Gene therapy offers significant potential to treat diseases with high unmet medical need. However, the unique nature of these therapies poses challenges in product development, namely: safety concerns, efficacy issues, or obstacles related to Chemistry, Manufacturing and Controls (CMC).
CMC is an integral aspect of gene therapy development. Done right, CMC development of complex manufacturing processes and analytical testing panels can accelerate clinical development and bring these important medicines to patients faster.
Open Orphan (LON:ORPH) was founded in 2017, with the goal of rapidly building Europe’s leading pharma services company by a management team with extensive industry and financial expertise. The company comprises of two commercial specialist CRO services businesses (Venn Life Sciences and hVIVO) and is also developing a genomics data platform business (Genomic Health Data).