The scientific biomedical field is encompassing a flourishing revolution with innovative techniques as massive sequencing capacity, gene editing and tissue engineering. It has fuelled the development of new unconventional drugs outside the consolidated target\lead compound\small molecule paradigm, named Advanced Therapy Medicinal Products (ATMPs) class.
The current panorama increasingly opens the doors to innovative drugs able to slow down or cure genetic disorders. Nevertheless, the advanced therapies cannot still be considered as a decisive opportunity to dramatically improve children’s life. Considering the high long-term impact on the quality of life, it is crucial that ATMPs are tailored to children’s development and made progressively available for the paediatric population.
In this scenario, EPTRI and RESTORE are collaborating to describe the map of competences available in the framework of the paediatric Advanced Therapies Medicinal Products research in pan-European countries in order to have the chance to promote the development of tailored paediatric advanced drugs and facilitate collaborations.
To this aim, a survey has been developed and it is available at this link.
The survey is addressed to professionals involved in the development of ATMPs and its compilation requires around 20 minutes.