB21N07
Related LinksWikipediaDrugBank