SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
14,766Targets
453Trials
84Drugs
7Datasets
6,987Sources
64,683Claims
72,052Evidence
29,649Hypotheses
DRUGpreclinicalgene therapy

voyage aav gene therapy

Mechanism

AAV gene therapy delivering codon-optimized SMN1 cDNA. Uses Voyager TRACER platform (target-specific in vivo capsid evolution) for enhanced CNS/spinal-cord tropism vs first-gen AAV9. Novartis license 2022.

Related claims (0)

No claims matched “voyage aav gene therapy” in free-text search.

Off-Target Findings (0)

No Boltz-2 / Chai-1 off-target panel claims recorded for this drug.

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