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GABRA1
- Development stage
- Preclinical
- Lead developer
- Grann
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
01
Overview
GABRA1 is a preclinical mRNA-based therapeutic being developed by Grann Pharmaceuticals for the treatment of neurological disorders and epilepsy associated with variants in the GABRA1 gene. The therapy utilizes mRNA technology to deliver the genetic code for the GABA-A receptor subunit alpha-1, aiming to restore or replace the function of defective subunits within the GABA-A receptor complex. This restoration is intended to normalize inhibitory neurotransmission in the brain. The drug is expected to utilize Grann's proprietary lipid nanoparticle (LNP) delivery systems, potentially incorporating blood-brain barrier (BBB) crossing technology to ensure effective central nervous system penetration.
- Other names
- Grann-GABRA1Grann-GABRA-1Grann-GABRA 1GABRA1 mRNAGABRA-1 mRNAGABRA 1 mRNA
02
Targets
GABRA1 (Gamma-aminobutyric acid type A receptor alpha 1 subunit)
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