SP7737

SP7737 is a micro-peptide (small peptide) that functions as a negative modulator of the mechanistic target of rapamycin complex 1 (mTORC1). It is being developed as a gene therapy for mTORopathies, specifically focal cortical dysplasia type II (FCDII) and associated drug-resistant epilepsies. In preclinical models, SP7737 is delivered via an adeno-associated virus (AAV) vector targeted at cortical excitatory neurons. The peptide works by inhibiting mTOR hyperactivation, which in turn rescues neuronal cytomegaly, restores proper neuronal migration and cortical lamination, and suppresses clinical seizures. This approach aims to provide a more targeted and effective alternative to systemic mTOR inhibitors like rapamycin, which often have poor blood-brain barrier penetration and significant systemic side effects.