UniQure gene therapy shows rise in blood-clotting protein in trial
UniQure NV said on Friday its gene therapy to treat patients with a blood disorder, hemophilia B, increased the levels of a protein that helps in blood clotting after 12 weeks.
Condition: Hemophilia B Intervention: Biological: PF-06838435/ fidanacogene elaparvovec Sponsor: Pfizer Not yet recruiting
om RNA viruses have been subjected to substantial engineering efforts to support gene therapy applications and vaccine development. Typically, retroviruses, lentiviruses, alphaviruses, flaviviruses rhabdoviruses, measles viruses, Newcastle disease viruses, and picornaviruses have been employed as expression vectors for treatment of various diseases including different types of cancers, hemophilia, and infectious diseases. Moreover, vaccination with viral vectors has evaluated immunogenicity against infectious agents and protection against challenges with pathogenic organisms. Several preclinical studies in animal model...
Roche Holding has entered a definitive merger agreement with Spark Therapeutics, as the Swiss drugmaker seeks to expand its ability to treat rare diseases through gene therapies, as well as build its hemophilia portfolio.
Roche Holding AG said on Monday it will buy Spark Therapeutics in a $4.3 billion deal, as the Swiss drugmaker builds its hemophilia portfolio and seeks to keep pace in gene therapy.
Gene therapy company UniQure NV said on Friday its treatment for hemophilia B increased the levels of a protein that helps in blood clotting after 12 weeks in a relatively small study.
Drug developer UniQure NV said on Friday its gene therapy to treat hemophilia B increased the levels of a protein that helps in blood clotting after 12 weeks in a small study.
Ahead of the opening this week, the German biotech firm signed two major partnerships with Boston-based Orchard Therapeutics and San Francisco-based Denali Therapeutics, and is also in the process of closing another partnership with a U.S. biotech company focused on hemophilia treatments.
In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are currently in clinical development, gene therapy holds the promise of a lasting cure with a single drug administration. Near-to-complete correction of hemophilia A (factor VIII deficiency) and hemophilia B (factor IX deficiency) have now been achieved in patients by hepatic in vivo gene transfer. Adeno-associated viral vectors with different viral capsids that have been engineered to express high-level, and in some cases hyperactive, coagulation factors were employed. Patient data support that sustained endogenous production of clo...
The deficiency of fibrinogen, prothrombin, factor V (FV), FVII, FVIII, FIX, FX, FXI, and FXIII, called rare coagulation disorders (RCDs), may result in coagulopathies leading to spontaneous or posttrauma and postsurgery hemorrhages. RCDs are characterized by a wide variety of symptoms, from mild to severe, which can vary significantly from 1 disease to another and from 1 patient to another. The most typical symptoms of all RCDs are mucosal bleedings and bleeding at the time of invasive procedures, whereas other life-threatening symptoms such as central nervous system bleeding and hemarthroses are mostly present only in som...