Although clinical trials are in progress, no treatment is still on the market and unfortunately new children are worldwide diagnosed, but also in Geneva.


actual research

Research Company MPS Type statut
Gene therapy Abeona Sanfilippo A & B Clinical
Gene therapy Lysogene Sanfilippo A Clinical
Chaperon Dorphan Morquio & GM1 pre-clinical
Imunosugar CMU – Geneva Heparanase fundamental research

stopped research

Research Company MPS Type statut
Greffe de la moelle Compagnie Type de MPS stopped
Modulation pharmacologique activité lysosomale Compagnie Type de MPS stopped
Chaperons chimiques N-acétylglucosaminidase Compagnie Type de MPS stopped

Portfolio

Abeona Therapeutics

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Approach:Gene editing/CRISPR
Funding Impact: Helped found company and fund pre-clinical safety and efficacy research

Lysogene

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Approach: Gene editing/CRISPR
Funding Impact: Helped found company and fund pre-clinical safety and efficacy research

Dorphan

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Approach: Gene editing/CRISPR
Funding Impact: Helped found company and fund pre-clinical safety and efficacy research

CMU - Geneva

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Approach: Gene editing/CRISPR
Funding Impact: Helped found company and fund pre-clinical safety and efficacy research