Specific Challenge:

Advanced therapies are based on gene, cell or tissue-engineered products which are defined according to the terms of Regulation 1394/2007. So far, only a small number of these products have been placed on the market, and of these, most are for rare diseases. However, in recent years, important discoveries and developments, some unprecedented, have been made in molecular and cell biology and in cell technology, which offer improved opportunities for advanced therapies development. The challenge is to use the new knowledge and new technologies to introduce greater innovation into the advanced therapy development chain as a basis for tackling diseases and conditions affecting large patient groups.

Scope:

Building on European strengths and using the definition set out in Regulation (EC) 1394/2007^[1], projects should create knowledge, testing and exploitation platforms around innovative concepts for advanced therapy development. Platforms should comprise the components and expertise necessary to create a solid foundation on which to build possible new therapeutic approaches and/or aim to overcome particular development bottlenecks. Possible components could include studying the basic biology of the potential therapy and investigating its mode of action, proof of concept (in vitro, in animal models – where necessary - or first-in–man studies); safety, efficacy, characterisation, refinement and manufacturing of the product could be considered. Projects should also propose a business model for exploiting results and carry out appropriate outreach and public information activities. Examples of issues that have been identified as holding back the field include gene delivery to cells, reducing off-target effects in gene therapy, immunogenicity of potential new therapies, cell homing and tracking, lack of adequate pre-clinical models, or responding to regulatory concerns, such as potency assays, product characterization, or bank-to-bank variability (non-exhaustive list for illustrative purposes only). Sex and gender differences should be investigated, where relevant. Potential ethical issues should be addressed.

Expected Impact:

• Strengthened competitive position of European advanced therapy research and development.
• Improved perspectives for treating diseases and conditions in large patient groups with advanced therapies.
• Technological progress in the advanced therapy field.