Press Release
Press Release
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.
Read more about this report - REQUEST FREE SAMPLE COPY IN PDF
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.
Expert Market Research (EMR) is a leading market research and business intelligence company, ensuring its clients remain at the vanguard of their industries by providing them with exhaustive and actionable market data through its syndicated and custom market reports, covering over 15 major industry domains. The company's expansive and ever-growing database of reports, which are constantly updated, includes reports from industry verticals like chemicals and materials, food and beverages, energy and mining, technology and media, consumer goods, pharmaceuticals, agriculture, and packaging.
EMR leverages its state-of-the-art technological and analytical tools, along with the expertise of its highly skilled team of over 100 analysts and more than 3000 consultants, to help its clients, ranging from Fortune 1000 companies to small and medium-sized enterprises, easily grasp the expansive industry data and help them in formulating market and business strategies, which ensure that they remain ahead of the curve.
Expert Market Research
Website: www.expertmarketresearch.com
Email: sales@expertmarketresearch.com
US & Canada Phone no: +1-415-325-5166
UK Phone no: +44-702-402-5790
*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR report.
Right People
We are technically excellent, strategic, practical, experienced and efficient; our analysts are hand-picked based on having the right attributes to work successfully and execute projects based on your expectations.
Right Methodology
We leverage our cutting-edge technology, our access to trusted databases, and our knowledge of the current models used in the market to deliver you research solutions that are tailored to your needs and put you ahead of the curve.
Right Price
We deliver in-depth and superior quality research in prices that are reasonable, unmatchable, and shows our understanding of your resource structure. We, additionally, offer attractive discounts on our upcoming reports.
Right Support
Our team of expert analysts are at your beck and call to deliver you optimum results that are customised to meet your precise needs within the specified timeframe and help you form a better understanding of the industry.