Orphan Disease
Making a difference to patients is one of Sygnature Discovery’s core values. Our experienced scientists understand the challenges of working in the field of rare and orphan diseases, using our knowledge of disease mechanisms to add value to your research projects.
Our co-located Chemistry, DMPK, Computational Chemistry and Bioscience teams work flexibly with our clients to design and implement tailored and informed strategies to advance your discovery project, enabling our clients to develop and commercialise novel therapeutics for rare, debilitating, and potentially fatal diseases.
Using our Integrated approach, we offer:
- A collaborative work ethic
- Intellectual input into compound design, project screening strategy and progression pathways
- Design of complex in vitro and ex vivo Screening Cascades
- Provision of data to support client patent applications
- Clinical Biomarker Identification and validation
- In vivo support via our established network of strategic partners
Sygnature Discovery has a proven track record in successfully supporting a number of challenging orphan disease projects, such as rare metabolic disorders (Corcept Case Study) and Lysosomal Storage Disease (LSD). By applying our extensive drug discovery knowledge and expertise, we have enabled the delivery of two orphan indication clinical candidates since 2011, one of which is currently in Phase II clinical development.