AVXS-201
/ Novartis
- LARVOL DELTA
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April 26, 2019
Rett syndrome gene therapy improves survival and ameliorates behavioral phenotypes in MeCP2 null
(AAN 2019)
- "AVXS-201 restores MECP2 expression and will be advanced for FIH testing."
April 19, 2019
Efficacy and Safety in Mice and Non-Human Primates of CSF Delivered AVXS-201 for the Treatment of Rett Syndrome
(ASGCT 2019)
- "Finally, toxicity studies in mice show AVXS-201 is safe and well tolerated. Together these data show that AVXS-201 is suitable for first in human studies."
April 19, 2019
Intrathecal Lumbar Administration of AAV9 in Nonhuman Primates is Safe and Effective in Targeting Cells of the Central Nervous System
(ASGCT 2019)
- "AAV9 is used by multiple academic and commercial groups as a research tool and a gene therapeutic including the AveXis product, AVXS-101. Intrathecal infusion of AAV9 vectors via lumbar puncture followed by Trendelenburg tilting of subjects was safe and well tolerated in juvenile cynomolgus macaques through 18 months post injection. Distribution of vector genomes, transgene mRNA expression, RNAi target knockdown and protein expression were shown in the brain, spinal cord, and peripheral tissues including the heart, liver and skeletal muscle."
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