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Novel therapeutics for MLD

Summary

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

Licensing

Application Date

05.02.2018

Application No.

WO2018EP52790 20180205

Classes

- international:
A61K9/00; C12N15/10
- cooperative:
C12N9/16; C12Y301/06008; A61K9/009

Others

Patent application

ID No.

4783

Country/Region

Germany

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