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Diseased, sickle-shaped blood cells can clog arteries. Source: National Institutes of Health.
Mirus Bio Corporation
, a gene therapy researcher, announced on Wednesday that it will receive a $1.26 million Phase II Small Business Innovation Research grant from the National Institutes of Health.
The funds will be used to help produce a gene therapy for the treatment of anemia, a blood disorder that kills red blood cells. This results in a lowered oxygen flow to tissues and organs and is associated with serious medical conditions such as cancer, kidney failure and AIDS.
Mirus delivery platform, Pathway IV, injects molecules of DNA into the body that carry genetic instructions to produce erythropoietin, or EPO, proteins, which help increase the volume of red blood cells. Once the EPO enters muscle cells, the expectation is that the body will respond by continually producing EPO to maintain a stable level of blood cells.
Recipients of the treatment will be given gradually increasing doses in a single treatment course, until their bodies are making enough cells. Afterward, the body should keep producing red blood cells without more injections of EPO.
The system has been successfully tested by Magdolna Sebestyen and David Lewis, Mirus researchers who previously received a Phase I SBIR grant for eliminating anemia in animal tests. Now that the team has received a second grant, they will focus on refining dosage and delivery procedures as the next step towards human treatment.
This product targets a large unmet medical need and complements our other programs for muscular dystrophy and peripheral ischemia, said Jon A. Wolff, co-founder and chief scientific officer of Mirus. With the development of our Pathway IV platform, we are able to pursue a repetitive dosing regimen to gradually restore the red blood cell population in a stepwise, controlled procedure.