As reported in Biospace, Fulcrum Therapeutics has announced it will be submitting an application to the FDA for FTX-6058 as an Investigational New Drug (IND) by the end of 2020 as an oral therapy to treat sickle cell disease.
Sickle Cell Disease
Sickle cell disease is the umbrella term for a group of rare genetic diseases that causes unusual hemoglobin molecules, which are found in red blood cells helping spread oxygen through the body. The mutation slows the oxygen transportation,c ausing the red blood cells to have a sickle shape. The symptoms begin at a young age, with symptoms such as anemia, frequent infections, and episodes of pain. The extent and type of symptoms vary, with some patients little effected, and others requiring intensive medical attention. Sickle cell anemia is the most known and severe of the SCD, which additionally causes painful swelling in the hands and feet, fatigue, and jaundice. SCD symptoms can escalate into pain, infections, stroke, heart disease, kidney or liver disease, and more.
Fulcrum Therapeutics, a biopharmaceutical company, focuses their clinical work on improving lives of patients with rare genetic diseases. Dr. Owen B. Wallace, the companies chief scientific officer, explained “Completed preclinical studies illustrate that FTX-6058 … has the potential to provide distinct advantages over biologics and gene therapies currently being used or developed for the treatment of sickle cell disease.” The pre-clinical studies found the treatment could increase HbF levels by about 30%. They believe this shows the treatment can help lessen the symptoms for patients with the disorder.