What if a therapy worked like a computer inside a cell?
First
we write the code
The code is a molecular program that guides the therapy once it is administered to the patient. An example of a molecular program is "Eliminate a cell that expresses factors A and B and does not express factor C". To design these programs, we analyze genomics, transcriptomics, and proteomics data to understand differential molecular profiles of diseased and healthy cells. We then use these profiles as the foundation for the DNA computer's instructions.
Second
we compile
We compile the molecular program into a DNA sequence in a systematic way using Pattern's proprietary technologies. The resulting DNA computer executes the program within patient cells. These computers have three main components: (1) the sense module that interacts with the cellular inputs as instructed by the code; (2) the compute module that executes the program; and (3) the respond module that activates a potent therapeutic effector when the program identifies a disease-specific molecular pattern inside a cell.
Third
we test
We use a data-driven bottom-up engineering approach to design and build our therapeutic candidates. Accordingly, their testing follows a systematic workflow that is similar to debugging a computer program. Failure modes can be quickly identified and corrected in an iterative process. We use sophisticated in vitro and in vivo disease models that recapitulate patients' molecular patterns and human biology to maximize translational relevance of preclinical testing.
See how we built a drug to reprogram liver cancer in mice.
The ability to program a therapy to make nuanced treatment decisions in single cells and address diseases at their root cause will greatly improve patient outcomes and create massive value for the healthcare system
We compute inside human cells using transcription factors, microRNAs, promoters and enhancers. Many of these inputs are key disease drivers that are generally undruggable with conventional small molecule or biologic therapies. Leveraging these inputs to drive treatment decisions in individual cells unlocks our ability to treat diseases at their root cause.
Ample knowledge exists about which molecules can efficiently eliminate or reprogram cells. However, these molecules are often very toxic on their own and cannot be administered to patients systemically. In combination with Pattern's precision molecular programs, they can be used as therapeutic effectors in a safe manner.
Our systematic design process generates therapeutic candidates at shorter timescales, reduced cost, and higher probability of success.
Pattern's approach has the potential to generate tailored cancer therapies that will eradicate tumors without causing significant adverse effects. This will unlock their use in early-stage cancer treatment as an alternative to surgery.
Pattern BioSciences mission is made possible by some of the most revolutionary.....
Keller Rinaudo Cliffton
Founder and CEO of Zipline