RUMI has developed a transformative, proven process for screening and testing genetic diseases- faster and more effectively than available technologies. By combining the work of Dr. Ali Brivanlou and Dr. Eric Siggia, RUMI has fused stem cell biology and physics to create a transformative method of growing human embryonic stem cells, which self-organize to generate a signature defined by geometry and size. The effect is to provide isogenic lines that can be isolated to test compounds directly on genetic diseases.
RUMI has already generated an isogenic line for the Huntington’s Disease mutation and demonstrated that its signature on our micropatterned colonies is significantly different than its mutation-free counterpart. We have also identified compounds capable of reverting the Huntington’s signature back to normal.
In addition to its groundbreaking work with Huntington’s Disease, the RUMI Technology will provide numerous benefits and opportunities versus the current landscape used by large pharmaceutical companies:
REVOLUTIONARY SCREENING PLATFORM
RUMI’s proprietary technology has created a unique and transformative screening platform for genetic diseases and potential treatments, utilizing single-cell, time-lapse imaging and other customized, analytic tools. No other existing platform can achieve the qualitative and quantitative level of resolution obtained with our technology.
NEW PARADIGM CREATED BY LEADERS OF TWO SYNERGISTIC SCIENCES
The RUMI Technology is the result of cooperation by the heads of the Physics and Stem Cell Biology Laboratories at Rockefeller University, who have worked together for over a decade and combine the disciplines of biology and physics. The result is a revolution in the landscape of drug discovery using a micropattern technology as a new path to grow human embryonic stem cells. The RUMI Technology grows human embryonic stem cells (hESCs) through the use of its own micropatterning technology such that hESCs self-organize to generate their own stereotypical signature defined by geometry and size, thus in effect, replicating the processes which occur in vivo during a cell's early embryonic development. By recapitulating the ongoing cell-to-cell signaling and studying the relationship between cells as they differentiate to give rise to all different human cell types, our RUMI Technology can be applied to solve myriad problems in the drug discovery process.
NUMEROUS ADVANTAGES OVER EXISTING PROCESSES
- Faster: RUMI Technology’s screening process provides significantly faster compound screening than classically targeted assays. It enables companies to perform trials on human micro patterns- leapfrogging time-intensive steps.
- Comparative: Our high-resolution evaluations of cellular behavior allow quick and easy comparisons between normal and diseased states.
- Better Targeted: By using isogenic lines with signature genetic mutations, the RUMI Technology allows specific targeting of genetic mutations, providing a superior method to testing compound efficacy.
- More Quantifiable: Thanks to the geometric and scale-based signature definition in its isogenic lines, the RUMI Technology provides for clear reading of mutations and compound efficacy. RUMI’s proprietary genome-editing tools recapitulate mutations that cause human genetic diseases, generating two stem cell lines that are otherwise identical (“isogenic lines”) and apply high throughput screens on these isogenic lines to identify compounds that revert the diseased cell back to normal.
- Universal: The RUMI Technology overcomes the requirement for a priori knowledge of the function of protein(s) affected in the disease, thus providing a universal platform that can be applied to any human genetic disease.
EXPONENTIAL GROWTH OPPORTUNITY FOR DRUG DEVELOPERS
The RUMI Technology offers numerous transformative benefits to any company interested in developing drugs to treat genetic diseases, including the following:
- Early discovery tool: The RUMI Technology can save companies significant time and money by quickly eliminating dead-ends and focus efforts on the most promising compounds and ideas
- The RUMI Technology can help extend the life of patents on key drugs, for example, by supporting an additional “use” patent claim on a drug or a class of drugs
- The RUMI Technology can fast-track the drug discovery process
- The RUMI Technology can evaluate compound toxicity
- The RUMI Technology provides pathways to accelerated regulatory approvals