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3DNA-miR146b-5p
- Development stage
- Preclinical
- Lead developer
- Code Biotherapeutics
- Modality
- Lipid-mediated Delivery → Gene Addition/Replacement → Gene Therapies, Electrical: Electroporation → Physical Methods → Gene Addition/Replacement → Gene Therapies, Acoustic/Optical: Sonoporation, laser photoporation → Physical Methods → Gene Addition/Replacement → Gene Therapies, Magnetic: Magnetofection → Physical Methods → Gene Addition/Replacement → Gene Therapies, Mechanical: Microinjection, microfluidics, gene gun → Physical Methods → Gene Addition/Replacement → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Pressure-driven: Hydrodynamic injection → Physical Methods → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous, Intraperitoneal
01
Overview
3DNA-miR146b-5p is a non-viral gene therapy candidate developed by Code Biotherapeutics for the treatment of perinatal white matter injury (WMI) and neuroinflammation in premature newborns. The therapy utilizes the proprietary 3DNA® platform, which consists of a multivalent, scaffolded DNA nanostructure, to deliver miR146b-5p (microRNA-146b-5p) specifically to microglia. The mechanism of action involves the modulation of microglia-related neuroinflammation, aiming to reduce the damage associated with WMI. By targeting DLG4 (Discs large homolog 4) or utilizing microglia-specific targeting, the 3DNA platform enables precise delivery of the therapeutic microRNA to the central nervous system.
02
Targets
CCDC6 (Coiled-coil domain-containing protein 6)Pyruvate dehydrogenase E1 subunit beta (PDHB) mRNATBC1 domain family member 14 mRNAPTEN (Phosphatase and tensin homolog deleted on chromosome ten)SWI/SNF-related matrix-associated actin-dependent regulator of chromatin subfamily A member 5 (SMARCA5) mRNAZNRF3 (Zinc and ring finger protein 3)DNMT3A (DNA methyltransferase 3 alpha)Kruppel-like factor 4 (KLF4) mRNAIRAK1 (Interleukin-1 receptor-associated kinase 1)
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