MONTRÉAL, May 19, 2020 – ExCellThera Inc., a clinical-stage molecular medicine company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, announced today that it has received a grant from the California Institute for Regenerative Medicine (CIRM) to fund a Phase 1 clinical trial using ExCellThera’s most advanced technology, ECT-001 Cell Therapy, for the treatment of severe sickle cell disease.

The study will be conducted in California at the Lucile Packard Children’s Hospital affiliated with Stanford University to demonstrate the safety and feasibility of ECT-001 Cell Therapy in children and young adults with severe sickle cell disease. Sandeep Soni, M.D., Associate Professor, Pediatric Stem Cell Transplant, Gene Therapy and Regenerative Medicine, Stanford University, will be responsible for the clinical evaluation and selection of patients for participation in this program.

Sickle cell disease is an inherited blood disorder, most commonly affecting people of African descent. It is caused by a genetic mutation, characterized by a sickle-shaped deformation in red blood cells. This mutation prevents the red blood cells from carrying oxygen effectively through the body and can obstruct the bloodstream, causing extreme pain, recurrent hospitalization and organ failure. It is estimated that sickle cell disease affects about 100,000 people in the United States and Canada.

“The current standard of care for severe sickle cell disease is a blood stem cell transplant, which is only available to patients who have a matched donor,” said Dr. Guy Sauvageau, CEO and founder of ExCellThera. “However, the unique properties of ECT-001 Cell Therapy enable patients without a matched donor to receive treatment, with a greatly reduced risk of post-transplant complications. This study introduces a new indication into the ongoing evaluation of our ECT-001 technology, which we hope will make this life-saving therapy available to a far greater number of severely ill patients.”

The ECT-001 technology, comprised of the UM171 small molecule and an optimized culture system, is used in novel curative blood transplant therapies for patients with blood cancers and disorders. ECT-001 Cell Therapy has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designation from the US FDA and orphan medicinal product designation from the European Medicines Agency.


About ExCellThera Inc.
ExCellThera is a clinical-stage molecular medicine biotechnology 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, autoimmune and other diseases. ExCellThera’s lead technology 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.