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…
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…
A press release from the US Food and Drug Administration (FDA) is announcing the approval of a new treatment for anemia in adult patients with the rare blood disorder beta…
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…
According to a publication from Fierce Biotech, Biotech company Sangamo recently updated investors on three trials it has underway testing experimental forms of gene and cell therapies. The trials are…
According to a story from Australasian Science Magazine, the dawn of the gene therapy "revolution" is upon us. While there are only a handful of gene therapies that have actually…
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…
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…
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…
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…
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…
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…
Josephine Bila was born with transfusion dependent beta thalassemia, a rare blood disorder. Recently, Josephine uploaded a video to Youtube in which she talks about her life as a patient,…
According to a publication from Blood Journal, an experimental beta thalassemia drug called luspatercept yielded positive results at the end of a phase 2 clinical study. Many patients enjoyed an…
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…
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…
According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma Inc., recently announced the release of results from a Phase 2 study which tested the drug luspatercept as a…
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.…
CRISPR Therapeutics and Vertex Pharmaceuticals have been collaborating to develop a new treatment option for Sickle Cell Disease. It's called CTX001, a type of stem cell therapy. This drug has…
According to a story from EurekAlert!, the 60th Annual Meeting of the American Society of Hematology featured a number of different presentations of the latest research from St. Jude Children's…
According to a story from PR Newswire, presentations at the 60th annual American Society of Hematology Meeting and Exposition are highlighting the results of four large scale clinical trials that…
Rare disease research is often underrepresented in the field of science. However, recent developments have made researchers more interested in studying this group of diseases. For instance, investments in gene…
The United States Food and Drug Administration has awarded Fast Track designation to an investigational drug called PTG-300 for treating chronic anaemia caused by ineffective erythropoiesis in patients who have…
According to a recent press release from Bluebird Bio, the European Medicines Agency has accepted and will review their marketing authorization application (MAA) for the investigational gene therapy LentiGlobin™ as…
According to a story from PR Newswire, the drug developer Protagonist Therapeutics recently announced that its investigational therapy PTG-300 has earned Fast Track designation from the US Food and Drug…
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