Scientific Platform

A transformative approach starts within

Restoring Protein Function

Using our proprietary mRNA therapeutic platform (MRT), we create mRNA that encodes functional proteins. When our mRNA is delivered to the target cell, the cell’s own machinery recognizes it and translates it, restoring or augmenting protein function to treat or prevent disease.

Figure 1. mRNA is encapsulated in a lipid nanoparticle and delivered to the target cell where the cell’s own machinery recognizes it and translates it, restoring or augmenting protein function to treat or prevent disease.

MRT Platform

Our MRT platform has enabled us to develop product candidates designed to deliver mRNA that can carry instructions to produce intracellular, transmembrane and secreted proteins. Our platform is also designed to be flexible and scalable by allowing for the development of MRT product candidates that vary only in the mRNA sequence and the tissue-specific delivery vehicle. This modular nature of our platform may allow us to rapidly advance into new indications after successfully establishing delivery vehicles for specific tissues. For example, we are utilizing our MRT platform to identify and rapidly develop new product candidates designed to address the underlying causes of additional diseases of the lung and liver.

Design, Delivery and Manufacturing

We believe that the mRNA design, delivery and manufacturing capabilities of our MRT platform provide us with the most advanced platform for developing product candidates that deliver mRNA encoding functional proteins for therapeutic uses.

Figure 2. Our MRT product candidates consist of two parts: the protein-coding mRNA and a delivery vehicle

Delivering the desired mRNA sequences is the first step to restoring healthy function to proteins. We design our proprietary mRNA sequences to encode the natural protein sequences and we use unmodified mRNA bases to replicate the composition and function of endogenous mRNA. We then further optimize the sequences to result in efficient protein production.

After we create the desired mRNA sequences, we then package our mRNA sequences into delivery vehicles, such as our LNPs, that are customized for delivery to specific tissues. We design our delivery vehicles for optimal size, surface charge and lipid composition.

Broad Applicability of our MRT Platform

We believe that our MRT platform may be applied across a broad array of indications and target tissues via multiple routes of administration for the treatment of rare and non-rare diseases, including CNS disorders, ocular diseases and blood disorders. We believe our platform may also be applied to produce therapeutic antibodies and vaccines in areas such as infectious disease and oncology. We are utilizing our MRT platform to identify and rapidly develop new product candidates designed to address the underlying causes of diseases beyond our initial efforts in CF and OTC deficiency.

Areas of Focus

Learn where our mRNA technology is having an impact