Oxyrane's work to enhance enzyme performance is disease-specific, but there are some common themes to the types of improvements.
Oxyrane aims to:
Enhance protein activity
A clipping enzyme has a limited time to degrade the undesired, accumulating product. The amount of this undesired product which can be removed is linked to: 1) how good the enzyme is at its job and 2) how long the enzyme is able to do its job before it is naturally degraded. Through engineering it may be possible to enhance an ERT on one if not both of these dimensions. Oxyrane's lead Pompe candidate OXY2810 is an example of improving how good an enzyme is at its job.
Reduce immunogenic response
Although the ERT product is a naturally occurring human product, individuals impacted by lysosomal storage diseases may have had little or no exposure due to the nature of the diseases. As such, it is common that individuals may have some immunological response to ERT products. Doctors that treat lysomal storage diseases are familiar with this challenge and can often effectively manage these responses; however, Oxyrane aims to reduce this situation where possible.
Skew drug distribution to the most impacted areas
M6P-based product targeting is very effective at helping product reach lysosomes throughout the body, but different tissues are more or less important depending on the specific lysosomal storage disease. It would be greatly desirable to direct more ERT product to the most impacted tissues/organs. Oxyrane is exploring a range of solutions to achieve this.