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tricyclo-DNA
- Development stage
- Preclinical
- Lead developer
- Sqy Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous, Subcutaneous, Intracerebroventricular, Intrathecal
01
Overview
Tricyclo-DNA (tcDNA) is a synthetic nucleic acid analog and antisense oligonucleotide (ASO) modality characterized by a rigid tricyclic modification in the sugar backbone. This structural modification confers unique pharmacological properties, including high binding affinity to complementary RNA, enhanced resistance to nucleases, and improved tissue distribution compared to earlier ASO chemistries. tcDNA is primarily utilized for splice-switching applications, such as inducing exon skipping to restore the reading frame in Duchenne Muscular Dystrophy (DMD) or modulating splicing in Spinal Muscular Atrophy (SMA). Developed by Synthena, tcDNA has demonstrated the ability to reach the central nervous system and various peripheral tissues after systemic administration, making it a promising candidate for treating neuromuscular and neurological disorders.
- Other names
- tc-DNAtricyclo-DNA oligomers
02
Targets
SMN2 (Survival motor neuron 2)Neuraminidase
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