SNG-101

N/A

Report date: 2026/05/12 20:00:17

Base Information

Drug NameSNG-101
Drug Alias NameABX-AAAV-NAGLUSanfilippo syndrome type-B gene therapyrAAV9.CMV.hNAGLUSC AAV9-NAGLU +[10] 
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV9 vector
Mechanisms of ActionGene transferenceHeparan sulfate sulfatase replacements
TargetsHeparan sulfate
Therapy Area/IndicationsMucopolysaccharidosis III
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited StatesGermanyFrance +[4] 
CombinationsNo
Global Highest StatusDiscontinued (Phase 2 Clinical)
Highest Status in ChinaN/A
Originator CompaniesNationwide Children'S Hospital
China Registration ClassN/A

Develop Information

Active CompaniesN/A
Inactive CompaniesSangrail Biologics IncAbeona Therapeutics Inc
Active IndicationsN/A
Inactive IndicationsMucopolysaccharidosis III

Other Information

ATC CodeN/A
Related LinksN/A