SB-509

SB-509 is an investigational gene therapy developed by Sangamo BioSciences (now Sangamo Therapeutics) for the treatment of neurological disorders such as diabetic neuropathy and amyotrophic lateral sclerosis (ALS). It is an injectable plasmid encoding a DNA-binding zinc finger protein transcription factor designed to upregulate endogenous expression of vascular endothelial growth factor A (VEGF-A). VEGF-A has angiogenic, neurotrophic, and neuroprotective properties. By increasing VEGF-A production in muscle and nerve cells at the injection site, SB-509 aims to promote nerve regeneration and repair damaged nerves and muscles. Clinical trials demonstrated increased intraepidermal nerve fiber density in treated patients with diabetic neuropathy, but development was discontinued after Phase 2 trials for ALS, diabetic neuropathies, brain injuries, spinal cord injuries, and stroke[3][6][8].