French clinical-stage biopharmaceutical company, Erytech Pharma, has partnered up with Queens University to advance the preclinical development of its eryminase program specifically for the treatment of arginase-1 deficiency, a rare and severe metabolic disorder related to arginine metabolism.
The biotech, of late has been applying its encapsulation technology to the treatment of metabolic diseases. The agreement with Queen’s is intended to further this early-stage work.
Arginase-1 deficiency is a rare, inherited disorder of the urea cycle caused by a mutation in arginase-1 gene that results in accumulation of toxic levels of the amino acid arginine in the blood. The symptoms generally appear in early infancy and include intellectual disability, non-ambulatory muscle stiffness and seizures. It is a debilitating, progressive disease with very limited treatment options currently available.
Lyon, France-based Erytech is best known for encapsulating the asparaginase enzyme in red blood cells to treat acute lymphoblastic leukemia. It now hopes to employ engineered RBCs to produce the enzyme L-asparaginase, which depletes the amino acid asparagine from the bloodstream to starve the tumor cells that, unlike healthy cells depend on the amino acid to survive.
The collaboration will leverage the expertise of Queen’s University and Erytech’s ERYCAPS platform technology with the
goal of generating in vivo proof-of-concept data in an arginase-1 deficiency animal model.“Arginase-1 deficiency is a severe, rare disorder affecting a biochemical pathway that disposes of toxic ammonia. Normally, our bodies are very efficient at removing any ammonia that accumulates after eating a protein-rich meal. However, in patients with arginase-1 deficiency, the ammonia is ‘partially detoxified’ leading to a large accumulation of the amino acid arginine in the patient’s blood and brain. ERYTECH’s product candidate eryminase aims to reduce the level of arginine in blood and thus, has significant potential to reduce negative consequences of this disorder. We look forward to working with ERYTECH to advance their preclinical program.” Said Prof. Colin Funk, PhD, Queen’s University.
