PROTAC for Rare Diseases

Rare diseases are an emerging area of concern. The cure of rare diseases and the development of new drugs for rare diseases are common problems faced by the global pharmaceutical industry. Although the incidence of rare diseases is low, there is still a high social demand for the therapy and drug development of rare diseases. Proteolysis targeting chimera (PROTAC) is a new drug discovery paradigm that is opening an emerging field that will bring surprises to drug design and development. PROTAC has attracted extensive attention in drug development due to its unique mechanism of action. Currently, there are already some PROTACs for rare diseases:

KSK29116 for relapsed refractory B-cell lymphoma

Targeting TRK for TRK-mediated cancers

Targeting Fak for triple-negative breast cancer (TNBC)