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DOL-001
- Development stage
- Preclinical
- Lead developer
- Dolinnov
- 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
- Administration
- Intrathecal
01
Overview
DOL-001 is a first-in-class, non-opioid lipid-conjugated antisense oligonucleotide (L-ASO) being developed by Dolinnov for the treatment of severe chronic pain, with primary focus on severe cancer pain and rare pain disorders. It acts as a Precision Pump Modulator (PPM) by targeting and down-regulating FXYD2 (FXYD domain containing ion transport regulator 2), which is the gamma subunit of the Na+/K+-ATPase pump. FXYD2 is highly expressed in the dorsal root ganglion (DRG) of the peripheral nervous system and plays a key role in regulating neuronal hyperexcitability. By silencing FXYD2, DOL-001 reduces pathological nerve activity and alleviates chronic peripheral pain without central nervous system exposure, thereby avoiding the side effects and addiction risks associated with opioids. The drug is currently in preclinical development.
- Other names
- ASODOL
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