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USE OF PHARMACOLOGICAL CHAPERONES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES
US2021179575
The present invention relates to the use of galactose analogues of formula (I) with the capacity to stabilize the structure of the #-GalA enzyme, for the treatment of lysosomal storage diseases and, in a preferred embodiment, for the treatment of Fabry disease. In addition, the present invention relates to pharmacological compositions having an effective amount of at least one of the galactose analogues described in the present document for the treatment of lysosomal storage diseases and, in a preferred embodiment, for the treatment of Fabry disease. The European Patent is granted and validated in UK, France, Germany and Italy
The galactose analogues of our formula (I) have several advantages over the conventional treatments for the lysosomal storage diseases: <p> - They have a homogeneous distribution: Being small molecules, they are capable of crossing biological membranes, which might include blood-brain barrier. Whilst, the recombinant α-GalA used in in ERT therapies does not reach the tissues that do not express the receptor Mannosium-6-phosphate. <p> - The galactose analogues of the formula (I) increase the therapeutic spectrum of patients who can use oral treatment as they have the ability to bind to the mutated α-GalA enzymes responsible for Fabry disease, which cannot be treated with the pharmacological chaperones currently in use. <p> - Production costs and contamination risks are much lower than those in the production of the biological drugs required for enzyme replacement therapy.