RE(ACT)x Switzerland: We Care for Rare

On 10 November 2016, the “RE(ACT)x Switzerland: We Care for Rare” brought together a group of motivated researchers and patients advocates to discuss about the creation of a Swiss Center for Therapeutics Discovery (SCTD) focused on rare diseases (RD).  The symposium organized by the BLACKSWAN Foundation on behalf of „Medicine Discovery & Delivery“ Health2030 workgroup, will be probably remembered as the turning point for translational research on rare diseases in Switzerland and in Europe. The event was organized at Campus Biotech in Geneva under the patronage of ProRaris, the Swiss alliance of rare disease patients.

The objectives of the day were:

  • to understand the unmet medical need in rare diseases, listening to patients voices and researchers working in that domain and learn from successful projects that made it from the bench to the bedside,
  • to solidify the Swiss society and the Swiss scientific community around a common project for the creation of a Swiss Center for Therapeutic Discovery focused on rare diseases.

Press release


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09:00-09:30 Registrations

09:30-09:45 Welcome message:

Pr. Patrick Aebischer (EPFL) and Dr. Olivier Menzel (BLACKSWAN Foundation)

Introduction: Anne-Françoise Auberson (President de ProRaris, Swiss Rare Disease Alliance)

09:45-10:15 Patient’s view: 

Mrs. Sharon S. Lagas, Board President, Alport Syndrome Foundation; USA: „The need to accelerate development of new treatments for rare diseases illustrated through a family’s journey with Alport Syndrome“

10:15-10:45 Researchers’ view: 

Dr. Olivier Dorchies (UNIGE), CH: „Pre-clinical evaluation of disease modifiers : Example of nutraceuticals and repurposed drugs for severe myopathies“

Pr. Gisou van der Goot (EPFL), CH: „Hyaline Fibromatosis Syndrome: in depth mechanistic understanding“

10:45-11:15 Institution’s view: 

Pr. Kay Davies (Director of the MRC functional genetics unit, governor of the Wellcome Trust and director of the Oxford Centre for Gene Function); UK : „Rare Disease Consortium at Oxford: from patient to drug screens to therapy“

11:15-11:45 Break


Dr. Rashmi Gopal-Srivastava (Director Extramural Research Program, Office of Rare Diseases Research, National Center for Advancing Translational Sciences, NCATS at the National Institutes of Health, NIH); USA: „Rare Diseases Clinical Research Network (RDCRN) Program: A Model for Collaboration with Patient Advocacy Groups & Research Resources/Initiatives at NCATS, NIH”

12:15-12:45 Pharma success story: 

Dr. Rudolf Hausmann (VP Technical Development & Operations at  Santhera), CH: „Learnings from challenges in orphan drug development: The Santhera story“

12:45-14:00 Lunch

14:15-14:45 Therapeutic discovery center in other countries: the experience, the sustainability: 

Dr. Michael Foley (Director of Tri-Institutional Therapeutics Discovery Institute), USA

14:45-16:30 Round table

Presentation of the Swiss Center for Therapeutic Discovery, Pr. Leonardo Scapozza; CH

Round table with committed people and the audience (moderators Dr. Prunotto and Pr. Scapozza on behalf of the Medicine Discovery and Delivery Health2030 group)


Conclusion remarks

16.45 Networking and farewell cocktail

Speakers' presentations


* The proposed “Swiss Centre for Therapeutics Discovery” (SCTD) would ideally be an Open Innovation no-profit public-private academic foundation dedicated to translational science located in the Campus Biotech in Geneva.

The SCTD mission is to foster novel therapeutics discovery & development (drugs – small molecules, antibodies, peptides or gene therapy – or diagnostic tests) to capitalize the available structures for drug discovery and to translate the potential of the basic science performed by the Swiss research centres into treatments for patients. The SCTD would ideally allow capitalizing the incredible knowledge that sequencing information will unveil. The mission will be achieved in partnership with participating/collaborating clinical centres up to proof of concept (PoC) level.

The strategic focus of the Swiss Centre for Therapeutics discovery will be on rare and orphan diseases. Any basic scientist, working in Switzerland, Europe or in any part of the world will be eligible to submit proposals to the SCTD. Scientists joining the SCTD as fellows will keep the scientific ownership of their projects contributing expertise to SCTD.

Fondation Suisse pour la Recherche sur les Maladies Orphelines