Therapeutic Focus

Leadership in Ubiquitin Drug Discovery

Progenra’s discovery platforms are derived from the ubiquitin-proteasome system, a major regulator of cellular functions. The therapeutic importance of ubiquitin is highlighted by the Nobel Prize awarded to its pioneers and the FDA approvals of proteasome inhibitors such as Velcade and ubiquitin ligase modulators exemplified by the IMiDs. Understanding of the key players in the enzymatic pathways of the ubiquitin proteasome system and their importance in various disease states has increased, and ubiquitin-based discovery research is quite active today. The most recent ubiquitin-related therapeutic modality – PROTAC® molecules – exploit the E3 ligase function by bringing together a ligase and an otherwise undruggable substrate to eliminate the substrate for therapeutic benefit. Progenra’s platforms address both small molecule ubiquitin pathway enzyme modulators and novel PROTACs.

Several Progenra scientists and collaborators were trained in leading ubiquitin laboratories, and they continue to develop first in class breakthrough small molecule drug candidates with in vivo proof of concept. Ubiquitin is not only a tag for protein degradation, but also a regulator of the cellular location or activity of extant proteins in ways that can be exploited therapeutically. We are continually incorporating newly uncovered roles for ubiquitin into Progenra’s technology. We are continually incorporating newly uncovered roles for ubiquitin, including E3 ligase based PROTACs,  into Progenra’s technology.

Progenra’s biologists, medicinal chemists, and external network enable the discovery and development of clinical candidates in cancer, cardiovascular disease, inflammation, neurodegenerative diseases, and orphan diseases such as cystic fibrosis.

PROTAC® is a registered trademark of Arvinas Operations, Inc., and is used under license.