DMPK CRISPR/Cas9 gene therapy (Vertex Pharmaceuticals/CRISPR Therapeutics)

N/A

Report date: 2025/06/11 11:24:05

Base Information

Drug NameDMPK CRISPR/Cas9 gene therapy (Vertex Pharmaceuticals/CRISPR Therapeutics)

Drug Alias NameN/A

ClassificationCell and gene therapy > Gene therapy > Gene editing > CRISPR > CRISPR-Cas9Cell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adenoviral vector

Mechanisms of ActionMyotonin-protein kinase inhibitors

TargetsMyotonin-protein kinase

Therapy Area/IndicationsMyotonic Dystrophy

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 CompaniesCrispr Therapeutics Ag

China Registration ClassN/A

Develop Information

Active CompaniesVertex Pharmaceuticals Gmbh

Inactive CompaniesN/A

Active IndicationsMyotonic Dystrophy

Inactive IndicationsN/A

Other Information

ATC CodeN/A