Patients Who Received CRISPR Gene Editing for Beta Thalassemia and Sickle Cell Disease Remain Pain Free
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Patients Who Received CRISPR Gene Editing for Beta Thalassemia and Sickle Cell Disease Remain Pain Free

  The initial CRISPR treatment for sickle cell disease was administered to Victoria Gray in July 2019. Victoria, a resident of Forest, Mississippi, was born with sickle cell and has…

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A CRISPR Tool With the Potential to Halt the Progression of Sickle Cell and Beta Thalassemia

According to an NBC AP news item, early results show that CRISPR, a “tool” that alters DNA permanently in blood cells, has the potential to not only stop the progression…

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Now Available: FTX-6058 Proof-of-Concept Data for SCD, Beta Thalassemia
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Now Available: FTX-6058 Proof-of-Concept Data for SCD, Beta Thalassemia

For years, clinical-stage biopharmaceutical company Fulcrum Therapeutics ("Fulcrum") has worked to develop therapies for patients with rare genetic disorders and diseases. Somewhat recently, their focus was on treating beta thalassemia…

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EMA Grants PRIME Designation to CTX001, a Sickle Cell Disease Treatment
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EMA Grants PRIME Designation to CTX001, a Sickle Cell Disease Treatment

CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment,…

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Luspatercept Demonstrates Further Benefits in Transfusion-Dependent Beta Thalassemia
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Luspatercept Demonstrates Further Benefits in Transfusion-Dependent Beta Thalassemia

According to a story from Hematology Advisor, the drug luspatercept (marketed as Reblozyl) recently demonstrated its ability to reduce transfusion burden in patients with beta thalassemia that depend on regular…

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New Approach Uses CRISPR-Cas9 to Boost Fetal Hemoglobin Production to Treat Blood Disorders
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New Approach Uses CRISPR-Cas9 to Boost Fetal Hemoglobin Production to Treat Blood Disorders

  An article published earlier this year by Fierce Biotech outlines an approach that was developed by researchers at the Fred Hutchinson Cancer Research Center. Using CRISPR-Cas9’s gene-editing method the researchers were…

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CRISPR/Cas9 Looks Promising for Transfusion Dependent Sickle Cell Disease and Beta Thalassemia Patients 
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CRISPR/Cas9 Looks Promising for Transfusion Dependent Sickle Cell Disease and Beta Thalassemia Patients 

   CRISPR/Cas9 technology reached another milestone with encouraging results in two recent trials of CTX001. An article in Pharmaceutical Technology carried an announcement by CRISPR Therapeutics and Vertex Pharmaceuticals about…

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New Treatment Shows Benefits for Patients with Sickle Cell Disease and Beta Thalassemia

  New knowledge of the genes that affect sickle cell disease and beta thalassemia has allowed for advances in treatments for these diseases. Advances in technology have also helped with…

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Researchers Find Gut Bacteria that Can Change Type A Blood to Type O
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Researchers Find Gut Bacteria that Can Change Type A Blood to Type O

Blood Donations For many people living with a rare disease such as Beta thalassemia, Hemophilia, Aplastic Anemia, Myelodysplastic syndromes, Acute Promyelocytic leukemia, and others, blood transfusions are not uncommon. For…

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ICYMI: Gene Therapy That Could Make β-Thalassemia Patients Transfusion Independent Receives Marketing Authorization in EU

Transfusion-Dependent β-Thalassemia (TDT) Transfusion-dependent β-thalassemia (TDT) is a rare disease that is caused by a mutated β-globin gene. This mutation causes the amount of hemoglobin in the body to be…

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Extremely Positive Results Announced from Interim Analysis of Phase 2 Clinical Trial for Hemochromatosis

Hereditary Hemochromatosis Hereditary hemochromatosis (HH) is a rare disease caused by hepcidin deficiency or hepcidin insensitivity. Hepcidin naturally regulates iron absorption/distribution in the body. Without hepcidin, HH patients suffer from…

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Experimental Beta Thalassemia Drug Gains Temporary Short-Term Approval in Europe
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Experimental Beta Thalassemia Drug Gains Temporary Short-Term Approval in Europe

According to a publication from BNN Bloomberg, regulatory authorities in the European Commission recently granted conditional marketing authorization (CMA) to Zynteglo. Zynteglo is a gene therapy designed to treat beta…

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