Lysosomal Storage Disorders (LSDs) are progresive genetic disorders, that affect different systems of the organism by deficiency or total lack of some enzyme in the lysosome. This set of diseases affect 1 out of 5000 live births. LysoCure, a Pronacera project, aims to provide better attetion and a higher quality of life to these patients through a specific treatment.
Patients with LSDS suffer from an excessive accumulation of macromolecules in the lysosomes, that are normally degaded by the enzymes that are in complete or total deficiency. The excess and deposition of these molecules have a toxic effect on tissues and organs.
As a result of the shortage of effective treatments the affected people usually suffer theis first symptoms in childhood or adolescence, shotening their life expectancy and provoking variable grades of disability
Pronacera, with the LysoCure line has develoed an oral treatment for lysosomal diseases thorough the use of pharmacological chaperonas for GM! and Morquio type B diseases and having in perspective other such importan ailments as Gaucher and Parkinsin diseases.
We have also developed screening platforms with product libraries that can be used for LSDs, contrubuting with personalized recommendations of an appropriate treatments ar "in vitro" level.
LysoCure has a great competitive advantage which lies in the development of oral drugs (using the basic research of research groups) for orphan diseases, which lack treatment and are also highly disabling. This type compounds are able to cross the blood-brain barrier.
On the other hand, drug screening platforms will help pharmaceutical companies to have a new tootl to evaluate the success of new treatments or the repositioning of treatments approved by EMA and FDA.
Pronacera has implemented a hybrid business model in which besides the line LysoCure with the development of neurodegenerative screening platforms, it obtains return with the distribution of genetic kits for assisted reproduction, gynecology, fertility, cancer, cardiology and human health.
- The project aims to help all patients worldwide suffering from GM1 and Morquio type B.
- LysoCure focuses on diseases whose prevalence is very low but with very disabling conditions, helping both patients and their caregivers improving tremendously their quality of life.
José A Sánchez Alcázar - Founder & Scientific director
David Cotán Marín - Founder & CTO
David Cotán Marín - Project Manager