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Update from Mucolipidosis Collaborative Network (MCRN) and Cure Mucolipidosis (Cure ML) on Mucolipidosis Type II
The Mucolipidosis Collaborative Network (MCRN) and Cure Mucolipidosis (Cure ML) announce that the breeding of the mucolipidosis type II (ML II) cat colony is progressing and will now enable us to start the Natural History studies on this model which will begin in earnest in 2022. We anticipate the ability to begin testing different modes of therapy shortly after the Natural History Study is completed.
MCRN and Cure Mucolipidosis representatives met with senior leadership at M6P Therapeutics last week and were very encouraged by the information they received. “We were very pleased to hear that the US Food and Drug Administration (FDA) has granted rare paediatric disease designation (RPDD) for both ML II and ML III,” said Jenny Noble, vice president of research at Cure Mucolipidosis. “In addition, the FDA has granted M6P Therapeutics orphan drug designation (ODDs) for its gene therapy program for mucolipidosis II and III.”
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