Rare Daily Staff

LogicBio Therapeutics said it entered into a research collaboration with Takeda Pharmaceutical to advance LB-301, an experimental therapy using LogicBio’s proprietary genome editing technology for the treatment of Crigler-Najjar syndrome.

The collaboration will bring together LogicBio’s propriety platform for genome editing and Takeda’s expertise in researching and developing gene therapies.

Under the agreement, LogicBio and Takeda will collaborate to further research and develop LB-301.  Takeda will provide funding for the research program under the collaboration agreement and will have an exclusive option to negotiate an exclusive, worldwide license to LogicBio’s LB-301 program.

Crigler‐Najjar syndrome is a rare monogenic pediatric disease caused by a deficiency in the liver‐specific UGT1A1 gene, resulting in severely high levels of unconjugated bilirubin in the blood starting at birth, with lifelong risk of permanent neurological damage and death. Current clinical practice consists of daily, intense phototherapy treatment for approximately 12 hours, but this treatment becomes less effective with age, ultimately leaving liver transplantation as the only therapeutic option for survival.

LB-301 is a recombinant adeno-associated viral (AAV) vector with a uridine disphosphate-glucuronosyltransferase-1 (UGT1A1) gene. It is based on the company’s GeneRide technology, which enables site-specific integration and lifelong expression of therapeutic transgenes, without the use of exogenous promoters or nucleases. LogicBio has demonstrated preclinical proof-of-concept of GeneRide in multiple animal models of the disease, improving survival and reversing disease pathology.

“LogicBio’s innovative, site-specific, genome editing platform has the potential to overcome the limitations that make it challenging to apply conventional gene editing and gene transfer in pediatric patients,” said Dan Curran, Head, Rare Diseases Therapeutic Area Unit at Takeda. “We see GeneRide as a promising approach to explore as part of our aspiration to develop transformative – or even potentially curative – therapies to patients living with rare diseases.”

Photo: Dan Curran, Head, Rare Diseases Therapeutic Area Unit at Takeda

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