Novel therapeutics for MLD
Engineering approach for the treatment of metachromatic leukodystrophy
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
metachromatic leukodystrophy treatment
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.
Licensing
05.02.2018
WO2018EP52790 20180205
- international:
A61K9/00; C12N15/10
- cooperative:
C12N9/16; C12Y301/06008; A61K9/009
Patent application
4783
德国
