CANbridge Acquires Rights to Gene Therapy Candidates

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by Margarida Maia |

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Gene therapy

Gene therapy

CANbridge Pharmaceuticals has entered a strategic collaboration and license agreement to gain global rights to LogicBio Therapeutics’ gene therapy candidates for rare diseases with limited treatment options.

Under the agreement, CANbridge now owns worldwide rights to develop, manufacture, and commercialize gene therapy candidates for Fabry disease and Pompe disease, as well as the option to advance two additional gene therapy programs for undisclosed indications.

LogicBio also granted CANbridge the option to license a gene-editing technology, LB-001, for methylmalonic acidemia — a genetic disorder in which the body is unable to break down certain proteins and fats — in Greater China (China, Taiwan, Hong Kong, and Macau).

“This important agreement advances our three-prong strategy to build CANbridge’s next-generation rare disease treatment pipeline: in-house research capability, as supported by the recent opening of our CANbridge rare disease research facility, in Massachusetts; collaborative academic research agreements, such as the two we have with the Horae Gene Therapy Center, at the University of Massachusetts Medical School; and partnerships with innovative biotechnology companies,” James Xue, PhD, founder, chairman, and CEO of CANbridge, said in a press release.

CANbridge is a biopharmaceutical company focused on therapeutic strategies, including gene therapy, for rare diseases with unmet medical needs.

In genetic disorders caused by a defective gene, gene therapy works by inserting a properly functioning gene inside cells via a carrier. The carrier usually is a virus that has been altered so it is no longer harmful to the body.

Using its proprietary sAAVy platform technology, LogicBio developed the adeno-associated virus (AAV) sL65 — a synthetic protein cage that mimics the shell of a virus — to deliver gene therapies directly to cells in the liver.

The liver is a central organ for metabolism in the body and is a significant source of globotriaosylceramide (Gb3), which is the fatty molecule that builds up to toxic levels in Fabry disease as a result of mutations in the GLA gene.

AAV sL65 may have the potential to overcome limited potency and undesirable immune responses, compared with other liver-targeting viral vectors, the company reported. It also may be easier to produce in large amounts, making it a valuable addition to CANbridge’s gene therapy programs for Fabry disease and Pompe disease.

The two additional, undisclosed gene therapy programs also are based on AAV sL65.

LB-001 is an investigational therapy whose development has been based on the GeneRide platform. LogicBio’s proprietary GeneRide platform makes use of the cell’s natural repair process to insert a healthy copy of a defective gene at its precise location.

This gene-editing technology is thought to provide a means to obtain immediate and durable gene expression in the modified cells. This means that shortly after treatment, the modified cells begin making the protein to treat the disease — and may do so for a long time.

“LogicBio’s cutting-edge capsid and gene editing technologies, which have the potential to address the limitations of existing therapies, supplies a vital component to the CANbridge long-term rare disease capability,” Xue said.

The agreement includes an upfront payment of $10 million in addition to up to $581 million in options as well as clinical, regulatory, and commercial milestones, and up to double-digit royalties on net sales.