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GAL101

Development stage: Preclinical
Lead developer: Galimedix Therapeutics
Modality: Gene Therapies
Administration: Oral, Ophthalmic, Intravitreal, Subcutaneous

Overview

GAL101 is an adeno-associated virus (AAV)-based gene therapy being developed by Galibra Neuroscience for the treatment of SLC6A1-related disorders, including epilepsy and intellectual disability. The therapy utilizes an AAV vector to deliver a functional copy of the SLC6A1 gene, which encodes the sodium- and chloride-dependent GABA transporter 1 (GAT-1). By restoring functional SLC6A1 expression, GAL101 aims to correct the transporter deficiency that leads to impaired GABA reuptake and subsequent neurological symptoms. The program is currently in the preclinical stage of development.

Targets

SLC6A1 (Sodium- and chloride-dependent GABA transporter 1)

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