Rare or orphan diseases can be inherited and may only affect a relatively small population worldwide. Alternatively, they can be rare forms of acquired diseases that span other therapeutic areas such as respiratory, oncology, inflammation and infectious diseases.
Rare/Orphan Diseases
A number of our clients specialize in finding life-changing medicines for rare/orphan diseases that may be poorly served by current medications.
Such diseases are often targeted by emerging biotech companies who make up the majority of our customers.
Inhouse Expert Teams
We are experienced in working with companies in the orphan/rare disease space and understand the needs of this sector.
This can include collaborating with partners to access specific sources of funding (such as grants) and where necessary, exploring flexible deal structures to ensure mutual success.
Our Track Record
Take a look at examples of work we have carried out in this area:
- How we supported Nflection to deliver the first clinical trial of NFX-179 Gel, a topically applied therapy for the mitigation of Cutaneous Neurofibromatosis Type-1
- Elaboration of existing hits to elucidate SAR
Download our eBook on developing effective assays:
