Trial Updates

CARBON trial: investigating CTX110 allogeneic CRISPR-Cas9–engineered CAR-Ts in R/R LBCL 1:23
CARBON trial: investigating CTX110 allogeneic CRISPR-Cas9–engineered CAR-Ts in R/R LBCL
Constantine Tam • 12 Dec 2022
The safety and efficacy of UCART123v1.2 in R/R AML: results from AMELI-01 2:52
The safety and efficacy of UCART123v1.2 in R/R AML: results from AMELI-01
David Sallman • 11 Dec 2022
Phase I study of CC-98633 in R/R multiple myeloma 2:45
Phase I study of CC-98633 in R/R multiple myeloma
Luciano Costa • 11 Dec 2022
MOMENTUM: ARU-1801 in sickle cell disease 1:57
MOMENTUM: ARU-1801 in sickle cell disease
Will Chou • 26 Jan 2022
DSG3-CAART: A novel CAAR T-cell therapy in pemphigus vulgaris 2:23
DSG3-CAART: A novel CAAR T-cell therapy in pemphigus vulgaris
Samik Basu • 26 Jan 2022
jCell: allogeneic cell therapy for retinitis pigmentosa 1:57
jCell: allogeneic cell therapy for retinitis pigmentosa
Gil Carrasquinho • 2 Jul 2021
RP-L301 lentiviral gene therapy for PKD 3:47
RP-L301 lentiviral gene therapy for PKD
Jose-Luis López-Lorenzo • 15 Nov 2021
An update on the Phase I trial evaluating RP-L301 in PKD 7:24
An update on the Phase I trial evaluating RP-L301 in PKD
Jose-Luis López-Lorenzo • 15 Nov 2021
LYS-GM101 AAV gene therapy for GM1 gangliosidosis 6:19
LYS-GM101 AAV gene therapy for GM1 gangliosidosis
Michael Hocquemiller • 15 Nov 2021
GNT0003: AAV gene therapy for Crigler-Najjar syndrome 3:42
GNT0003: AAV gene therapy for Crigler-Najjar syndrome
Lorenzo D'Antiga • 18 Nov 2021
The safety of AVR-RD-01 gene therapy for Fabry disease 4:31
The safety of AVR-RD-01 gene therapy for Fabry disease
Mark Thomas • 29 Oct 2021
LYS-SAF302 AAV gene therapy for MPS IIIA 2:38
LYS-SAF302 AAV gene therapy for MPS IIIA
Michael Hocquemiller • 15 Nov 2021
Overcoming immunogenicity of gene therapies 1:16
Overcoming immunogenicity of gene therapies
Timothy Miller • 18 Nov 2021
RESKUE: AAV-based gene therapy for Krabbe disease 0:59
RESKUE: AAV-based gene therapy for Krabbe disease
Timothy Miller • 18 Nov 2021
ATA3219, ATA3271 & ATA188: T-Cell immunotherapy products in development 3:59
ATA3219, ATA3271 & ATA188: T-Cell immunotherapy products in development
Pascal Touchon • 22 Oct 2021
AAV-based gene therapy for X-linked retinitis pigmentosa 2:03
AAV-based gene therapy for X-linked retinitis pigmentosa
Sue Washer • 22 Oct 2021
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