FORMA Therapeutics, a biopharmaceutical company headquartered in Massachusetts, just sent out a press release announcing that the company raised $100 Million (Yes, a hundred million :O). A monstrous round in Series D Financing will fuel the development of their therapy FT-4202 and FT-7051 that is treating patients suffering from rare blood disorders.
FORMA’s FT4202 may potentially play a critical role for being the next therapy for sickle cell. By targeting the mechanism of action that often leads to the development of blood disorders in patients, one can reverse and prevent the development of sickle cell disease. Patients who suffer from this disease tend to have a deficiency of PKR. FORMA’s investigational drug addresses this issue by being an activator for pyruvate kinase RNA (PKR). The activation leads to PKR activation and the binding of hemoglobin to oxygen for these patients.
In a research paper, Allosteric inhibitors of pyruvate Kinase, pyruvate kinase activators can be used potentially as a pharmaceutical treatment for patients with PK deficiency. Because kinase influences the metabolic process and glycolytic pathway, 2,3-diphosphoglycerate, a protein critical in letting hemoglobin to have the affinity it needs to bind with oxygen.
Most drugs that are produced by pharmaceuticals and biotechnology companies try to treat the symptoms of the disease. By reversing and preventing the development of this disease, an activator of PKR can potentially give better results for patients with rare hematologic disorders.
We are no longer creating traditional drugs that just treat the symptoms of a patient, BUT RATHER we are now looking at the underlying cause of a disease.
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