LogicBio Therapeutics Announces Research Collaboration with Daiichi Sankyo
"I am very pleased to announce this collaboration as a new milestone in the validation of our GeneRide platform. In 2020, we cleared our first Investigational New Drug (IND) application for GeneRide for the treatment of pediatric methylmalonic acidemia (MMA) patients and initiated the Phase 1/2 SUNRISE clinical trial. We also formed our first research agreement with another major pharmaceutical company to develop a drug candidate for Crigler-Najjar syndrome," said
The GeneRide platform harnesses a cell's natural DNA repair process leading to durable therapeutic protein expression levels. GeneRide supports the development of a new generation of genetic medicine able to insert a DNA segment into the human genome while potentially avoiding certain risks associated with other gene editing technologies. The therapy uses a viral vector to deliver the therapeutic gene, known as a transgene, to the nuclei of a patient's cells via one-time infusion.
LogicBio's two lead development programs based on the GeneRide platform are focused on rare pediatric conditions. The company is currently evaluating LB-001 in the Phase 1/2 SUNRISE trial in MMA and LB-301 in Crigler-Najjar syndrome through another research collaboration. LogicBio is also advancing the development of sAAVyTM, its next-generation AAV capsid platform, designed to overcome limitations with the current generation of AAV-based gene therapies.
About LogicBio
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