DMPK CRISPR/Cas9-gene therapy (Locana/University of California San Diego/University of Florida)

N/A

Report date: 2025/01/26 19:12:54

Base Information

Drug NameDMPK CRISPR/Cas9-gene therapy (Locana/University of California San Diego/University of Florida)
Drug Alias NameAAV9-RCas9 (Locana/University of California San Diego/University of Florida)AAV9-packaged PUF-E17 gene tharapy (Locanabio)RCas9-CTG (Locana/University of California San Diego/University of Florida)RNA-targeting gene therapy (Locana/University of California San Diego/University of Florida)CUG-targeting RCas9 (Locana/University of California San Diego/University of Florida) +[6] 
ClassificationBiologics
Mechanisms of ActionMyotonin-protein kinase modulators
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 CompaniesUniversity Of California San Diego
China Registration ClassN/A

Develop Information

Active CompaniesLocanabio IncUniversity Of FloridaUniversity Of California San Diego
Inactive CompaniesN/A
Active IndicationsMyotonic Dystrophy
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A