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
Legend scientific founder returns to ASCO with new ambition for high-yield, non-gene-editing CAR-T platform
Nine years after wowing the audience at the American Society of Clinical Oncology annual meeting with a CAR-T candidate that would become Carvykti—now the world’s most successful cell therapy—Legend B
AstraZeneca, after phase 3 miss, posts subgroup data behind pitch for rare disease approval
AstraZeneca has shared data it believes can rescue anselamimab from a phase 3 failure, linking the rare disease drug candidate to a 62% improvement in survival in a prespecified subgroup.
FDA extends Beren’s rare disease review after info request, further stretching saga
The FDA has delayed its approval decision on Beren Therapeutics’ rare disease drug candidate, extending the asset’s long path toward the U.S. market.
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.