M6P Therapeutics receives six rare paediatric disease designations from the U.S. FDA for company’s deep pipeline of programs for lysosomal storage disorders

M6P Therapeutics

28 January 2021 - U.S. FDA also grants two orphan drug designations for the company’s gene therapy programs for Gaucher disease and mucolipidosis.

M6P Therapeutics today announced that the U.S. FDA granted six rare paediatric disease designations for various programs within its development pipeline for LSDs, including four recombinant enzyme and two gene therapy programs. 

In addition, the FDA granted two orphan drug designations for its gene therapy programs for Gaucher disease and mucolipidosis.

Read M6P Therapeutics press release

Michael Wonder

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Michael Wonder