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GMS-P11
- Development stage
- Preclinical
- Lead developer
- Gifted Molecular Science
- 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
GMS-P11 is a preclinical-stage antisense oligonucleotide (ASO) candidate being developed by Gifted Molecular Science for the treatment of central nervous system (CNS) genetic disorders. The therapeutic is designed using the company's proprietary fourth-generation Druggable-PNA (peptide nucleic acid) platform technology. This platform incorporates a cationic lipid segment into the nucleobase of a metabolically stable PNA backbone, which enhances cell permeability and increases binding affinity to target mRNA sequences. GMS-P11 is intended to modulate or correct disease-associated genetic targets in the CNS, though the specific molecular target has not been publicly disclosed.
- Other names
- Genetic Disorder program
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