Scenic is unlocking the ground-breaking science of genetic modifiers to develop breakthrough therapies - we call this approach - Evolution Inspired Medicine.

Scenic Biotech is a pioneer in the discovery of genetic modifiers – an untapped class of drug targets – enabling the development of disease modifying therapeutics. While our programs are primarily focused on rare genetic diseases and cancer our experimental approach is pathway agnostic and can pinpoint genetic modifier targets relevant to diverse diseases.

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Genetic modifiers – a completely new class of therapeutic/drug targets.
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 end up having 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 does 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 large-scale pioneering human cell genetics platform.

Genetic modifier white paper – read our white paper entitled: “Exploiting genetic modifiers to treat disease”

Cell-Seq – proven technology platform for target discovery
Our platform, Cell-Seq, enables a comprehensive overview of 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). 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 pinpointed for target development.

The Cell-Seq platform is used on healthy and diseased cells in parallel to unlock disease-specific genetic modifiers, including disease suppressors.
Video – Watch how we discover genetic modifiers using our Cell-Seq Platform.

Read more about genetic modifiers:
1: Brockmann M, et al. Nature. 2017 PMID:28562590
2: Mezzadra R, et al. Nature. 2017 PMID:28813410
3: Staring J et al. Nature. 2017 PMID: 28077878.
4: Nieuwenhuis J, et al. Science. 2017 PMID: 29146869
5: Blomen VA, Science. 2015 PMID: 26472760.