Gene-Editing Technology Shows Promise For Blood Disorders
Patients with sickle-cell disease and beta-thalassemia who were treated with a CRISPR-based gene-editing therapy showed promising positive results.
AP:
Gene-Editing Treatment Shows Promise For Sickle Cell Disease
Scientists are seeing promising early results from the first studies testing gene editing for painful, inherited blood disorders that plague millions worldwide. Doctors hope the one-time treatment, which involves permanently altering DNA in blood cells with a tool called CRISPR, may treat and possibly cure sickle cell disease and beta thalassemia. Partial results were presented Saturday at an American Society of Hematology conference and some were published by the New England Journal of Medicine. (Renault, 12/5)
Stat:
CRISPR Treatment For Blood Diseases Shows Sustained Benefit
Ten patients treated with a CRISPR-based gene-editing therapy for the inherited blood disorders sickle cell disease and beta-thalassemia have shown a consistent and sustained response with manageable side effects, according to interim results from two clinical trials reported Saturday. (Feuerstein, 12/5)
In other pharmaceutical developments —
Stat:
J&J's CAR-T Drug Effective Against Myeloma, Shaded By Neurotoxicity
More than three-quarters of the multiple myeloma patients treated with an experimental CAR-T therapy from Johnson & Johnson are alive at least one year without their cancer worsening, according to updated clinical trial results presented Saturday at the annual meeting of the American Society of Hematology. (Feuerstein, 12/5)
Stat:
Constellation's Myelofibrosis Drug Shows Improved Benefit
More patients and an improved response rate might once again shift the sentiment on Constellation Pharmaceuticals and its contentious drug for myelofibrosis — this time in a positive direction. With 63 myelofibrosis patients now treated and evaluable in the company’s mid-stage study, the 24-week spleen response rate to its oral drug, CPI-0610 — when used on top of Jakafi, Incyte’s market-leading drug — stands at 67%. The new data were presented Saturday at the annual meeting of the American Society of Hematology. (Feuerstein, 12/6)
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Kura's Genetically Targeted Drug Induces Remission In AML Patients
Kura Oncology presented early clinical trial results Saturday showing for the first time that its genetically targeted cancer drug can induce responses — including complete remission — in patients with advanced leukemia. (Feuerstein, 12/5)
Also —
Stat:
CDC May Have Claims On Remdesivir Patents Held By Gilead
Six years ago, a team of scientists at the Centers for Disease Control and Prevention began researching dozens of compounds supplied by Gilead Sciences for use in combating different viruses. And they discovered that one compound, in particular, appeared effective in treating Ebola. (Silverman, 12/7)
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FTC Says The Number Of Pay-To-Delay Cases Remains Low, But Vows To Keep Watch
Although more drug makers are settling patent infringement lawsuits, the U.S. Federal Trade Commission found that the number of so-called pay-to-delay settlements — which the agency argues unfairly robs Americans of lower-cost alternatives to their prescription medicines — remain very low. (Silverman, 12/4)
