uniQure recently announced updated clinical data for three patients who have been treated with AMT-061, the company’s investigational gene therapy for patients with severe and moderately severe hemophilia B. AMT-061 consists of adeno-associated virus serotype 5 AAV5, which has been demonstrated to be safe and well-tolerated, acting as a. La Hemofilia B es el resultado de un déficit de factor IX factor anti hemofílico B. Esta afecta, aproximadamente, a un nacimiento de 30.000 infantes de sexo masculino. Esta enfermedad es transmitida por las mujeres, según un mecanismo recesivo vinculado al cromosoma X. Las mujeres que pueden transmitir la enfermedad se denominan conductoras. 30/01/2017 · UniQure has received Breakthrough Therapy Designation from the FDA, granting fast track to its gene therapy for hemophilia B. UniQure is one of the companies leading the development of gene therapy for hemophilia. Now, the FDA has granted Breakthrough.
uniQure also announced last month that it had reacquired the rights from Chiesi Group to co-develop and commercialize its hemophilia B gene therapy in Europe and other select territories and to terminate their co-development and license agreement. uniQure now has. uniQure is continuing to enroll participants for its global Phase 3 trial NCT03569891 testing the potential gene therapy AMT-061 in men with severe or moderately severe hemophilia B, according to an update from the company. UniQure N.V. has announced that is has “scaled up” the manufacturing process at its large facility in Lexington, MA. According to a uniQure press release, the facility is designed to manufacture the company’s proprietary hemophilia B gene therapy candidate, AMT-060. Research & Development. Our mission at uniQure is to deliver curative gene therapies that transform the lives of patients. We have an ongoing clinical program in hemophilia B and preclinical proof-of-concept for a gene therapy in Huntington's disease. Based in Lexington, Mass. and Amsterdam, The Netherlands, uniQure updated its Phase IIb trial of AMT-061 in hemophilia B. AMT-061 is an AAV5-based gene therapy that contains a FIX-Padua variant, meaning a patent-protected form of Factor IX, the missing.
01/08/2017 · Chiesi has cut its ties to uniQure’s hemophilia B gene therapy. The split gives uniQure full rights to AMT-060 but leaves it without a partner to cofund R&D as it. Subject: UniQure Aiming To Be First With Hemophilia B Gene Therapy Add a personalized message to your email. Cancel. Send. Please Note: Only individuals with an active subscription will be able to access the full article. All other. 14/01/2019 · QURE’s gene therapy targets hemophilia B, reducing the cost of current FIX replacement therapy. Its edge over competition is in the use of the AAV5 caspid. Despite competition, QURE has positioned itself to capture part of the hemophilia B market. uniQure. / EIN News / - ~ Actividad FIX sostenida a niveles terapéuticos1 Hasta 50% de lo normal al año después de la administración de etranacógeno Dezaparvovec.
Uniqure’s second try at haemophilia B looks better than its first. The first three patients dosed with the new version of its gene therapy AMT-061 saw their factor IX levels rise to 31% of normal, on average, after six weeks' treatment. 03/09/2019 · UniQure's main competition is from Pfizer, which is developing a rival hemophilia B therapy it licensed from Spark Therapeutics. Its Phase 3 study began in July 2019, and is set to read out results in July 2021. Sangamo Therapeutics is also developing a gene editing approach to treating hemophilia B.
Gene therapy uniQure NV Amsterdam reported that factor IX levels of patients with severe hemophilia B raised to a mean of 38% of normal 12 weeks after a single injection of AMT-061. 18/06/2018 · There are two main types of the condition: A and B. Your child's type depends on which protein, or clotting factor, he lacks. There are 13 of these clotting factors in our blood. With hemophilia B, you don't have enough factor IX. More than half the people with it have a severe case; they have almost no factor IX in their blood. On the stock market today, Uniqure stock popped 10%, to 36.49. The biotech is rivaling Dow Jones component Pfizer and partner Spark Therapeutics with a gene therapy for hemophilia B treatment. Uniqure tested its gene therapy in three patients over 12 weeks. 28/06/2018 · Uniqure's AMT-061 can potentially disrupt and transform hemophilia B treatment on three fronts, Kumar said in the initiation note: The company's recently issued patent should eliminate any concerns tied to the patent estate, which makes the company "the dominant firm" in hemophilia B. 21/05/2018 · uniQure Presents New Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-Existing Anti-AAV5 Neutralizing Antibodies. Email Print Friendly Share. May 21, 2018 07:00 ET Source: uniQure Inc.
19/10/2017 · In a PhIII showdown with Spark, uniQure switches out its lead gene therapy for hemophilia B. And one-time player Dimension Therapeutics was forced out of hemophilia B by weak results, with Ultragenyx picking up the rest of the pipeline in a buyout. The race goes on. 19/01/2018 · La hemofilia B es el resultado de la incapacidad del cuerpo para producir suficiente factor IX. La hemofilia B es causada por un rasgo hereditario recesivo ligado al cromosoma X, con el gen defectuoso localizado en el cromosoma X. 01/08/2017 · Chiesi Group has ended its four-year-old collaboration with uniQure to co-develop and license a gene therapy for hemophilia B and has returned its development and commercialization rights to the gene therapy candidate in Europe and other areas, uniQure acknowledged. In a statement yesterday, Chiesi. UniQure is switching the gene therapy it plans to move into a pivotal trial in hemophilia B patients because the new version could have clinical benefits. Regulatory agencies in the US and Europe have given the change a green light, and uniQure plans to begin a pivotal trial in 2018.
UniQure stock approached a breakout Friday after its experimental gene therapy showed promise in hemophilia B treatment. X. On the stock market today, shares of UniQure popped 6.7%, to 61.10, in above average volume. uniQure: AMT-061 para la Hemofilia B. En agosto, uniQure anunció el tratamiento del primer paciente con hemofilia B grave o moderadamente grave como parte del estudio de confirmación de dosis en fase 2b de la compañía de su producto de terapia genética en investigación AMT-061. Gene therapy uniQure NV Amsterdam reported that factor IX levels of patients with severe hemophilia B raised to a mean of 38% of normal 12 weeks after a single injection of AMT-061. AMT-061, an AAV5 vector, expresses a gene cassette with the Padua variant of Factor IX, which lacks in hemophilia B. 14/05/2019 · A gene therapy developed by the Dutch biotech uniQure increased the activity of a blood clotting protein enough to stop bleeds in patients with the blood disorder hemophilia B. Hemophilia B is a rare genetic condition in which patients have a mutation in the blood clotting protein factor IX FIX, reducing its activity below 6% of.
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