Autologous genetically modified hematopoietic stem cell therapy (Rocket Pharmaceuticals)

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Report date: 2026/04/17 20:02:02

Base Information

Drug NameAutologous genetically modified hematopoietic stem cell therapy (Rocket Pharmaceuticals)
Drug Alias NameAutologous genetically modified CD34+ cell therapy自体转基因CD34 +造血干细胞疗法-Rocket PharmaceuticalsCG001（科金生物）Autologous genetically modified CD34+ cellsPKLR gene therapy +[10] 
ClassificationCell and gene therapy > Cell therapy > Donor > AutologousCell and gene therapy > Cell therapy > Stem cell therapy > Hematopoietic stem cells therapy
Mechanisms of ActionPyruvate kinase gene transference
TargetsPyruvate kinase
Therapy Area/IndicationsPyruvate kinase deficiency
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited StatesSpain
CombinationsNo
Global Highest StatusPending (Phase 2 Clinical)
Highest Status in ChinaN/A
Originator CompaniesCiematCibererIis-Fjd
China Registration ClassN/A

Develop Information

Active CompaniesN/A
Inactive CompaniesRocket Pharmaceuticals Inc
Active IndicationsN/A
Inactive IndicationsPyruvate kinase deficiency

Other Information

ATC CodeN/A
Related LinksN/A