Dendrimeric pyrrolidine synthesis / medical use
The invention suggests a new approach to rare genetic diseases, orphan of a definitive cure and currently only symptomatically treated, by multivalent molecules having an interesting biological activity towards human enzymes involved in these pathologies. In particular the target are lysosomal storage disorders (LSDs), a group of rare metabolic diseases caused by deficiency of lysosomal enzymes.
The invention targets the development of an effective and non-invasive therapy for the treatment of two metabolic diseases, the Mucopolysaccaridosis IV (Morquio A) and the Mucopolysaccaridosis II (Hunter disease). Lysosomal storage disorders, though individually rare, involve more than 1 in 5000-8000 newborn globally. Such diseases are poorly known even from the competent Health Authorities and result orphan from both a treatment and a homogeneous regulation, helping for diagnosis, prevention and an input towards scientific research for the development of new approaches. To date only symptomatic approaches are available for these disorders. We here propose new multivalent iminosugars as pharmacological chaperones (PC) and stabilizers of the recombinant enzymes (ERT) as a new approach for the treatment of the two above mentioned diseases.
- Pharmacological Chaperones for Morquio A;
- Combined ERT/PC therapy for Morquio A;
- Combined ERT/PC therapy for Hunter disease.
- PCs based therapy has considerable advantages, such as: oral administration; rescue of the endogenous enzyme activity and low production costs;
- Dendrimeric iminosugars can be used in a combined ERT/PC therapy stabilizing the exogenous enzyme administered with the ERT.