LogicBio Therapeutics, Inc. (NASDAQ:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients today announced a research collaboration with Takeda Pharmaceutical Company Limited (Takeda) to further develop LB-301, an investigational therapy using LogicBio’s proprietary, promoterless, nuclease-free genome editing technology, GeneRide™, for the treatment of Crigler-Najjar syndrome. LB-301 is a recombinant adeno-associated viral (AAV) vector with a uridine disphosphate-glucuronosyltransferase-1 (UGT1A1) gene. The collaboration will bring together LogicBio’s propriety platform for genome editing and Takeda’s expertise in researching and developing gene therapies.
“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.”
“We are thrilled to be working with Takeda to advance our GeneRide™ platform in a second indication,” said Fred Chereau, CEO of LogicBio. “Their insights and expertise in rare diseases drug development is expected to significantly accelerate the development of a much-needed therapy for this devastating pediatric disease. This collaboration recognizes GeneRide™ as a promising approach for bringing the transformational power of genome editing to children with an array of relentless, progressive pediatric diseases.”
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.
LogicBio has demonstrated that a murine GeneRide™ construct of LB-301 can correct the gene deficiency in an animal model of Crigler-Najjar syndrome. The introduction of a UGT1A1 gene into the albumin locus in mouse liver cells resulted in normalization of bilirubin levels and long-term survival of mice deficient in UGT1A1 from fewer than 20 days to at least one year. The results from this research were published in EMBO Molecular Medicine (Porro et al., 2017).