The paper “Ensuring a future for gene therapy for rare diseases” by Alessandro Aiuti, Francesca Pasinelli, and Luigi Naldini has been recently published in the Nature Medicine journal.  

In this paper, authors discuss the advantages and disadvantages of autologous hematopoietic stem and progenitor cell gene therapy (HSPC-GT) as treatment for several inherited rare diseases (RDs), including primary immunodeficiencies.  

Authors focus on the elevated costs associated with these therapies due to their complex development and production processes, leading to disinvestment on several gene therapy programs by pharmaceutical companies, despite demand from patients and their families.  

This will lead to a substantial unmet clinical need for these patients.  

Authors conclude that “these treatments should eventually be made available to all who need them, or we risk a future where they are only available in a few rich countries or through private clinics. Additional public investment in the manufacturing of gene therapies for ultra-rare diseases and administration under a nonprofit scheme could allow lower regulatory and production costs, without impacting safety.” 

The full manuscript is available here.