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EPTRI is member of TRANSFORM

The scientific biomedical field is encompassing a flourishing revolution with innovative techniques as massive sequencing capacity, gene editing and tissue engineering which has stimulated the development of new drugs outside chemical paradigm as the advanced therapies (ATMPs).

The ATMPs represents an opportunity for inherited disorders, occurring in about 2%-5% of all live births and accounting for up to 30% of paediatric hospital admissions, of a lifelong cure and a highly positive impact on the quality of life.

In this scenario, EPTRI is working to provide all the expertise and the technologies needed to support the paediatric research for discovery and development of innovative paediatric medicines as the ATMPs.

In this respect, EPTRI has become a member of the European Alliance for Transformative Therapies (TRANSFORM), which is a multi-stakeholder Alliance endorsed by Members of the European Parliament and policy-makers, that connects patient groups, medical experts and associations, scientists, researchers, industry actors, networks and other relevant stakeholders.

The aim of the Alliance is to foster dialogue and provide evidence-based policy recommendations to enable safe and timely patient access to cell and gene therapies, whilst ensuring sustainability of healthcare systems. Within the Alliance, EPTRI strengthened the paediatric point of view contributing to the Position Paper that will be released in the next weeks.

In addition, EPTRI will participate as speaker at the TRANSFORM virtual meeting “Realising the Potential of Transformative Therapies. A European Pharmaceutical Strategy that Enables Safe and Timely Patient Access to Cell and Gene Therapies” on 30 June 2021 hosted by TRANSFORM and TRANSFORM MEP Interest Group.


The event will be the occasion for the launch of its Recommendations for Action for Cell and Gene Therapies in the context of the European Pharmaceutical Strategy which have been developed collaboratively with expert stakeholders.


More information on the event is available at this link.