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Trial Updates
1:57
MOMENTUM: ARU-1801 in sickle cell disease
Will Chou
• 26 Jan 2022
2:23
DSG3-CAART: A novel CAAR T-cell therapy in pemphigus vulgaris
Samik Basu
• 26 Jan 2022
1:57
jCell: allogeneic cell therapy for retinitis pigmentosa
Gil Carrasquinho
• 2 Jul 2021
3:47
RP-L301 lentiviral gene therapy for PKD
Jose-Luis López-Lorenzo
• 15 Nov 2021
7:24
An update on the Phase I trial evaluating RP-L301 in PKD
Jose-Luis López-Lorenzo
• 15 Nov 2021
6:19
LYS-GM101 AAV gene therapy for GM1 gangliosidosis
Michael Hocquemiller
• 15 Nov 2021
3:42
GNT0003: AAV gene therapy for Crigler-Najjar syndrome
Lorenzo D'Antiga
• 18 Nov 2021
4:31
The safety of AVR-RD-01 gene therapy for Fabry disease
Mark Thomas
• 29 Oct 2021
2:38
LYS-SAF302 AAV gene therapy for MPS IIIA
Michael Hocquemiller
• 15 Nov 2021
1:16
Overcoming immunogenicity of gene therapies
Timothy Miller
• 18 Nov 2021
0:59
RESKUE: AAV-based gene therapy for Krabbe disease
Timothy Miller
• 18 Nov 2021
3:59
ATA3219, ATA3271 & ATA188: T-Cell immunotherapy products in development
Pascal Touchon
• 22 Oct 2021
2:03
AAV-based gene therapy for X-linked retinitis pigmentosa
Sue Washer
• 22 Oct 2021
1:52
Optogenetics in advanced retinitis pigmentosa
Sue Washer
• 22 Oct 2021
2:12
Treating achromatopsia with AAV-based gene therapy
Sue Washer
• 22 Oct 2021
2:40
LUMEVOQ gene therapy for LHON
Magali Taiel
• 6 Oct 2021
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