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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Editor’s Choice: Patients Helping Patients In the Midst of a Pandemic

Happy Friday! Sept. 24 is FH Awareness Day. Familial hypercholesterolemia is a gentic disorder characterized by the inability of the body to remove LDL -- sometimes called "bad cholesterol" --…

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CRISPR Editing Appears Effective in Three Patients with Rare Diseases
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CRISPR Editing Appears Effective in Three Patients with Rare Diseases

According to a story from interestingengineering.com, the capabilities of CRIPSR gene editing technology have been creating a buzz in the medical field for years now, with many seeing the technology…

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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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Rare Disease Advocates in India are Tirelessly Pushing for New Policies
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Rare Disease Advocates in India are Tirelessly Pushing for New Policies

Rare disease advocates in India are pushing for the Health Ministry to create a new fund for rare diseases. Additionally, they're pushing for medicines and medical equipment used for these…

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Gene Therapy: The New Frontier for Cancer

  Gene therapy is the treatment of disease by way of the transfer of genetic material into cells. According to a recent article in Labiotech, an EU publication, gene therapy…

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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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Gene Therapy for Severe Beta Thalassemia Earns Conditional Marketing Approval in the EU
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Gene Therapy for Severe Beta Thalassemia Earns Conditional Marketing Approval in the EU

According to a story from BioPortfolio, the gene therapy company bluebird bio, Inc. recently announced that its gene therapy ZYNTEGLO™ has earned conditional marketing authorization in the European Union from…

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Experimental Treatment for Beta Thalassemia Earns Fast Track Designation
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Experimental Treatment for Beta Thalassemia Earns Fast Track Designation

According to a story from globenewswire.com, Vertex Pharmaceuticals Inc. and CRISPR Therapeutics have recently announced that the US Food and Drug Administration (FDA) has awarded CTX001, an experimental gene-edited stem…

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Gene Therapy for Beta Thalassemia Recommended for EU Approval

According to a story from Stockwatch, the gene therapy company bluebird bio, inc. recently announced that the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has…

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A Beta Thalassemia Patient will be the First Dosed with CRISPR-Based Gene Therapy in Company Sponsored Trial
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A Beta Thalassemia Patient will be the First Dosed with CRISPR-Based Gene Therapy in Company Sponsored Trial

According to a story from MedCity News, a patient with the rare blood disorder beta thalassemia will be the first patient dosed in a Phase I/II clinical trial involving a…

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Updates From Celgene in Follicular Lymphoma, Marginal Zone Lymphoma, Beta Thalassemia, and other Rare Diseases

Celgene Corporation has recently announced two exciting updates for the rare disease community! Update #1 The first big announcement from Celgene was that their supplemental New Drug Application (sNDA) for…

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Clinical Trial Results Look Good for Possible Treatment for Transfusion Dependent Beta Thalassemia
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Clinical Trial Results Look Good for Possible Treatment for Transfusion Dependent Beta Thalassemia

According to a story from Market Screener, the biopharmaceutical company Orchard Therapeutics recently announced that the results of a recent clinical trial testing the company' investigational product OTL-300 were encouraging.…

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