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PNL3
- Development stage
- Preclinical
- Lead developer
- Islamic Azad University
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
01
Overview
PNL3 is an experimental PEGylated nanoliposome (PNL) formulation developed as a multifunctional therapeutic candidate for the treatment of synucleinopathies, such as Parkinson's disease. It is part of a series of five nanoliposomes (PNL1 through PNL5) designed to cross the blood-brain barrier and target multiple aspects of alpha-synuclein (α-syn) pathology. PNL3 is engineered to inhibit the fibrillization of α-syn proteins, attenuate microglial activation (thereby reducing the production of pro-inflammatory cytokines like TNF-α and IL-6), and silence the expression of the SNCA gene through RNA interference. While PNL3 was evaluated in comparative preclinical studies, the research specifically highlighted PNL1 and PNL2 for their significant efficacy in silencing SNCA and inhibiting α-syn aggregation in both cell cultures and transgenic mouse models.
- Other names
- PEGylated nanoliposome 3
02
Targets
SNCA (Alpha-synuclein)
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