Gene therapies

In recent years, gene therapy has shown a remarkable potential to offer definitive treatments for otherwise incurable diseases. By the end of 2017, seven gene therapy products have reached the market and many more are entering clinical testing. These recent successes are largely attributable to incremental step-wise improvements in the efficiency and safety of delivery tools, the most advanced of which are still represented by viral vectors.

However, there are yet unsolved hurdles associated with these approaches, such as their limited precision on genome and tissue targeting, imperfect ability to recapitulate physiological control of transgene expression, and uncertainties whether the turnover of the targeted tissue/cells will sustain life-long disease correction. Because of these reasons, the field is questing for radical technological advances that overcome these limitations.

UPGRADE gathers together key opinion leaders and top-level EU scientists who have already provided seminal contributions to the gene therapy field, including inventing new technological platforms for gene delivery, genetic and epigenetic editing, and launching several first-of-its-kind in the world clinical trials of new AMPs, many of which have been successful leading to the registration of the first gene therapy drugs on the EU market.

The ambitious research and technological chain set in place by UPGRADE will generate groundbreaking knowhow and innovative gene therapy platforms. As many UPGRADE scientists have long been engaged in collaboration with pharma industry for the development and commercialization of new AMPs, it is expected that this virtuous model will also apply to valorise the output of UPGRADE towards fostering the competitiveness of EU pharmaceutical companies in the field, while guaranteeing the highest level of health protection for patients.