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Caladrius Biosciences, Inc., announces that its product candidate CLBS03 (autologous expanded polyclonal regulatory T cells, or Tregs) for the treatment of recent-onset type 1 diabetes (“T1D”) was granted orphan-drug designation by the US Food and Drug Administration (“FDA”) for the treatment of type 1 diabetes mellitus with residual beta cell function.
The scientific basis for this therapeutic stems from the use of Tregs to treat autoimmune diseases caused by T cell imbalances in an individual’s immune system. This novel approach seeks to restore immune balance by enhancing Treg cell number and function. Tregs are a natural part of the human immune system and regulate the activity of T effector cells, which are responsible for protecting the body from viruses and other foreign antigens. When Tregs function properly, only harmful foreign materials are attacked by T effector cells. In autoimmune diseases, it is thought that deficient Treg activity permits the T effector cells to attack the body’s own beneficial cells, and in the case of T1D, insulin-producing pancreatic beta cells.
“Obtaining orphan drug designation is a key step in our regulatory and development strategy for CLBS03,” said David J. Mazzo, PhD, Chief Executive Officer of Caladrius. “Coupled with the progress we are making in advancing the product through to clinical milestones, we believe that this will make CLBS03 an even more attractive opportunity for a potential partner.”
