SYN1

SYN1 and SYN2 are lead small molecule regenerative therapy (SMRT) candidates developed by Thera Neuropharma for the treatment of Amyotrophic Lateral Sclerosis (ALS). Licensed from Southern Research, these compounds are designed to selectively activate the NF-κB p65 subunit and increase the expression of manganese superoxide dismutase (MnSOD/SOD2) in neurons. SYN1 specifically targets superoxide dismutase 1 (SOD1) misfolding and the formation of toxic intracellular aggregates. This multi-target approach is intended to protect motor neurons from oxidative stress and protein misfolding, promote neuritic and axonal regeneration to restore neuronal networks, and activate compensatory mechanisms against neurotrophic deficits. In preclinical SOD1-G93A animal models, these SMRT compounds demonstrated significant efficacy, including a greater than 50% increase in survival, reduced weight loss, and delayed disease progression.

Other names

SMRTSmall Molecule Regenerative TherapyThera Neuropharma-NF-κB p65-MnSOD-SOD1-small molecule (SMRT - Small Molecule Regenerative Therapy)-Amyotrophic Lateral Sclerosis (ALS)