LogicBio Enters Gene-Editing Collaborations with CANBridge and Daiichi Sankyo

Rare Daily Staff

LogicBio Therapeutics announced two collaborations and option agreements that leverage its GeneRide gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood.

Under the terms of the agreement with Chinese pharmaceutical CANbridge, LogicBio granted an option to an exclusive license for LB-001, an experimental therapy for the potential treatment of methylmalonic acidemia (MMA) in Greater China (China, Taiwan, Hong Kong and Macau).

“Partnering with LogicBio is a vital part of our strategy to build a world class rare disease program,” said James Xue, founder, chairman and CEO of CANbridge Pharmaceuticals.

CANbridge would assume responsibility and costs for all future development, regulatory, commercial, and potentially manufacturing activities in the territory upon exercise of the option. The agreement also grants CANbridge a worldwide license for the adeno-associated virus (AAV) sL65, the first capsid produced from LogicBio’s sAAVy platform, and development support on gene therapy candidates for the treatment of Fabry and Pompe disease plus options on two additional undisclosed gene therapy indications.

LogicBio is eligible to receive an upfront payment of $10 million. The agreement also includes options payments, clinical, regulatory, and commercial milestone payments of up to $581 million and up to double-digit royalties on net sales.

“Together both of our companies aim to develop and commercialize rare disease treatments, which are desperately needed in China and the rest of the world. This new partnership positions us well to reach patients who often have no or limited treatment options available including patients affected by MMA,” said Daniel Gruskin, senior vice president and head of clinical development at LogicBio.

LogicBio Therapeutics also entered into a research collaboration and exclusive option agreement with Japanese pharmaceutical Daiichi Sankyo for a gene editing program based on LogicBio’s proprietary GeneRide platform.

Under the terms of their agreement, the companies will collaborate on the development of treatments for two undisclosed indications leveraging GeneRide. The agreement also grants Daiichi Sankyo an option to negotiate and acquire worldwide licenses for LogicBio’s development programs in these two indications. Financial terms of the collaboration are not disclosed.

“This collaboration is a new milestone in the validation of our GeneRide platform,” said Frederic Chereau, president and CEO of LogicBio. “By partnering with Daiichi Sankyo, a global leader in developing innovative medicines, we are reinforcing our commitment to bring innovative therapeutics to people living with serious genetic conditions including many that can present symptoms as early as infancy.”

LogicBio’s gene delivery and gene editing platforms address rare and serious diseases from infancy through adulthood. The company says Its proprietary GeneRide platform is a new approach to precise gene insertion that harnesses a cell’s natural DNA repair process leading to durable therapeutic protein expression levels while potentially avoiding certain risks associated with other gene editing methods such as CRISPR. The company’s capsid development platform is designed to support development of treatments in a broad range of indications and tissues.

In 2020, the company cleared its first Investigational New Drug application for GeneRide for the treatment of pediatric methylmalonic acidemia (MMA) patients and initiated the phase 1/2 SUNRISE clinical trial. LogicBio also signed its first research agreement with another major pharmaceutical company to develop a drug candidate for Crigler-Najjar syndrome.

Photo: Frederic Chereau, president and CEO of LogicBio