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
Longeveron’s phase 2b stem cell flop spurs cost cutting, exploration of ‘all options’
A phase 2b study of Longeveron’s stem cell therapy in a pediatric heart disease has missed its primary endpoint, prompting the cash-strapped biotech to “review all options” for the business.
Longeveron’s shares crash as stem cell therapy flunks mid-stage cardio trial
With just over $10 million in cash available to fuel operations through the end of the year, Longeveron is undertaking a strategic review after the failure of a cardiovascular study.
Cellares and GenomeFrontier Therapeutics Partner to Advance Automated Manufacturing of GF-CART01
The partnership marks Cellares’ first collaboration with a cell therapy developer in Asia and will evaluate automated manufacturing of GenomeFrontier’s virus-free CAR T process for clinical manufactur
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.