Development
Pipeline
From target discovery to clinical translation
ARC-001
DYT-SGCE (Myoclonus-Dystonia)
AAV Gene Replacement
Discovery
Preclinical
Phase 1
Phase 2
Phase 3
Approval
ARC-002
Neurological - TBD
AAV Gene Therapy
Discovery
Preclinical
Phase 1
Phase 2
Phase 3
Approval
ARC-003
Rare Movement Disorder - TBD
AAV Gene Therapy
Discovery
Preclinical
Phase 1
Phase 2
Phase 3
Approval
| Program | Discovery | Preclinical | Phase 1 | Phase 2 | Phase 3 | Approval |
|---|---|---|---|---|---|---|
ARC-001 DYT-SGCE (Myoclonus-Dystonia) AAV Gene Replacement | ||||||
ARC-002 Neurological - TBD AAV Gene Therapy | ||||||
ARC-003 Rare Movement Disorder - TBD AAV Gene Therapy |
Our Lead Program
ARC-001 targets DYT-SGCE myoclonus-dystonia through AAV-mediated gene replacement of epsilon-sarcoglycan. Currently in preclinical development with computational target validation and vector optimization underway.