LogicBio Therapeutics Inc. has entered a research collaboration with Tokyo-based Takeda Pharmaceutical Co. Ltd. to further develop an experimental treatment for a rare genetic disease in children.
Cambridge, Mass.-based LogicBio's LB-301 uses the proprietary genome editing technology GeneRide to treat Crigler-Najjar syndrome, a rare pediatric disease caused by a deficiency in the liver‐specific UGT1A1 gene. The condition leads to severely high levels of unconjugated bilirubin, a substance found in bile, in the blood starting at birth.
The disease carries a lifelong risk of permanent neurological damage and death. Currently, treatment for the disease includes daily, intense phototherapy for about 12 hours, but this therapy becomes less effective with age, and liver transplantation becomes the only therapeutic option for survival.
LogicBio, which develops medicines to treat rare diseases in pediatric patients with significant unmet medical needs, and Takeda will work together to further research and develop LB-301. Takeda will provide funding for the research program.
Takeda will also have an option to negotiate an exclusive, worldwide license to LogicBio's LB-301 program.
Financial terms of the agreement were not disclosed.