FDA Awards FT-4202 Orphan Drug for Sickle Cell Disease
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Last Update: 2020-06-25
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Source: Internet
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Author: User
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FORMA, a clinically phased biopharmaceutical company focused on rare haematology diseases and cancers, recently announced that the FDA has awarded its FT-4202 orphan drug designation, which is currently under clinical development and can be used as a potential treatment for sickle cell disease (SCD)FT-4202 is an activator of acetone kinase R (PKR), and preclinical studies have shown that FT-4202 can treat SCD anemia and vascular aphation"Despite the FDA's recent approval of therapies for SCD, there are no innovative treatments that can really alleviate the disease," commented FRANK Lee, CEO of FORMASickle cell disease (SCD) is one of the most common diseases caused by single gene mutationsSCD is a pure lysy abnormal hemoglobin (HbS) genetic diseaseVascular obstruction can lead to recurrent pain attacks (formerly known as sickle cell crisis) as well as serious organ system complications that can lead to lifelong disability and/or premature deathHbS is the sixth-ranked glutamate in the beta-gloprotein chain, which is replaced by proline, which produces a hemoglobin tetrapolymer (alpha2/betaS2), which is virtually insoluble during deoxygenation
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