AAV based gene therapy (Hospital for Sick Children)

N/A

Report date: 2025/03/14 20:00:48

Base Information

Drug NameAAV based gene therapy (Hospital for Sick Children)

Drug Alias NameN/A

ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vectorCell and gene therapy > Gene therapy > Gene editing > CRISPR > CRISPR-Cas9

Mechanisms of ActionUtrophin activators

TargetsUtrophin

Therapy Area/IndicationsMuscular Dystrophy, Duchenne

Global Approved IndicationsN/A

Approved Indications in ChinaN/A

Original Approved Country/TerritoryN/A

Original Approved DateN/A

Country/TerritoryCanada

CombinationsNo

Global Highest StatusPending (Preclinical)

Highest Status in ChinaN/A

Originator CompaniesThe Hospital For Sick Children

China Registration ClassN/A

Develop Information

Active CompaniesN/A

Inactive CompaniesThe Hospital For Sick Children

Active IndicationsN/A

Inactive IndicationsMuscular Dystrophy, Duchenne

Other Information

ATC CodeN/A