H-002 (Orphina Biotechnology)

N/A

Report date: 2025/01/26 17:29:09

Base Information

Drug NameH-002 (Orphina Biotechnology)

Drug Alias Nameadeno-associated virus based gene therapy (Orphina Biotechnology)AAV based gene therapy (Orphina Biotechnology)GNE gene modulator (Orphina Biotechnology)

ClassificationBiologics

Mechanisms of ActionBifunctional UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase modulators

TargetsBifunctional UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase

Therapy Area/IndicationsHereditary inclusion body myopathy (HIBM)

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 CompaniesOrphina Biotechnology

China Registration ClassN/A

Develop Information

Active CompaniesOrphina Biotechnology

Inactive CompaniesN/A

Active IndicationsHereditary inclusion body myopathy (HIBM)

Inactive IndicationsN/A

Other Information

ATC CodeN/A