Delivering large molecules to intracellular targets
The most important drug targets for both infectious and non-infectious diseases are intracellular macromolecules like DNA, RNA, and proteins.
The challenge
Biomacromolecules like DNA, RNA, and peptides can regulate gene expression and protein activity, functioning as powerful therapeutics for treating a wide range of diseases including cancers, genetic disorders, and infectious diseases. However, their large size and polarity have limited their impact, as they cannot readily cross the cell membrane to realize their therapeutic potential.
Introducing the MNM
Interna has developed a novel delivery molecule designed to cross the cell membrane, which we call the molecular nano-motor (MNM). Have a look:
Attachment
Through simple covalent attachment, almost any cargo can bind to the MNM molecule, including DNA, RNA, and proteins.
Detachment
Within the cell, the bond is reduced, detaching the MNM from its cargo.
Membrane association
The MNM is hydrophobic and can pass freely through the cell membrane.
Dipole inducing
The relative negative end of the MNM dipole will be drawn towards the center of the cell membrane.
The MNM can be easily attached to almost any large molecule cargo, including DNA, RNA, proteins and more. The simplicity of the MNM platform means it is well-prepared for rapid adaptation and deployment in diverse therapeutic areas.
Any macromolecule cargo
The MNM’s simple covalent attachment supports myriad different large molecule cargo such as siRNA, ASO, peptides, and nanobodies.
Through the cell membrane
Cargo is delivered directly into the cytoplasm via a number of different pathways, including membrane flip-flop and endocytosis. Once inside the cytoplasm, the MNM detaches, liberating cargo to perform its therapeutic task, while the remnant MNM is metabolized and excreted.
Leveraging a universal source of energy
If DNA, RNA and protein molecules cannot cross the membrane on their own, it is because of the large energetic cost associated with the interaction of charged molecules with the hydrophobic core of cell membranes.
The MNM overcomes this energetic barrier by interacting with an electrical potential inherent within all cell membranes: the membrane dipole potential.*
The electric energy is sourced from the internal membrane electric field and translated into kinetic energy, enabling movement of the MNM and its cargo within the hydrophobic membrane core.
* Wang L. Annual Review of Biochemistry 81: 615-635, 2012Interna is the first and only company to make use of this source of energy for medical applications.
Any administration method
An MNM solution is stable at room temperature and water soluble. This means it can be distributed in a large array of administration methods, including intranasal spray, inhalation, intravenous injection, oral administration, subcutaneous and intertumoral injection.
Any therapeutic area
The scope of our MNM platform is one of its key strengths; it is a highly versatile solution capable of impacting a growing number of therapeutic areas. Interna Therapeutics is currently focusing on infections and non-infectious respiratory diseases.