COMPUTATIONALLY GUIDED DEVELOPMENT OF RNA-BASED THERAPIES.

Name: SKiP therapeutics
Address: 2 אילן רמון, נס ציונה, IL
Telephone: Tel: +972-(0)8-955-3149

Unlocking Therapeutic Potential at the Convergence of Computation and RNA

ABOUT

SKIP Therapeutics develops RNA-based therapies for rare genetic disorders and common diseases – utilizing a proprietary computational discovery engine to optimize target selection and therapeutic design.

We employ sequence-based molecules, called Antisense Oligonucleotides (ASO), to restore function of mutated genes in rare genetic disorders or to modify functionality of key genes in major disease pathways.

Convergence of SKIP’s novel computational discovery engine alongside strong experimental capabilities form a unique venture in the landscape of RNA-based therapies.

Antisense oligonucleotides (ASOs) are emerging as an important therapeutic modality that can be utilized to modulate gene function in the cell. These molecules are designed to bind regulatory regions on the RNA through precise complementary base pairing and induce change in RNA-splicing, expression, and functionality. Strategies include:

TECHNOLOGY

Modulating RNA using ASOs

Antisense oligonucleotides (ASOs) are emerging as an important therapeutic modality that can be utilized to modulate gene function in the cell. These molecules are designed to bind regulatory regions on the RNA through precise complementary base pairing and induce change in RNA-splicing, expression, and functionality. Strategies include:

Exclusion (skipping) of exons to overcome stop codon mutations.

Prevention of abnormal splicing caused by point mutations.

Elevation of gene expression through targeting RNA regulatory regions.

Modulation of isoform balance and creation of novel isoforms.

Control of gene function through manipulation of protein domains.

TEAM

SKIP has created a computational platform, that identifies and evaluates potential ASO-based strategies to restore mutated gene’s function in rare genetic disorders, or to modify gene function in common disease pathways. Leveraging genomic and proteomic data and “learning from” the comprehensive landscape of known pathogenic mutations, our method can assess the functionality of new isoforms. High throughput of SKIPs computational engine enables large scale analysis of mutations from patient registries and organizations and identification of “treatable” patient-cohorts around the world.

SKIP’s Computational Engine.

SKIP has created a computational platform, that identifies and evaluates potential ASO-based strategies to restore mutated gene’s function in rare genetic disorders, or to modify gene function in common disease pathways. Leveraging genomic and proteomic data and “learning from” the comprehensive landscape of known pathogenic mutations, our method can assess the functionality of new isoforms. High throughput of SKIPs computational engine enables large scale analysis of mutations from patient registries and organizations and identification of “treatable” patient-cohorts around the world.