We are a pioneer in the discovery of genetic modifiers and the subsequent development of novel therapies.
Enabling the development of disease modifying therapeutics
This is how we unlock groundbreaking science
The genes that counteract the effect of a disease-causing gene
They explain why some people with genetic mutations linked to severe disease have only mild or no symptoms. Genetic modifiers therefore positively influence the severity of disease and act as a ‘natural form of protection’. To date genetic modifier or suppressor genes have been difficult to identify, especially for rare genetic disorders. Now, for the first time, Scenic is systemically exploring genetic modifiers using its proprietary platform technology for a large variety of disorders, including inherited diseases.
Genetic modifiers or disease suppressors are genes that counteract the effect of a disease-causing gene.
They explain why some people with genetic mutations linked to severe disease have only mild or no symptoms. Genetic modifiers therefore positively influence the severity of disease and act as a ‘natural form of protection’.
To date genetic modifier or suppressor genes have been difficult to identify using population genetics, especially for rare genetic disorders. Furthermore, data from population genetics alone do not provide direct insight into the underlying biological process that causes disease suppression.
Scenic Biotech has overcome the technical challenges of reliably finding genetic modifiers or the “hidden disease protection factors” using Cell-Seq, its unique discovery platform. This has resulted in a rapidly growing pipeline of novel disease therapeutics, and collaborations with partners.
Our platform, Cell-Seq, enables a comprehensive overview beyond genetic modifiers for many different disease phenotypes to select the best targets.
Cell-Seq uses special human cells that carry only a single copy of every gene (haploid), instead of the usual two (diploid) In addition to the primary disease-causing mutation, individual genes in billions of these haploid human cells are inactivated to create a very large library of mutant cells. This inactivation involves a process called gene-trap mutagenesis so that nearly every gene in the genome is inactivated/disabled, often several thousands of times.
The library of mutant cells is then stained using fluorescent labelling for a specific disease/pathway marker, such as a disease-associated process, to quantify how the gene mutation affects the cell’s health.
Cells with the cellular phenotype of interest are isolated using a FACS (fluorescent activated cell sorter) and the experimentally introduced mutations are determined using modern DNA sequencing technology.
Bioinformatics analysis then identifies every gene that modifies the cellular phenotype to enable high resolution “disease maps” to be generated so that the best disease genetic modifiers can be viewed and then selected for target validation and drug development. Hundreds of these “maps” have been generated to date.
The Cell-Seq platform is used on healthy and diseased cells in parallel to unlock disease-specific genetic modifiers, including disease suppressors. In addition, our proprietary data-mining tools allow to map previously uncharted biological pathways and interrogate disease biology in an unprecedented fashion.
We are enabling the development of disease modifying therapeutics for devastating diseases including inherited rare diseases and cancer.