Rare or orphan diseases affect only a small patient population, but are often burdensome, with early onset and severe or even life-threatening consequences. Many are of genetic origin but some develop without a genetic cause, such as certain autoimmune diseases or cancers. There are ~7,000 known rare diseases with many lacking effective treatment. Evotec has a longstanding experience in supporting the integrated R&D value chain across numerous rare diseases associated with different therapeutic areas and modalities.
- Huntington’s Disease (HD) multi-year drug discovery alliance with the CHDI Foundation since 2006, including the development of a PET ligand for HD
- Haematology disorders, haemophilia, von Willebrand disease, ADAMTS13 deficiency, hereditary angioedema and sickle cell disease treated via gene therapy
- Muscular disorder treatments for for dysferlinopathy, FSHD, and Duchenne’s muscular dystrophy explored with the Jain Foundation, Facio Therapies, and more clients
- Lysosomal Storage Disorders such as Gaucher disease, modelled with iPSCs in collaboration with rare disease specialist Centogene
- Retinal disease collaboration with the Center for Regenerative Therapies Dresden (CRTD) initiated to drive the identification of iPSC-based cell therapies
- Dravet syndrome treatment ensured through API development and commercial manufacture of FINTEPLA on behalf of Zogenix
Key capabilities of Evotec’s rare riseases discovery platform
Multi-modality
- Small molecules
- Antibodies
- RNA interference
- Gene Therapy
- Cell Therapy
