Stores genetic information
Instructions to create proteins
Basic building blocks of cells
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.
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).
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.
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.