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GABRG2
- Development stage
- Preclinical
- Lead developer
- Grann
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intrathecal
01
Overview
GABRG2 is an mRNA-based therapeutic candidate being developed by Grann Pharmaceuticals for the treatment of epilepsy and other neurological disorders associated with GABA-A receptor variants. The drug is designed to deliver mRNA encoding the gamma-2 subunit of the GABA-A receptor (GABRG2) to the central nervous system, aiming to restore functional inhibitory neurotransmission in patients with mutations in this subunit. Grann utilizes its proprietary lipid nanoparticle (LNP) or solid lipid nanoparticle (SLN) delivery systems, which may incorporate blood-brain barrier (BBB) crossing technology to facilitate delivery to target neurons. The program is currently in the preclinical stage of development.
- Other names
- Grann-GABRG2Grann-GABRG-2Grann-GABRG 2GABA-A receptor subunit gamma-2 mRNA
02
Targets
GABRG2 (GABA-A receptor subunit gamma-2)
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