Lysosomal storage disorders (LSDs) are inherited rare diseases caused by the lack or malfunction of proteins involved in lysosomal activity. Enzyme replacement therapies (ERTs, based on the administration of a functional version of the defective enzyme) have gained clinical relevance in LSD health care. Therefore, production of therapeutic enzymes is a key aspect in the development of therapies for many rare diseases.
A large variety of nanoparticle-based delivery systems have become increasingly important for therapeutic applications. A critical issue lies in the challenge of scaling-up the synthesis and formulation of the nanomaterial from the lab to pilot and then to the industrial scale, while maintaining control over their quality attributes, such as particle mean size . The DELOS Platform is a robust procedure to overcome these challenges, delivering consistent, high quality, therapeutic nanoformulations. In the frame of Smart-4-Fabry, the DELOS procedure has been successfully scaled-up to the pilot plant and consistent, high-quality nanoliposomes loaded with GLA enzyme have been delivered for preclinical GLP toxicology assays.
Within the development and innovation process, clarity about the safety surrounding new technologies is one of the most important conditions for acceptance of the technology. Particularly for nanomaterials, safety is an essential point of attention due to the uncertain risks.
Discover the similarities between the molecualr self-assembly and LegoTM bricks with this newsletter.
SMART4FABRY project deals with the development of a novel drug formulation for Fabry disease. A strict development pathway has to be followed for any medical innovation, moreover for nanomedicines.
Rare diseases, like Fabry disease, mean a major health problem for a significant number of people in total, but affecting small amount of patients for each individual diseases.