Mechanism of Action

Programmable
biology, observed

Helix Bio designs sequence-specific oligonucleotide and gene-therapy modalities — engineered to recognize, replicate intent, and express therapeutic proteins with single-base precision.

38 Targets
04 Modalities
11 Clinical programs
Binding 01

Target
recognition

A guide strand scans the genome and docks to its complementary motif. Watson–Crick pairing is read base-by-base, conferring sequence-specific affinity that discriminates a single mismatch from a perfect site.

0.4 nM
Affinity
>99.6%
Specificity
02 Replication

The strand
unwinds

Helicase activity separates the duplex; polymerase templates each parent strand into a faithful copy as the cell commits to division. The morph you are watching is replication captured in place — order propagating through living matter.

5'  ATG CGT TAC GGA CCT AGC TAA GCT 3'
Expression 03

Therapeutic
protein, on demand

Ribosomes translate the corrected transcript into functional protein. Dosed expression restores the missing biology — measured, transient, and reversible by design.

12.4×
Yield
36h
Half-life
<0.1%
Off-target
Pipeline
Program
Modality
Indication
Phase
HX-204
siRNA
Hepatic fibrosis
Phase II
HX-118
ASO
Neuromuscular
Phase I
HX-330
AAV gene
Retinal dystrophy
IND-enabling
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