New Dendrimeric Pyrrolidines

Technology Overview

The invention proposes a new approach to rare genetic diseases for which no cures are available, only symptomatic treatments through the preparation of novel multivalent molecules active toward the human enzymes responsible for them. Lysosomal storage diseases (LSDs) are a group of rare metabolic diseases caused by deficits in lysosomal enzymes to date orphaned by treatments

The invention is aimed at developing an effective, noninvasive therapy for the treatment of two metabolic diseases, Mucopolysaccharidosis IV (also known as Morquio A) and Mucopolysaccharidosis II (also known as Hunter’s disease). Lysosomal storage diseases, a group of inherited metabolic disorders, are rare when taken individually, but are substantially numerous when taken as a whole since about one in 5000-8000 infants is affected. These diseases are “orphaned” by treatments that provide a definitive cure, but are treated only symptomatically. These molecules are proposed as pharmacological chaperones (PCs), molecules capable of restoring deficient enzyme activity, or as stabilizers for the current marketed treatment, enzyme replacement therapy (ERT), which has various problems including poor stability of the exogenous enzyme.

Stage of Development

The TRL of the invention is 3.

Benefits

Pharmacological chaperone (PC)-based therapies have multiple advantages including, the possibility of oral administration, restoration of endogenous enzyme activity, and low cost of therapy; in addition, dendrimeric immino sugars can also be used in combined ERT/PC therapy as recombinant enzyme stabilizers.

Applications

The patented technology has been tested in the following case studies:

1. Pharmacological chaperones for Morquio A;
2. Combined ERT/PC therapy for Morquio A;
3. Combined ERT/PC therapy for Hunter’s disease.

Opportunity

The patent is available for outright assignment, as well as for exclusive and non-exclusive licensing. Licenses are available for the remaining term of the patent titles.

The Research Group is available for new collaborative and third-party research activities, in-depth technical investigations, scientific advice, also aimed at raising the TRL of the technology.