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    Home > Active Ingredient News > Drugs Articles > Vtx801 orphan drug, a genetic drug for copper metabolic disorders, was awarded by us and European regulators

    Vtx801 orphan drug, a genetic drug for copper metabolic disorders, was awarded by us and European regulators

    • Last Update: 2017-09-29
    • Source: Internet
    • Author: User
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    Source: Biovalley 2017-09-29 today, vivet therapeutics announced that the company's genetic drug vtx801 for the treatment of Wilson's disease (WD) was awarded the title of orphan drug by us and European regulatory authorities WD is a genetic disease caused by the mutation of copper transporter ATP7B gene The incidence of WD is about 1 / 40000 In WD patients, the pathway of metabolism and elimination of copper is destroyed, which leads to the accumulation of copper in the tissues and leads to tissue toxicity (especially liver and central nervous system toxicity) If WD is not treated in time, the patient's body will gradually decline and life safety will be endangered Jean Philippe combal, CEO of vivet therapeutics, said: "physiological recovery of copper homeostasis in WD patients is the current treatment idea However, regulators in Europe and the United States are aware that this treatment cannot meet the treatment needs in terms of safety and effectiveness Fortunately, our genetic drug vtx801 has good potential in treating WD patients " Vtx-801 is an adeno-associated virus (AAV) drug carrying small-scale functional atp-7b copper transporter gene, which aims to restore the copper metabolism of hepatocytes through exogenous introduction of copper transporter gene It is the first drug awarded the title of orphan drug by vivet therapeutics Vtx-801 has greatly promoted the value of vivet therapeutics When vtx-801 is finally approved to enter the U.S and European markets, it will dominate the market of WD patients treatment for 7-10 years "For WD and other liver rare metabolic genetic diseases, we are looking for cooperation with FIMA (fundaci ó n para la investigaci ó n m é DICA aplicada) to jointly build a gene therapy R & D platform," said Jean Philippe combal The key of gene therapy design is to match the right AAV type The platform we built has obtained the AAV subtype named aav-anc80, which is a new type of specific target hepatocyte serotype authorized by mee (Massachusetts Eye and ear) of Harvard Medical School In the future, we will further explore more effective gene therapy on this basis "
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