CERE-130 (Ceregene)

CERE-130 is an experimental gene therapy developed by Ceregene for the treatment of amyotrophic lateral sclerosis (ALS). It utilizes an adeno-associated virus serotype 2 (AAV2) vector to deliver the human cDNA for insulin-like growth factor 1 (IGF-1) under the control of a chicken beta-actin (CAG) promoter. The therapy is designed to provide sustained, local production of IGF-1 in the central nervous system, specifically targeting motor neurons. IGF-1 is a neurotrophic factor that enhances motor neuron function and provides protective support against the degeneration characteristic of ALS. Preclinical studies in SOD1G93A mouse models demonstrated that intraspinal delivery of CERE-130 resulted in robust and long-term IGF-1 expression, partial rescue of lumbar spinal cord motor neurons, delayed disease onset, and increased animal survival.

Other names

AAV-IGF-1AAV-IGF1AAV-IGF 1CERE130CERE-130CERE 130