Delivering solutions through RNA therapeutics.

How Genes Work

Strands of DNA within the nucleus of every cell in the human body contain the instructions for assembling proteins. Those genetic instructions are copied to messenger RNA (mRNA) which travel from the nucleus to the cytoplasm. In the cytoplasm, ribosomes use the instructions carried by mRNA to construct proteins out of amino acids. This process is called protein synthesis and it is what makes life possible.

DNA

Stores genetic information

mRNA

Instructions to create proteins

Proteins

Basic building blocks of cells

What Can Go Wrong

Errors in the instructions stored within DNA lead to the absence, overproduction, or dysfunction of specific proteins. When these genetic errors disrupt normal bodily functions, the consequences can be serious and even fatal. Over 10,000 monogenic disorders have been identified to date.

The Potential of RNA Therapeutics

RNA-based therapeutics represent a novel approach to correcting genetic disorders. There are three leading RNA therapeutic modalities, each of which has a distinct method of modulating and regulating gene expression.

mRNA

For disorders characterized by insufficient or faulty protein production, therapeutic mRNA can be crafted to provide instructions for ribosomes to create more of the missing protein.

If the correct instructions are properly delivered, therapeutic mRNA can be used to address formerly “undruggable” targets.

RNAi

For disorders characterized by overproduction of a particular protein, RNA interference (RNAi) can be utilized to silence the improper instructions carried by mRNA from the nucleus.

RNAi therapeutics are derived from a naturally occurring mechanism within cells and active therapeutics are called small interfering RNA (siRNA).

Gene Editing

Finally, gene editing systems can be used to permanently fix disease-causing errors by cutting the DNA within cells at precise locations and inserting corrected copies of the affected genes.

Gene editing systems in development today include CRISPR, ZFNs, TALENs, and meganucleases.

The Challenge of RNA Therapeutics

Efficient and effective intracellular delivery of RNA therapeutics is difficult because these therapeutics are prone to rapid degradation and excretion in the bloodstream and do not pass freely through cell membranes. Furthermore, while the full suite of RNA modalities have the potential to treat nearly every genetic disorder, biopharmaceutical companies are singularly focused on individual modalities.

Our Pan-RNA Approach

Through our industry-leading lipid nanoparticle (LNP) and ligand conjugate delivery platforms, we are able to select and pursue the optimal approach for any given disease. We intend to discover and develop products in-house and form partnerships to build a diverse pipeline of therapeutics across leading RNA modalities, including RNAi, mRNA, and gene editing.


Our Goals

Multiple Programs in the Clinic by 2020

By 2020, we aim to have 5 to 10 RNA programs in the clinic targeting a range of genetic disorders with limited or no treatment options.

Industry Leader in RNA Therapeutics

We plan to develop products in-house and form partnerships to build a diverse pipeline of RNA therapeutics across multiple modalities.

Positive Impact on the Lives of Patients

We hope to deliver innovative and clinically meaningful therapies that improve the lives of patients with genetic disorders.

Interested in learning more?

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