KLTO-202

KLTO-202 is an investigational gene therapy being developed for the treatment of amyotrophic lateral sclerosis (ALS). The therapy is designed to deliver the gene that encodes the secreted alpha-Klotho protein (s-KL) using an adeno-associated virus (AAV) vector under the control of a muscle-specific promoter (desmin), targeting its expression to neuromuscular junctions and muscle and nerve cells. Its mechanism relies on increasing s-KL protein levels in these tissues, aiming to provide neuroprotective, anti-inflammatory, and antioxidant effects that can slow or prevent neuronal loss, preserve motor neuron function, delay disease progression, and prolong survival. KLTO-202 is administered intravenously and has received FDA Orphan Drug Designation. It is currently in preclinical development, with plans for Phase 1/2 trials in ALS. KLTO-202 was licensed from the Autonomous University of Barcelona, which initially developed the s-KL gene therapy technology.[1][2][3][4][5][6][7][8][9]

Other names

AAV8-desmin-soluble klothoAAV-8-desmin-soluble klothoAAV 8-desmin-soluble klothoAAV8-Des-sKLAAV-8-Des-sKLAAV 8-Des-sKL