Large molecules like DNA, RNA, proteins, and peptides can function as effective macromolecular medicines for treating a wide range of diseases. However, their efficacy is often hampered by the challenge of traversing the cell membrane into the cytoplasm.
Our unique delivery platform, the Molecular Nano Motor (MNM) technology, makes it possible to bring large molecules through the cell membrane and into the cell, unlocking new pharmaceutical possibilities.
Our MNM platform is highly versatile and can be deployed in diverse therapeutic areas, equipped with various types of molecular cargo. Given the great benefits of the MNM technology, we are focusing on prominent infectious respiratory viruses such as SARS-CoV-2, influenza, and RSV, as well as chronic respiratory diseases, neurological disorders, and intracellular peptide-drug delivery.
Respiratory viruses are a cause of significant morbidity and mortality worldwide. Three respiratory viruses are under constant surveillance by health authorities: SARS-CoV-2, influenza, and RSV. Interna is on a mission to develop a prophylaxis and post-treatment solution in the form of an intranasal formulation that contains a highly potent siRNA-MNM conjugate targeting a conserved site in each virus. For each virus there is an active research & development program whereby the SARS-CoV-2 is the most advanced. In September 2024 Interna began a Phase I clinical trial with an intranasal anti-SARS-CoV-2 MNM-siRNA drug that demonstrated in preclinical studies high therapeutic efficacy and favorable safety profile.
Idiopathic pulmonary fibrosis (IPF) is a life-debilitating chronic lung disease characterized by progressive scarring of the lung. Patients with IPF suffer from shortness of breath, persistent dry cough, fatigue, and if left untreated, the progressive scarring can lead to the necessity for lung transplantation. Despite advancements in the understanding of the pathophysiology of the disease, treatment options for IPF are limited. Interna Therapeutics has an active anti-IPF program that is based on an MNM-siRNA conjugate targeting a gene of interest. Positive proof of concept was observed in a bleomycin-induced IPF mouse model, and several other candidates are being studied.
As our understanding of the pathogenesis of neurodegenerative disease deepens, it has become possible to identify specific genetic sequences whose active downregulation using gene therapy has the potential to treat diseases for which no treatments exist. Direct administration into the spinal canal (Intrathecal administration) of MNM conjugated to siRNA provides a mode of direct administration via which our revolutionary delivery technology can be brought to bear on these inherited neurological diseases. Interna is working in collaboration with its partners on tailoring the MNM platform to a range of severe and untreated heritable diseases of the nervous system.
Peptides are very suitable for targeting intracellular protein-protein interactions but have poor cell permeability. Conjugating MNM to peptides or small proteins can potentially lead to effective interactions with their intracellular target. We have shown in a POC study that an MNM-peptide led to an improved intracellular activity as compared to a CPP-peptide. Moreover, MNM conjugation to this peptide in addition to the CPP unit resulted in a ~20-fold enhanced intracellular activity indicating a synergistic effect.
The MNM platform has an incredibly broad scope. It offers the potential to address additional therapeutic areas such as gastrointestinal conditions, hearing disorders, cardiovascular diseases, eye diseases, and more.