Last update 20 Jun 2024

CG-001(CorrGene)

Overview

Basic Info

Drug Type
CRISPR/Cas, AAV based gene therapy
Synonyms
Autologous CD34+ cells edited with CRISPR/Cas9 and transduced with an adeno-associated virus vector serotype 6 containing the codon-optimized version of PKLR gene, CG001 (DanausGT), CG-001
+ [2]
Target
Mechanism
PKLR modulators(Pyruvate kinase isozymes R/L modulators)
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhasePhase 1
First Approval Date-
RegulationOrphan Drug (US), Orphan Drug (EU)

R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Pyruvate Kinase Deficiency of Red CellsPhase 1
CN
04 Aug 2023
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Clinical Result

Indication
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Regulation

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