Sanfilippo syndrome gene therapy (Lacerta/Sarepta)

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Report date: 2025/01/26 19:14:56

Base Information

Drug NameSanfilippo syndrome gene therapy (Lacerta/Sarepta)
Drug Alias NameAAV gene therapy (Lacerta/Sarepta)AAV gene therapy (Sarepta)CNS-1 Sarepta/Lactera
ClassificationBiologics
Mechanisms of ActionUpdating
TargetsUpdating
Therapy Area/IndicationsMucopolysaccharidosis III
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited States
CombinationsNo
Global Highest StatusPreclinical
Highest Status in ChinaN/A
Originator CompaniesLacerta Therapeutics Inc
China Registration ClassN/A

Develop Information

Active CompaniesLacerta Therapeutics IncSarepta Therapeutics Inc
Inactive CompaniesN/A
Active IndicationsMucopolysaccharidosis III
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A