Dorphan SA



Phone021 693 8550

PSE, EPFL site

PSE-D - 3rd floor

1015 Lausanne


Responsible Mr.   Dr Stéphane Demotz

Activity domain

DORPHAN S.A. is a Swiss biopharmaceutical company whose mission is the development of innovative therapeutic solutions for rare diseases. The company operates by identifying, acquiring and further developing promising early stage research programs.

Keywords : Orphan drug, Mucopolysaccharidosis, Lysosomale storage diseases, genetic disorders, Preclinical drug development, Molecular chaperones, rare neurodegenerative infantile diseases

Dorphan SA is a spin-off of Swiss Sanfilippo Foundation



DORPHAN S.A. is a Swiss biopharmaceutical company whose mission is the development of innovative therapeutic solutions for orphan and rare genetic diseases affecting mainly children. The company operates by identifying, acquiring and further developing promising early stage research programs.

The main objective of DORPHAN is the constitution of a portfolio of drug candidates for the treatment of orphan and rare genetic diseases, followed by the development of these molecules up to a deal point for out-licensing to large pharmaceutical companies. These companies would then pursue and complete the drug candidate clinical evaluation as well as commercialization.

Business Model

Our business model exploits the fact that more than 8000 rare genetic diseases have been identified thus far, with only a few having effective treatments. Today, the development and marketing of drugs for rare diseases are mainly conducted by biotechnology companies and only recently to a limited extent by large pharmaceutical groups. This trend is nevertheless changing. Major players of the pharmaceutical sector have recently announced initiatives aiming at participation in the therapeutic area of rare diseases.

DORPHAN identifies promising projects by conducting searches in the scientific literature and patent databases for compounds developed by academic institutions and pharmaceutical companies for other pathologies. The hypothesis is that these substances are therapeutically active in the rare genetic diseases selected by DORPHAN. This strategy is complemented by scouting for selected research activities in academic institutions and by exploiting our network of contacts. This approach critically relies on a deep understanding of the biology of the diseases for which treatments are sought. Once promising novel molecules have been identified, negotiations for in-licensing or partnership to conduct further pre-clinical development are undertaken.


Three projects initially conducted with the financial support of the Swiss Sanfilippo Foundation have been transferred to DORPHAN for further preclinical development. These three projects consist in the development of families of molecules for the treatment of Sanfilippo disease (mucopolysaccharidosis of type IIIB), Morquio disease (mucopolysaccharidosis of type IVB) and Sly disease (mucopolysaccharidosis of type VII). This work was started and run for a couple of years in French academic institutions, the Pierre and Marie Curie University in Paris, the University of Poitiers and the University of Orléans. The development of these compounds is still currently conducted in these three institutions, and now also in Swiss universities (University of Lausanne and University of Geneva) and by private service providers in Switzerland and abroad.

A program involving research groups from several Swiss academic institutions (EPFL and HES-Sion) is being assembled. Its objective is the screening of various compound libraries for the identification of drug candidates aimed at the treatment of Sanfilippo disease (mucopolysaccharidosis of type IIIA) and possibly other lysosomal storage diseases (e.g. acid phosphatase deficiency).

Thorough scientific literature and patent searches have resulted in the identification of compounds, which could potentially be assessed as drug candidates for the treatment of several types of mucopolysaccharidoses. Some of the compounds being considered have been already used in the clinic. This feature contributes to substantially lower the risk profile of these drug candidates. Discussions with investigators and potential partners have been initiated to outline development plans for the evaluation of these molecules in the rare genetic diseases selected by DORPHAN.

Altogether the compounds currently developed by DORPHAN constitute a diversified portfolio of promising drug candidates, from molecules in the discovery phase and early preclinical testing, to substances already used in humans for different therapeutic applications.


Dr Stéphane Demotz has joined DORPHAN beginning of 2013. Dr Demotz has over 20 years of experience in drug development gained in large pharmaceutical companies, start-ups and academic institutions in Switzerland and abroad. He is in charge of the operations of DORPHAN.

Dr Julie Charollais-Thoenig, as Project Manager, has been running the activities outsourced by DORPHAN since June 2012 and will continue to do so in 2013.

Mr Sebastien Kraft, laboratory technician in biology, is in charge of in house research development activities.

The company is led by a Board of Directors comprising Me Dunant, Mr. Maier and Dr Demotz. Me Dunant, a reputed lawyer in Geneva and Partner of the Borel & Barbey Law Firm, is the representative of the main investors. Mr. Maier has a strong business experience of the life science industry. He has occupied various positions in large companies and start-ups. He brings valuable experience in the contractual and commercial areas.


The majority of the research activities of DORPHAN is outsourced. This mode of operations maintains the running costs of the company to a minimum. Funds, otherwise invested in laboratory equipment, infrastructure and salaries of laboratory personnel, are used to run strictly defined projects in selected laboratories from academic institutions and private service providers. A small part of development and research activities are directly conducted by DORPHAN in collaboration with University of Lausanne. The investment of the company is hence presently limited to office material and office space for a staff of 3 employees. Depending on how projects will develop, the hiring of 1-2 project managers will be considered for the future.