CORRECTING DYSFUNCTIONAL RNA,
THE ROOT CAUSE OF MANY GENETIC DISEASES
Our CORRECTX platform of RNA binding protein systems precisely target and correct the dysfunctional messenger RNA that causes many genetic diseases. Our gene therapy approach may lead to potentially life-changing therapies for patients suffering from these devastating disorders with a one-time administration.
CORRECTING DYSFUNCTIONAL RNA,
THE ROOT CAUSE OF MANY GENETIC DISEASES
Our CORRECTX platform of RNA binding protein systems precisely target and correct the dysfunctional messenger RNA that causes many genetic diseases. Our gene therapy approach may lead to potentially life-changing therapies for patients suffering from these devastating disorders with a one-time administration.

The CORRECTX platform enables the development of modular RNA binding protein systems that can modify RNA with multiple mechanistic approaches; cleavage, splicing, gene replacement, translation enhancement and editing. These systems offer unique features that address the limitations of other RNA targeted approaches and may lead to new genetic medicines for patients with neurodegenerative, neuromuscular and retinal diseases.

PRECISELY
targets and corrects
disease-causing RNA
PRESERVES
the healthy mRNA
transcript
SELF-CONTAINED
with no need for
cellular machinery
DELIVERED
in a one-time
gene therapy
administration

SCIENTIFIC POSTERS AND PUBLICATIONS