SKIP Therapeutics develops RNA-based therapies, specifically Antisense Oligonucleotides (ASOs), to address rare genetic disorders and common diseases. The company utilizes a proprietary computational discovery engine to optimize target selection and therapeutic design for modulating gene function. This platform integrates genomic and proteomic data to efficiently identify and evaluate ASO strategies for splicing modulation and gene expression control.
Funding
Funding not disclosed
Founders
Product
Problem
Many rare genetic disorders and common diseases lack effective treatments due to the difficulty in targeting mutated genes and restoring proper protein function. Identifying patient cohorts that would benefit from specific therapies and designing effective RNA-based treatments remains a significant challenge.
Solution
SKIP Therapeutics develops RNA-based therapies, specifically Antisense Oligonucleotides (ASOs), using a computational discovery engine to identify treatable patient cohorts and optimize target selection. Their approach focuses on restoring the protein function of mutated genes in rare genetic disorders or modifying key genes in major disease pathways. The computational platform leverages genomic and proteomic data to assess the functionality of new isoforms and identify potential ASO-based strategies.
Target Audience
The primary target audience includes patients with rare genetic disorders and common diseases, as well as medical institutions, sequencing centers, and patient organizations.
Features
- Proprietary computational discovery engine for identifying and evaluating ASO-based therapeutic strategies
- Utilizes sequence-based molecules (ASOs) to modulate gene function by binding to regulatory regions on RNA
- ASO strategies include: exclusion of exons, prevention of abnormal splicing, elevation of gene expression, modulation of isoform balance, and control of gene function through manipulation of protein domains
- High-throughput analysis of mutations from patient registries to identify treatable patient cohorts
- Pipeline includes programs focused on retinal disease, pulmonary disease, muscular dystrophy, renal disease, and CNS disorders