GenEngNews: UK-based Oxyrane initiates ERT therapy for Pompe Disease

Enzyme replacement therapies (ERT) firm Oxyrane raised $26.5 million in a series D round of financing. Participants in the fundraising round included Forbion Capital Partners, Morningside Groups, and existing investor New Science Ventures. Oxyrane said it will use the funds to progress its lead candidate through the completion of preclinical development to the end of Phase I/II trials, and also to continue preclinical development of other ERTs. Pompe disease is a potentially fatal lysosomal storage disease caused by alpha-glucosidase enzyme deficiency, which leads to glycogen accumulation and nerve cell damage.

U.K.-based Oxyrane is exploiting a Yarrowia lipolytica yeast-based glycoengineering platform to develop novel and biosuperior versions of ERTs for lysosomal storage diseases. The firm claims that in comparison with current commercial processes, its yeast platform enables the production of human lysosomal enzymes with 3–15 times the amount of mannose 6-phosphate, the targeting sugar that facilitates effective enzyme uptake and localization.

This increase in the levels of targeting results in an over 20-fold improvement in cellular uptake without altering the naturally occurring protein enzyme, Oxyrane says. The firm has already been carrying out 1,000 liter production using a simple batch process, and believes it can scale up manufacturing to 100,000 liters.