Disease Specific Research

Genescient is a Genomics 2.0 corporation with unique expertise, technology, and focus on general age-associated disorders like heart disease and acquired metabolic disorders. In contrast to the industry standard approach (Genomics 1.0), Genescient has a unique genomic platform for finding the genomic targets of many human diseases. Our platform has already identified clear targets for cardiovascular disease and acquired metabolic disorders.

In our terminology, Genomics 1.0 technologies take advantage of the nearly complete DNA sequencing of the human genome accomplished within the past decade. With genome-wide tests for differences in the genetic make-up or genetic functioning of patients, the hope was that we would soon be able to discover the key foundations of most, if not all, chronic human disorders, from heart disease to cancer. This was a legitimate expectation, because before the advent of genomics research it was difficult to consider more than a few genetic pathways at a time as candidate mechanisms determining the onset of endogenous medical disorders.

Among the Genomics 1.0 technologies are Genome-Wide Association Studies (or “GWAS”). GWAS are used to try to identify the genes that underlie common human disorders. GWAS research has shown its power to uncover a few key genetic loci that are responsible for major elevations in the risk of such disorders as macular degeneration and Alzheimer’s Disease. In addition, GWAS research has identified many more genes that slightly increase the risk of developing such disorders. But GWAS and other genomic 1.0 technologies have generally failed to supply medicine with definitive diagnostic tools. This has also led to concern that the genetic pathways uncovered by Genomics 1.0 technologies will not provide useful targets for the treatment of most diseases. You may have read the recent New York Times article that addresses this concern, an article that echoed several papers published in the New England Journal of Medicine.

Genomics 1.0 researchers feel that they can do much better when they are working from nearly-complete human genome sequences. We agree, that we will indeed do better when they have access to such information. But there remains the general problem that the variation present in human genomes provides only a faint signal for inferring the underlying genetic risk factors associated with chronic age-associated disorders.

Genescient was started by scientists who realized a decade ago that Genomics 1.0 was unlikely to provide the scientific power and technological leverage to make progress with the most common age-associated disorders. From that starting point, they have developed a model-system strategy that is designed to increase the medical value of genomic technologies. At the core of their strategy is increasing the power of genomic signals. We call this powered-up approach Genomics 2.0.

Genomics 2.0 is based on approaches that increase the amplitude of genomic signals in model animal species. This is key to increasing the power of genomics, because genomic tests over entire genomes tend to have so much statistical noise that is very hard for scientists to discern the “signal” of biomedical value without some type of amplification. There are a variety of conceivable Genomics 2.0 strategies, but we believe that we are the first corporation to be based on a fully-developed Genomics 2.0 technology.

The amplified genomic signal that Genescient uses is based on long-sustained and well-replicated selection experiments. We select on model organisms for long-term maintenance of optimal health and function, over hundreds of biological generations, using multiple populations in parallel. Our most analyzed selected model organism is Drosophila (i.e. fruit flies), and we fully expect that Genescient will soon have data on selected laboratory mice as well.

Lab selected animals can have their functional lifespans, or “healthspans,” increased three to five fold by sustained selection. In order to live for that long, we know that they need to have their cardiac, metabolic, and other functions greatly “tuned up.” This tuning up takes place throughout the genome. Modern genomic tools enable us to identify many of the genes associated with this tuned-up state. Because selection accumulates signal every generation, in genomic terms the animals with increased healthspan provide unparalleled genomic information. Whereas a Genomics 1.0 approach to these animal models might detect one or at best a dozen or so key genetic pathways, our Genomics 2.0 approach enables us to detect hundreds of such pathways. That is, we increase the amplitude of genomic signals between 100 and 1000-fold.

Using its patent-pending technology, Genescient takes the strong-signal genomic information that massive selection provides and searches through existing GWAS human genomic datasets. This is done to confirm in humans, the animal genomic findings. This is a key step, because not everything that is important for the health of a laboratory animal will also be important for human health or have an impact on human disease. When we have such confirmation of the animal genomic signal in human genomics, we are confident that we have found a key biomedical pathway that plays a role in the health of both the animal model and human patients.

Genescient then uses the model animals to identify, test, and validate candidate medicines for treating the disorders which are shared by both the animal model and patients. Among the disorders we have targeted this way are cardiovascular and neurological disorders.

We have already cleared major hurdles in the application of our Genomics 2.0 strategy, and welcome the opportunity to partner with companies and advocacy organizations seeking genomic solutions for specific diseases.

For more information on collaboration opportunities with Genescient, please Contact Us.