Trial Updates

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
Optogenetics in advanced retinitis pigmentosa 1:52
Optogenetics in advanced retinitis pigmentosa
Sue Washer • 22 Oct 2021
Treating achromatopsia with AAV-based gene therapy 2:12
Treating achromatopsia with AAV-based gene therapy
Sue Washer • 22 Oct 2021
LUMEVOQ gene therapy for LHON 2:40
LUMEVOQ gene therapy for LHON
Magali Taiel • 6 Oct 2021
ProTrans: Wharton’s jelly-derived MSC therapy for type I diabetes 2:13
ProTrans: Wharton’s jelly-derived MSC therapy for type I diabetes
Lindsay Davies • 7 Jun 2021
Cord blood infusion in children with autism spectrum disorder 6:25
Cord blood infusion in children with autism spectrum disorder
Joanne Kurtzberg • 22 Jun 2021
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