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SKiP-CNS
- Development stage
- Preclinical
- Lead developer
- Skip Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
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Overview
SKiP-CNS is an antisense oligonucleotide (ASO) therapy being developed by SKiP Therapeutics for the treatment of central nervous system (CNS) diseases. The program utilizes ASO-mediated exon skipping to modulate the splicing of disease-related pre-mRNA transcripts. By inducing the cellular splicing machinery to bypass specific exons, the therapy aims to restore the translational reading frame or otherwise modulate the expression of proteins associated with CNS pathology. As of early 2024, the program is in the preclinical stage of development, utilizing the company's proprietary computational platform for target identification and experimental validation.
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