AAV Gene Therapy Platform
Precision vectors.
Rare disease targets.
Real impact.
Building next-generation AAV gene therapies for rare neurological diseases, from target discovery to clinical translation.
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Therapeutic Programs
AAV
Gene Therapy Platform
1
Published Review
Our Mission
Engineering precision for rare disease
Arcivus is developing next-generation AAV gene therapies targeting rare neurological disorders. Our platform combines computational biology with precision vector engineering to deliver therapeutic payloads where they're needed most.
Starting with DYT-SGCE myoclonus-dystonia, we're building a scalable approach to gene replacement therapy, one disease at a time, with the rigor and precision these patients deserve.
Research
Therapeutic Focus Areas
Our platform addresses rare diseases through three interconnected pillars of innovation.
Neurological Disorders
Targeting rare movement disorders through precision AAV-mediated gene replacement in the central nervous system.
AAV Vector Platform
Engineered adeno-associated viral vectors optimized for tropism, expression, and safety in target tissues.
Computational Biology
Data-driven target validation using protein structure analysis, variant interpretation, and pathway modeling.
Latest in Gene Therapy
Recent developments from across the industry
FDA adcomm shoots down Capricor's Duchenne cell therapy after meeting marked by statistical dispute
A panel of experts today voted against Capricor’s cell therapy for Duchenne muscular dystrophy, capping off an advisory committee meeting marked by statistical confusion.
UniQure welcomes possible adcomm for Huntington’s asset as regulatory ‘jitters’ spread through biopharma
UniQure is ready to put its Huntington’s disease gene therapy through an advisory committee meeting—even as the broader rare disease space has been put on edge by recent FDA scrutiny of assets by Repl
We're building precision AAV vectors for the diseases that need them most.
Jongmin Lee
Co-Founder & Research Lead
Partner with us
We're seeking collaborators in AAV vector engineering, preclinical models, and rare disease advocacy. Let's advance gene therapy together.