Q347X-mutation specific G6PC gene editing therapy (Beam Therapeutics)

N/A

Report date: 2025/12/12 17:32:42

Base Information

Drug NameQ347X-mutation specific G6PC gene editing therapy (Beam Therapeutics)

Drug Alias NameN/A

ClassificationCell and gene therapy > Gene therapy > mRNACell and gene therapy > Gene therapy > Gene editing > Base editing

Mechanisms of ActionGlucose-6-phosphatase modulators

TargetsGlucose-6-phosphatase

Therapy Area/IndicationsGlycogen Storage Disease Type I

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

China Registration ClassN/A

Inactive CompaniesN/A

Active IndicationsGlycogen Storage Disease Type I

Inactive IndicationsN/A

ATC CodeN/A