Gene Therapy Centre for Rare Diseases

In the our project, we will focus on establishing a center with qualified staff and appropriate infrastructure, capable of fully carrying out the work under Phase I.

The ultimate goal of this and subsequent projects related to the construction of the Center will be to enable Warsaw to carry out the entire gene therapy process, from detection/diagnosis to clinical intervention. This will allow Warsaw to become a leading center for gene therapy in Poland and Europe, and above all, it will enable the development of state-of-the-art, life-saving therapies, especially for children.

The Center will specialize in developing gene therapies for rare and ultra-rare pediatric disorders, which—although they occur sporadically in individual patients—collectively currently affect as many as 10% of newborns worldwide. Worse still, 30% of these children do not live to see their fifth birthday. Therefore, rare and ultra-rare pediatric genetic disorders already represent—and will continue to represent in the future—a key challenge and a clinical priority of the highest order. Furthermore, due to their individual nature, diagnostic difficulties, and lack of treatment options, they lead to the complete exclusion of patients from the healthcare system. However, these disorders do not affect only children. They impact entire families. Children affected by these disorders often require round-the-clock care, which means that at least one parent must give up work. The heavy financial burden and stress often lead to the breakdown of family structures, which is associated with a very high divorce rate—a situation that further exacerbates the devastating impact of these disorders on other family members, including healthy siblings. Nor can we overlook the significant costs borne by the healthcare system, as many children require frequent medical procedures and hospitalizations.