RARE DISEASES

Life Sciences: Rare Diseases, New Approaches, 2009–2014

The research programme «Rare Diseases – New Approaches» started in 2009 and invested CHF 2 mio. p.a. There was a yearly call open to researchers at Swiss universities, university hospitals, federal institutes of technology, research institutions affiliated with Swiss universities and universities of applied sciences. Calls were launched in early spring, application deadline was in summer, decisions are taken in autumn. The 2014 call was the sixth and last call of the programme.

Goals: Objective of the Programme

The goal of the initiative «Rare Diseases – New Approaches» was to improve the diagnosis and treatment of rare genetic diseases. The programme was aimed at bridging the worlds of basic and clinical research: projects with new approaches or technologies were supported that focused on a clinical or diagnostic application. The project results had to lead to a better understanding of the genetic, molecular and biochemical processes underlying these diseases and to pave the way towards new diagnostics and new forms of treatment. The ideal project scenario was to show concrete compounds and diagnostic tests from which patients suffering from a rare disease could benefit.

The focus had to be on innovation, feasibility and effectiveness, while attaining high scientific and technological standards. The call was directed at researchers developing and implementing innovative approaches or technologies to address currently unresolved needs.

Results: What was achieved

With the goal of developing diagnostics and drugs for treating rare diseases, in 2009 Gebert Rüf Stiftung began funding research in this often-neglected area. Since then, six annual calls for project submission have been made. Swiss research groups have prepared and submitted some 332 proposals to our foundation; of these, 31 have been accepted with a budget of total CHF 12.3 mio. Almost half of these endeavours have achieved a diagnostic or therapeutic success. In order to promote the development and the transfer to market of these promising projects, a collaboration has been launched with the newly formed biotech company, Geneva Biotech Center. With the help of investors, potential drugs and therapies reach market maturity earlier than they would otherwise.

These past years have not been dormant: Significant successes have been achieved at many levels, and progress continues to evolve. Indeed, the development of a long-overdue National Rare Disease Policy has been launched by the government. The Swiss National Science Foundation joined the E-Rare network with it’s transnational call for funding multinational research projects in the field of “Innovative Therapeutic Approaches for Rare Diseases”. The umbrella organisation, ProRaris, was founded in 2010.

And, last but not least, one sees a clear trend of establishing centres of excellence in Switzerland. For example, the Rare Disease Initiative Zurich (RaDIZ), whose aims are to improve care and treatment of patients with rare diseases and, simultaneously, position Zurich as a leading European research hub for rare diseases.

Successor Programme: Microbials

With its initiative, Gebert Rüf Stiftung seeks to generate a significant impact by exploiting the potential and widespread use of microbial resources in health and technology.

Microbials – Direct Use of Micro-Organisms

Approved projects Rare Diseases