About
Organization, vision, and platform trajectory
Beyond One Patient
Arcivus Lab begins with a single patient and a single mutation. But the architecture being built — patient-specific genetic characterization, mutation-informed strategy selection, individualized construct design, and self-quantified outcome measurement — is not specific to DYT-SGCE. It is a framework.
Rare diseases are not rare in aggregate. There are over 7,000 known rare diseases affecting 300 million people globally. Most share a common structural pattern: a single gene, a known mutation, and no approved therapy — because each individual condition is too small for conventional drug development economics.
Arcivus Lab is building toward a future where each patient's molecular identity drives a custom therapeutic — not one drug for one disease, but one therapy for one person.
Platform Trajectory
n-of-1 Proof of Concept
Develop and validate AAV-SGCE gene replacement for a single patient. Build the computational and experimental pipeline.
SGCE Expansion
Generalize the pipeline to other DYT-SGCE patients with different mutation architectures.
Monogenic Neuro Platform
Extend to other monogenic movement disorders and rare neurological diseases with analogous therapeutic logic.
Full Platform
AI-driven mutation analysis, automated construct design, patient-matched delivery optimization.