ABCA4 gene therapy (Fondazione Telethon)

N/A

Report date: 2025/01/26 15:58:40

Base Information

Drug NameABCA4 gene therapy (Fondazione Telethon)

Drug Alias NameAAV5-ABCA4 gene therapy (Fondazione Telethon)AAV8-ABCA4 gene therapy (Fondazione Telethon)adenovirus gene therapy (Fondazione Telethon)

ClassificationBiologics

Mechanisms of ActionRetinal-specific phospholipid-transporting ATPase ABCA4 stimulants

TargetsRetinal-specific phospholipid-transporting ATPase ABCA4

Therapy Area/IndicationsStargardt Disease

Global Approved IndicationsN/A

Approved Indications in ChinaN/A

Original Approved Country/TerritoryN/A

Original Approved DateN/A

Country/TerritoryItaly

CombinationsNo

Global Highest StatusPreclinical

Highest Status in ChinaN/A

Originator CompaniesFondazione Telethon

China Registration ClassN/A

Develop Information

Active CompaniesFondazione Telethon

Inactive CompaniesN/A

Active IndicationsStargardt Disease

Inactive IndicationsN/A

Other Information

ATC CodeN/A