Mechanism of Action

Programmable
biology, observed

Helix Bio builds sequence-specific oligonucleotide and gene therapies that recognize a target, copy intent, and express the protein a patient is missing, all read to single-base precision.

Four therapeutic modalities. Thirty-eight validated targets. Eleven programs now in the clinic, each one traced back to a single base that was out of place.

Binding

Target
recognition

A guide strand walks the genome and docks onto its complementary motif. Watson and Crick pairing is read base by base, so affinity is sequence-specific: a single mismatch reads differently from a perfect site.

0.4 nM
Affinity
>99.6%
Specificity
Replication

The strand
unwinds

Helicase peels the duplex apart; polymerase reads each parent strand and lays down a faithful copy while the cell commits to division. What morphs on screen is replication caught in place, order copying itself forward through living matter.

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

Therapeutic
protein, on demand

Ribosomes translate the corrected transcript into working protein. The dose restores biology that was missing, then clears: measured, transient, 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
Collaborate

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therapeutic with us.

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