Restoring Erythropoiesis in Myelodysplastic Syndrome

Researchers at QMUL and Université de Bretagne-Occidentale have developed a method of restoring erythropoiesis in a patient suffering from a SF3B1 mutant myelodysplastic syndrome, involving the administration of splice switching antisense oligonucleotides (SSO) capable of correcting COASY mis-splicing.

Ineffective erythropoiesis and anaemia are hallmarks of myelodysplastic syndrome. Erythropoiesis stimulating agents have an initial response rate of approximately 50%, but they eventually become resistant. Most patients eventually become dependent on red-blood-cell transfusions, reducing quality of life and increasing the risk of progression to acute myeloid leukaemia.

A patent has been filed under the Université de Bretagne-Occidentale and Queen Mary University of London and we are now seeking partners for licensing.