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Novel therapeutics for MLD

Engineering approach for the treatment of metachromatic leukodystrophy
Technology Benefits
Novel approach for treating MLD
Demonstrated proof of concept: exceeds clearance seen in previous ERT trials by far
Lower amounts of enzyme need to be administered → to expect significantly fewer side effects compared to previous studies
Technology Application
metachromatic leukodystrophy treatment
Detailed Technology Description
The inventors have developed an engineering approach for the therapy of MLD: By exchanging three amino acids, the activity of the wildtype human ASA polypeptide could be increased approximately 5‑fold. When tested by intravenous enzyme replacement therapy (ERT) of MLD-mice, the triple mutant ASA reduced sulfatide storage in MLD mouse brain 3.4-fold more efficiently than wildtype ASA, resulting in a storage reduction of approximately 50%. Previous clinical studies have demonstrated the feasibility of enzyme compensation therapy for treating MLD.
Type of Cooperation
Application Date
Application No.
WO2018EP52790 20180205
- international:
A61K9/00; C12N15/10
- cooperative:
C12N9/16; C12Y301/06008; A61K9/009
Patent application
ID No.

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