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Hemophilia and von Willebrand disease are common congenital bleeding disorders. Fifteen other bleeding disorders have been identified along with hemophilia A and B. These include factor VII deficiency, factor XIII deficiency, factor I (fibrinogen) deficiency, afibrinogenemia, hypofibrinogenemia, dysfibrinogenemia, factor II deficiency, factor V deficiency, factor X deficiency, factor XI deficiency (hemophilia C), Glanzmann’s thrombasthenia, Bernard-Soulier syndrome, platelet storage pool deficiency, and acquired hemophilia.
Leading companies are combining experience in protein design with collaborations in the international scientific community to develop effective and safe treatments for individuals with hemophilia and other rare bleeding disorders. Scientists are testing innovative, long-acting and subcutaneous treatment solutions for rare blood disorders and hemophilia. Such solutions seek to reduce current treatment burden and improve clinical outcomes. For example, gene therapy seeks to treat a genetic disease or disorder by providing individuals with working copies of the gene to correct the disease or disorder. Multiple approaches to gene therapy including gene transfer, cell therapy, and gene editing (CRISPR).
Anti-tissue factor pathway inhibitor (TFPI) or anti-TFPI aims to decrease bleeding by regulating tissue factor-induced coagulation through factor Xa-dependent feedback inhibition of the tissue factor-factor VIIa complex. In other words, anti-TFPI reinstates hemostatic balance by blocking one of the anti-coagulants – TFPI - and prevents it from working normally; as anti-coagulants reduce clotting, altering the way they function enables clotting.
Encapsulated cell therapy involves placing in the body specialized capsules containing cells created to make clotting factors. The clotting factors are administered into the body at a constant rate. The capsules protect the cells from the human immune system.
RNA interference (RNAi) therapy targeting antithrombin targets an anti-coagulant called antithrombin. This is a new treatment system that aims to re-establish hemostatic balance by reducing antithrombin which enables the formation of sufficient thrombin (a coagulant), to prevent bleeding. As it does depend on replacing a particular clotting protein, such as factor VIII or factor IX, it may be employed to prevent bleeding episodes in hemophilia A as well as hemophilia B.
In 2021, uniQure and CSL Behring announced trial updates for hemophilia B gene therapy. It was announced that etranacogene dezaparvovec, an investigational hemophilia B gene therapy in clinical trials, had achieved a primary endpoint of “non-inferiority” in annualized bleeding rate (ABR) 18-months post administration. The non-inferiority element of a clinical trial assessed if the investigational therapy was not worse/less efficacious than a currently available product. Here, etranacogene dezaparvovec was compared to baseline factor IX prophylactic replacement therapy as part of the phase III HOPE-B pivotal trial. It was also reported that etranacogene dezaparvovec was generally well-tolerated with above eight percent of adverse events “considered mild. In 2021, CSL Behring received accelerated CHMP (Committee for Medicinal Products for Human Use) assessment for etranacogene dezaparvovec for European patients living with hemophilia B.
In 2021, Novo Nordisk announced that it entered into a definitive agreement to acquire Dicerna Pharmaceuticals (Dicerna). The acquisition was a strategic addition to the company’s existing research technology platforms and supported the strategy of employing a wide range of technology platforms applicable across all Novo Nordisk’s therapeutic focus areas. Dicerna was a publicly held company engaged in RNAi-based therapeutics. Employing its proprietary GalXC™ and GalXC-Plus™ RNAi technologies, Dicerna developed RNAi-based therapies to selectively silence genes causing or contributing to disease.
In 2019, Bayer announced a five-year partnership with the World Federation of Hemophilia (WFH) Humanitarian Aid Program to bring education, training, and access to Bayer’s recombinant factor VIII (rFVIII) therapies to healthcare providers in over 60 countries.
Market Analysis by Disease Type, Drug Class, and Region:
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