Capsid Engineering
Proprietary AAV variants with enhanced CNS tropism and immune evasion. Superior transduction across BBB and intrathecal routes.
Learn More →Genosyne engineers precision AAV gene therapy vectors for durable correction of devastating neurological conditions.
Our Technology
Three integrated capabilities purpose-built for the central nervous system — from vector design to precise in vivo delivery.
Proprietary AAV variants with enhanced CNS tropism and immune evasion. Superior transduction across BBB and intrathecal routes.
Learn More →Cell-type-specific promoters and regulatory elements ensure gene expression exclusively in target neuronal populations.
Learn More →CRISPR base editing, prime editing, and antisense oligonucleotide approaches for gain- and loss-of-function targets.
Learn More →Pipeline
Genosyne targets monogenic neurological conditions with high unmet need and well-validated genetic drivers.
Our lead ALS program targets SOD1 gain-of-toxic-function mutations, the most genetically validated driver of familial ALS. Using intrathecal delivery of an engineered AAV9 variant with SOD1-targeting silencing constructs, we aim to achieve durable knockdown across motor cortex and spinal motor neurons in a single administration.
Targeting the polyglutamine expansion in mutant huntingtin (mHTT) with a combined AAV + ASO platform designed for allele-selective silencing. Our approach preserves wild-type HTT function while achieving deep striatal penetration through intrastriatal and intraventricular delivery routes.
KCNQ2 loss-of-function variants cause severe neonatal-onset epileptic encephalopathy refractory to current antiepileptic drugs. Genosyne is developing a neuronal-specific gene replacement strategy using a compact capsid suited for cortical and hippocampal transduction in neonatal patients.
Leadership
CEO & Co-Founder
Michael Torres trained in computational neuroscience at Stanford and previously led viral vector engineering at a major gene therapy company. He holds 12 issued patents in AAV capsid design and has co-authored 15+ peer-reviewed publications in Nature Methods, Nature Neuroscience, and Cell. He founded Genosyne to translate next-generation capsid science into durable medicines for patients with no other options.
Get In Touch
We welcome conversations with patient advocacy groups, potential partners, and qualified investors.
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