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.
3+
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 grants uniQure’s Huntington’s gene therapy priority review, teeing up ‘defining milestone’
The FDA has accepted uniQure’s priority review and application for its gene therapy treatment for Huntington’s disease.
Gilead reveals partial clinical hold on 2 CAR-T cell therapy trials
Gilead Sciences announced on its third-quarter earnings call Oct. 29 that the company has placed a partial clinical hold on two studies for the CAR-T cell therapy KITE-753 in diffuse large B-cell lymp
Cell therapy investment grows as safety events rankle Big Pharma's CAR T autoimmune push
Sail Biomedicine’s $925 million deal with Johnson & Johnson helped boost the cell and gene therapy sector over biologics in terms of venture capital fundraising in the third quarter.
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.