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ADP065-ABC
- Development stage
- Preclinical
- Lead developer
- Alector
- 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
- Administration
- Subcutaneous
01
Overview
ADP065-ABC is an early-stage therapeutic candidate being developed by Alector in collaboration with GSK for the treatment of neurodegenerative conditions, including Alzheimer's disease and Parkinson's disease. It consists of a small interfering RNA (siRNA) designed to silence the expression of NLRP3 (NOD-, LRR-, and pyrin domain-containing protein 3), a key component of the inflammasome that drives neuroinflammation. To overcome the blood-brain barrier, the drug utilizes the proprietary Alector Brain Carrier (ABC) technology, which facilitates targeted delivery to the brain by binding to a distinct region of the transferrin receptor (TfR). The program is currently in the research phase, focused on lead candidate selection.
02
Targets
NLRP3 (Nod-like receptor family pyrin domain-containing protein 3)TFRC (Transferrin Receptor)
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