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    Home > Medical News > Latest Medical News > Rare disease best-selling drug Ikuzhu single anti-competition opponent sickle cell disease gene therapy update data.

    Rare disease best-selling drug Ikuzhu single anti-competition opponent sickle cell disease gene therapy update data.

    • Last Update: 2020-10-21
    • Source: Internet
    • Author: User
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    On June 11, 2020, the 25th EHA Conference was opened, affected by the new crown epidemic, the session was held online, the meeting announced a number of lymphoma, bone marrow growth abnormal syndrome, sickle cells and other blood disease research progress.
    two advances in EHA are of particular concern: 1. Sickle cell gene therapy LentiGlobin updates the key data for sickle cell disease, HGB-206, which is expected to accelerate Lenti Globin's approval for sickle cell disease; PEGASUS data update, Pegcetacoplan vs Ekuzhu monoantigen significantly improve patients' hemoglobin levels and blood transfusion dependence, Pegcetacoplan will change the standard therapy for hairy sleep hemoglobin uremia.
    one. Sickle cell disease: Gene therapy LentiGlobin Innovative Treatment Solutions On March 25, 2019, LentiGlobin reached its first milestone, with the ACCELERAT-205 and Northstar (HGB-204) clinical data accelerated approval by the EU For non-beta-0/beta-0 genotype transfusion dependence β thalassemia over 12 years of age, LentiGlobin brings new options to patients with non-hematopoietic stem cell transplantation to adrenalemia, and Lenti Globin is the only approved gene therapy for thalassemia.
    LentiGlobin is a CD34 plus autosociogenic hematopoietic stem cell that uses BB305 LVV to express the beta-A-T87Q-globin gene, and more details about this gene therapy and related clinical data can be found on bluebird bio.com.
    LentiGlobin production process Bluebird bio, EHA: LentiGlobin update sickle cell disease key data HGB-206 This EHA, LentiGlobin in addition to updating Northstar-2 (HGB-207), Northstar-3 (HGB-212) data, the other most important update is sickle cell disease HGB-206 C group data.
    sickle cell disease: about 300,000 newborns worldwide, most 45-year-old death sickle cell disease is a genetic disease, hemoglobin β chain of glutamate is replaced by proline, mutated hemoglobin easily formed a tetum, red blood cells twisted into a typical new moon or sickle shape, deformation significantly decreased.
    Sickle Cell Disease, PMID: 29542687 Connect above, patients with sickle cell disease are more susceptible to vaso-occlusive crisis, VOC, acute chest syndrome (acute chest, ACS) due to reduced erythrocyte deformation.
    more than half of the patients are difficult to exceed 45 years of age.
    Bluebird bio, clinical benefits are significant: the incidence of vascular obstruction risk and acute chest syndrome decreased by 99% Bluebird bio, Safety: Bluebird bio published the results of deaths, deaths may not be related to LentiGlobin in 25 patients the same updated safety data, but also explained the cause of the previous death, autopsy results suggest that death may not be related to LentiGlobin, but cardiovascular events caused by the safety of the disease.
    Bluebird bio, Bluebird bio, HGB-206 Group C data will support the accelerated approval of this breakthrough gene therapy, while the confirmed clinical trial HGB-210 has been launched, and LentiGlobin is expected to reach another important milestone in the second half of 2021 and is expected to expand to sickle cell disease.
    noteworthy is that another CRISPR/Cas9 gene editing therapy, CTX001, has also shown good early results in development, which is not covered in detail in this paper.
    two. Burst sleep haemoglobinuria: Ikuzhu single resistance ushered in an important challenge In the author's previous article, "Kurida breaking 10 billion vitavi and Dupixent performance leapfrog growth" we can easily see that the world's best-selling TOP50 drugs, its adaptive disorders are mostly tumors, autoimmune, diabetes and other large-adaptive disorders.
    of these, Alexion developed the rare disease drug Ikuzhu single resistance on the list, Ikuzhu single resistance has become a standard treatment for bursts of sleep-based hemoglobin uremia, a rare disease in the field of best-selling drugs, sales of $3.946 billion in 2019.
    , PEGASUS: Pegcetacoplan significantly increases hemoglobin levels Blood transfusion dependence reduces PEGASUS (APL2-302); NCT03500549) Clinical Design
    Pegcetacoplan:compstatin derivatives, inhibiting classic, bypass, coagulant three complement activation paths, about 4,700 cases of hemoglobinuria patients in the United States, clinically accompanied by blood Hydrant formation and hemolysis, up to now, Ikuzhu monoantigen is the only approved standard treatment for cystic sleep-based hemoglobin uremia, but only 29% of patients receiving icuponumin resistance can have hemoglobin levels of up to 12 g/dL.
    Pegcetacoplan is a compstatin derivative, C3 inhibitor, the drug can be through C3 to inhibit the classic, bypass, coagulant three complement activation path, which is not the same as Ikuzhu monoantitor.
    PEGASUS data clearly show that Pegcetacoplan vs. Ikuzhu single anti-transfusion dependence can significantly improve patient hemoglobin levels, 16-week hemoglobin levels, .2.37 vs. -1.47, significantly better than Ikuzhu monoantigen;
    Pegcetacoplan will undoubtedly bring new treatment options to PNH patients, and according to the company's update, Pegcetacoplan will submit a listing application in 2020, and the drug is also being developed for condensation, cytopathy, and ophthalmological map shrinkage.
    С3first-in-class,PNH!
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