MELPIDA

N/A

Report date: 2026/05/15 20:01:45

Base Information

Drug NameMELPIDA
Drug Alias NameSelf-complimentary adeno-associated virus 9 gene therapy containing codon optimized human AP4M1 (CureSPG50)MELPIDA
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV9 vector
Mechanisms of ActionAP-4 complex subunit mu-1 modulators
TargetsAP-4 complex subunit mu-1
Therapy Area/IndicationsFailure to ThriveParaparesis, SpasticIntellectual Disability +[6] 
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryCanadaUnited States
CombinationsNo
Global Highest StatusPhase 2 Clinical
Highest Status in ChinaN/A
Originator CompaniesUniversity of Texas Southwestern Medical CenterCureSPG50
China Registration ClassN/A

Develop Information

Active CompaniesUniversity of Texas Southwestern Medical CenterElpida Therapeutics SPCThe Hospital For Sick Children
Inactive CompaniesN/A
Active IndicationsFailure to ThriveParaparesis, SpasticIntellectual Disability +[6] 
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A