Genetic Medicines for Mucosal Tissues


Over the millennia, mucus has evolved into a formidable barrier against foreign invaders, which has bedeviled gene therapy companies – until now.

Mucosal tissue

Mucosal tissues such as the bladder, lungs, and gut, comprise a vast surface area of the human body. Their exposure to the external environment makes these tissues attractive targets for breakthrough modalities like gene therapy.

While gene therapy has revolutionized many fields of medicine, it has left behind patients suffering from illnesses that manifest in mucosal tissues. enGene was founded on the principle of addressing these underserved conditions with novel genetic medicines.

Gene therapy

Our patented non-viral gene therapies are dosed directly into the lumen of the targeted mucosal tissue. For example, our lung therapies are crafted to be inhaled.

Once inside the luminal cavity, our nanoparticles transport nucleic acid medicines into the mucosal epithelial cells.

The transported genetic medicines can encode for proteins, peptides, antibodies – even small interfering or micro RNAs.

DDX Nanoparticle
no systemic administrations.
no blood exposure.

Coupling DDX to a nucleic acid cargo (like plasmid DNA), we generate gene therapies that are formulated as nanoparticles for direct dosing to the the tissue of interest. In this way, we engage the most impactful biology directly where it’s needed, circumscribe the therapy to the intended site, and mitigate the risk of systemic toxicities.

the carrier for genetic medicines
to mucosal tissues

Dually derivatized chitosan (DDX) is our proprietary carrier for genetic medicines to mucosal tissues and is the foundation for our nanoparticle formulations.

We developed DDX at enGene to penetrate mucus barriers and to deliver genes to mucosal epithelial cells in a way that is re-dosable, biocompatible, and scalable.

Non-viral. Large capacity. High flexibility.

Plasmids afford us the luxury of space to deliver very large genes, or multiple genes, when required.

Plasmids also allow us to apply molecular biology tools, so that we can rationally engineer our therapies, making them our choice nucleic acid source.