MONTRÉAL, January 7, 2021 – ExCellThera Inc., a clinical-stage molecular medicine company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, announced today the publication of new data describing the mechanism of action of UM171, the proprietary molecule which forms the basis of ECT-001 Cell Therapy.
The new data, published in the peer-reviewed journal, Cell Stem Cell, demonstrates that UM171 leads to specific epigenetic modifications that have a profound impact on human stem cell self-renewal, and potentiates the activity of a novel first-in-class E3 ligase complex which plays a role in the degradation of several proteins, including LSD1. The full publication can be accessed here.
LSD1 is a well-know target in cancer therapy and UM171 represents the first LSD1 degrader. To develop the newly identified potential of UM171, ExCellThera also announced today that it has recently incorporated a new subsidiary named RejuvenRx Inc., which will own and develop all of the assets and rights related to the UM171 family of molecules in the field of targeted therapy for the treatment of cancers and other diseases. This includes their newly identified potential as epigenetic modifiers and protein degraders (PROTACs).
“The discovery of this new complex provides insight into stem cell renewal and expansion, but perhaps more importantly, we can identify promising new avenues to develop UM171-based targeted therapies to treat cancers and other diseases. This promise is further amplified by the fact that members of this protein complex are expressed in most cell types, possibly suggesting that its activity is more ubiquitous than what is described in this work.” said Dr. Guy Sauvageau, CEO and founder of ExCellThera, President of RejuvenRx and senior author on the publication.
ExCellThera has already shown the clinical success of UM171 through ECT-001 Cell Therapy, which has been used in over 70 patients with severe blood disorders. ECT-001 Cell Therapy has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designation from the U.S. Food and Drug Administration as well as orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification and priority medicines (PRIME) designation from the European Medicines Agency.
About ExCellThera Inc.
ExCellThera is a clinical-stage cell and molecular medicine company delivering molecules and bioengineering solutions to expand stem and immune cells for use in novel one-time curative therapies for patients with hematologic malignancies and other diseases. ExCellThera’s most advanced technology, ECT-001 Cell Therapy, combines a proprietary small molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its portfolio of products, as well as supporting best-in-class clinical trials. excellthera.com