Science & Pipeline
We’re unlocking brand-new therapeutic possibilities by modulating mitochondrial function
Dysfunctional mitochondria are involved in more than 50 diseases. The most severe of these are broadly termed mitochondrial diseases, a group of rare genetic conditions which affect individuals of all ages. Mitochondrial dysfunction also plays an important role in more common diseases, including aging-related disorders. None of these diseases has an effective treatment option currently available.
In addition, modulating mitochondrial biology presents a potential approach to the treatment of diseases not directly caused by mitochondrial dysfunction, for instance cancer and metabolic diseases.
Our therapeutic platform joins a deep mechanistic understanding of mitochondrial biology with an array of cutting-edge technologies with which to fine-tune mitochondrial function.
The critical role of mitochondria
The primary function of mitochondria is to generate the energy needed to power our cells. In a process called oxidative phosphorylation, or oxphos for short, a series of protein complexes embedded in the mitochondria’s inner membrane turn nutrients into the cellular energy source, ATP.
Mitochondria have their own DNA, known as mitochondrial DNA (mtDNA). mtDNA is unique because it is exclusively inherited maternally and is organized in a circular structure.
Anywhere from a few hundred to tens of thousands of copies of mtDNA can exist within a single cell, and each individual has many different genetic subtypes of mitochondria present throughout their cells.
Our Approach
We’re tackling the roots of mitochondrial dysfunction in three ways:
Genome Correction
Mutations in mitochondrial DNA (mtDNA) can lead to disease. However, because of mtDNA’s unique properties, not all genome editing approaches (like CRISPR) are possible. We are harnessing specialized gene-editing tools to silence mutated mtDNA and allow healthy copies of mtDNA to proliferate.
This approach could potentially be therapeutic for rare genetic disorders caused by mitochondrial mutations.
Genome Expression Modulation
Information held in mitochondrial DNA is expressed through the processes of DNA replication, transcription and translation. Many different enzymes are involved in these processes. We are developing small-molecule medicines that act on these enzymes in order to modulate mtDNA expression.
This approach could potentially be therapeutic for rare genetic disorders caused by mitochondrial mutations, as well as diseases in metabolism, oncology and neurodegeneration.
Mitochondrial Quality Control
Mitochondria have built-in quality control systems that maintain the health and balance of the organelle, but sometimes they can go awry. We are developing small-molecule medicines that target this machinery to therapeutically increase or decrease mitochondrial function.
This approach could potentially be therapeutic for rare genetic disorders caused by mitochondrial mutations, as well as diseases in metabolism, oncology and neurodegeneration.
Our Pipeline
