Scientific Platform
PROTON: A new way to restore protein expression.
Precise RNA-Oriented Targeted Output Normalization — our proprietary approach to boost full-length, functional protein from healthy genes.
Upregulate
Antisense oligonucleotides that increase productive mRNA from the healthy gene copy.
Restore
Bring protein levels back to normal in cells missing one functional gene copy.
Target
Focused on haploinsufficiencies — diseases where a single working gene isn't enough.
Expand
Applicable across neurology, ophthalmology, and beyond.
Pipeline
Program
Indication
Stage
VTC-001
Dravet Syndrome
Phase 3
VTC-002
Autosomal Dominant Optic Atrophy
Phase 1/2
VTC-101
SYNGAP1-related disorder
Preclinical
VTC-201
Undisclosed CNS target
Discovery