CRISPR Continues to Cut Impressive Numbers in Beta-Thalassemia, Sickle Cell Disease, and More

A year ago, Paddy Doherty’s doctor told him that he had a rare hereditary disease called transthyretin (ATTR) amyloidosis, the same disease that had killed his father. As reported in…

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Promising Results from Clinical Trial for Stem-Cell-Based Gene Therapy for Beta-Thalassemia
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Promising Results from Clinical Trial for Stem-Cell-Based Gene Therapy for Beta-Thalassemia

According to a recent article, a new stem-cell-based gene therapy is showing promising results for treating beta (β)-thalassemia in a phase 1 clinical trial. Beta Thalassemia Beta thalassemia is a…

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Gene Therapy Reduces Need for Transfusions in Patients with Beta-Thalassemia
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Gene Therapy Reduces Need for Transfusions in Patients with Beta-Thalassemia

According to a recent article, a study shows that treatment with gene therapy reduces the need for patients with non-β0/ β0 genotype Beta-thalassemia (β-thalassemia)to get transfusions. Beta-thalassemia (β-thalassemia) Beta thalassemia…

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Patient Worthy’s One-Year-Checkup on Victoria Gray, the First Sickle Cell Disease Patient to Receive CRISPR in the US
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Patient Worthy’s One-Year-Checkup on Victoria Gray, the First Sickle Cell Disease Patient to Receive CRISPR in the US

   CRISPR is a gene-editing technique that has revolutionized the medical world with its approach to making precise changes in DNA. CRISPR found its match in 35-year-old Victoria Gray of…

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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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