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New Enzyme Delivery System for the treatment of Lysosomal Storage Diseases

Country of Origin: Spain
Reference Number: TOES20181107001
Publication Date: 20 November 2018

Summary

A Spanish university has developed a new enzyme delivery system  for the treatment of Lysosomal Storage Diseases. The system has exhibited a great capacity to immobilize and transport different enzymes used in enzyme replacement therapies (ERTs) reducing the dose of enzymes used in ERTs treatments. As a result, the stays at the hospitals and the side effects can also be reduced. The university is looking for license agreements with pharmaceutical companies interested in this technology.

Description

Lysosomal storage disorders (LSDs) are different metabolic disorders (over 70) designated as ‘orphan’, 'minority' or 'rare' diseases that affect a small number of people compared to the general population. In Europe, a disease is considered to be rare when it affects 1 per 2000 people. LSDs consist in born errors of metabolism caused by defects in genes that encode proteins involved in the lysosomal activity. At present, there are no known cures for lysosomal storage disorders, and most of them do not have specific treatments yet. However, progress is being made in the search for therapies, and there are specific treatments available for some of the lysosomal storage disorders that greatly improve the quality of life for those affected. 



Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. It's generally agreed that ERT products achieve varying degrees of benefit to patients suffering LSDs. Unfortunatly, the recombinant enzymes used in LSDs has shown some limitations and side effects. These enzymes show a high plasmatic clearance that, in consequence, requires a very frequent administration of high doses to reach therapeutic concentrations at the lysosomal level.



The Spanish university has developed a new system based on low toxic, biodegradable and biocompatible lipid systems that has exhibit a great capacity to immobilize different enzymes used in ERTs for the treatment of LSDs. The system has the capacity to target the enzyme to the lysosomes within cells of different tissues. In addition, the studies of the university have shown that these formulations can reach different organs and tissues including muscle, central nervous system and other target tissues for LSDs. This system can facilitate the administration of lower doses of the immobilized enzymes by intravenous bolus injection and as a result, the patients' long stays at the hospital can be avoided, the side effects reduced and their quality of life improved. 



The Spanish university is willing to reach license agreements with pharmaceutical companies that are dedicated to the manufacture and distribution of orphan drugs.

Advantages and Innovations

- All the components used for the preparation of this system are safe and are authorized for use in parenteral administration.

- The lipid components can be easily metabolized by lysosomal lipases. This fact facilitates and guarantees the release of the ERT enzyme at this level.

- The size and structure of the system are compatible with mechanisms of cellular uptake and cellular transport, responsible for its accumulation in lysosomes.

- The surface can be easily modified to modulate its biodistribution and promote its accumulation in tissues and target organs. 

- It can be obtained as a solid powder easy to handle and reconstitute, which has an advantage for its preparation, management and commercialization.

Stage Of Development

Field tested/evaluated

Requested partner

The Spanish university is willing to reach license agreements with pharmaceutical companies that are dedicated to the manufacture and distribution of orphan drugs. The companies will have the capacity to carry out the clinical trials and bring the product to market.