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Name

Recombinant antibody for easy detection of mucolipidosis type II and III and selective purification of proteins with mannose 6-phosphate residues for ERT

Organization name

Ascenion GmbH

Profile

Challenge

Currently, the diagnosis of ML II and III can only be carried out in a few specialized laboratories throughout the world due to a complex procedure. In addition, purification of enzymes for ERT requires expensive chromatographic procedures which do not discriminate between therapeutically active and inactive forms, i.e. with or without Man6P modification.

Technology

The inventors have generated a recombinant antibody (M6P-1) specifically binding to Man6P. They have shown on a laboratory scale that it allows

  • easy diagnosis of ML II and III by western blots
  • selective purification in a one-step procedure of recombinant high-affinity uptake forms of lysosomal enzymes on affinity media (see figure)
  • histological staining of lysosomes.

The protein expresses well in a prokaryotic host, can be purified to homogeneity, is stable over several months at 4 °C and can be immobilized on affinity media without loss of activity.

Commercial Opportunity

The technology is offered for co-development of a diagnostic kit and an optimized cost-efficient purification procedure or for in-licensing.

Patent Situation

A European patent application has been filed in August 2008 and a PCT-application in August 2009.

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