Memorandum

Orgenesis receives $1.5 million term loan to fund beta cell replacement research, announces agreement with MaSTherCell to scale up operations for clinical trials – July 21, 2014

Executive Highlights

  • Orgenesis announced that it has received a new $1.5 million term loan to advance its research on autologous beta cell replacement therapy.
  • Orgenesis has signed an agreement with MaSTherCell that will enable it to bring its manufacturing process “from lab scale to clinical scale.”

Orgenesis Inc., a US-based development-stage company investigating a novel beta cell replacement therapy for type 1 diabetes, announced on June 25 that it has raised $1.5 million through a senior secured term loan facility to support research on its novel beta cell replacement procedure. Orgenesis CEO and chairperson Ms. Vered Caplan said the funds will be directed to the Belgian subsidiary Orgenesis SPRL, which focuses on product manufacturing of cells for clinical trials in Europe. Additionally, Orgenesis announced that it has signed an agreement with MaSTherCell, a Belgian company that specializes in large-scale manufacturing of cell therapy products, to scale up its manufacturing process to a level necessary for clinical trials.

Orgenesis’ research efforts are focused on using trans-differentiation to convert liver cells into insulin-producing beta cells that could be used for transplantation in patients with type 1 diabetes. In the procedure, the patient’s own biopsied liver cells are cultured with a cocktail of growth and transcription factors, including PDX-1, that induce them to differentiate into “pancreatic beta cell-like” cells, and those differentiated cells are then transplanted back into the patient’s liver. Preclinical studies have demonstrated that this method can successfully induce permanent differentiation of human liver cells into cells that release mature insulin in a glucose-dependent manner and ameliorate hyperglycemia in several animal models. Importantly, the trans-differentiated cells seem to resist the autoimmune attacks characteristic of type 1 diabetes, and none of the animals developed hypoglycemia or malignancies in response to treatment.

Though many questions about the efficacy, safety, and durability of this therapy will need to be addressed in clinical trials, if successful, it would represent a major advance over current beta cell transplantation techniques, which are hindered by the shortage of donor islet cells, the need for immunosuppressants to prevent rejection of foreign tissue, and the threat of an autoimmune attack. Orgenesis management characterized the technique as “a completely different approach” from other cell-based therapies for type 1 diabetes, as the cells are specifically programmed to produce insulin in a glucose-dependent fashion and appear to be capable of remaining in the liver and producing insulin on a long-term basis. See this article for more information about the science behind this technique and Orgenesis’s website for more details about their development program.

 

--by Emily Regier, Manu Venkat, and Kelly Close