Global Hemophilia Gene Therapy Market, by Hemophilia Type (Hemophilia A and Hemophilia B), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) is estimated to be valued at US$ 330.9 Million in 2022 and is expected to exhibit a CAGR of 43.6% during the forecast period (2022-2030), as highlighted in a new report published by Coherent Market Insights.
Key players in the market are focusing on adoption of inorganic growth strategies such as acquisition for development of the cell and gene therapy to treat hemophilia. For instance, in October 2020, Bayer, Pharmaceutics Company and acquired Asklepios (Asklepios Kliniken), German Hospitals Company, to broaden innovation base in cell and gene therapy to treat hemophilia. Acquisition fuels Bayer’s cell and gene therapy platform with potential to bring urgently needed treatments to patients across multiple disease areas with high unmet need / AskBio's industry leading Adeno-associated virus (AAV) AAV-based gene therapy platform to treat Hemophilia.
Global Hemophilia Gene Therapy Market– Impact of Coronavirus (COVID-19) Pandemic
The COVID-19 pandemic and lockdowns in various countries across the globe have impacted the financial status of businesses across all sectors, including the private healthcare sector. The COVID-19 pandemic has impacted the entire supply chain of the healthcare industry, mainly due to strict lockdown in several regions. Private healthcare is one such sector that has been impacted significantly by the COVID-19 pandemic.
However, the COVID-19 pandemic had a negative impact on the global hemophilia gene therapy market, owing to disruption of supply chain of drugs due to Covid 19 pandemic. For instance, in September 2021, according to the National Center for Biotechnology Information, in response to the coronavirus disease 2019 (COVID-19) pandemic, many countries in the WHO South-East Asia Region began implementing lockdowns and other measures in March 2020. India’s lockdown began at midnight on 24 March 2020, initially for a period of 3 weeks, followed by multiple extensions. Due to the lockdown imposed by the government of different countries the supply of drugs was hampered. Ultimately, the effects of the COVID-19 crisis on the markets for opiates and synthetic drugs will depend on the intensity of the restrictive measures and their duration. The availability of precursor substances required in the manufacturing of synthetic drugs are mostly trafficked within the region and through unofficial border crossings.
Global Hemophilia Gene Therapy Market: Key Developments
In May 2022, BioMarin Pharmaceutical Inc., Biotechnology company, updated results from its ongoing open-label Phase 1/2 study, which represents the longest duration of clinical observation for valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. The company plans to share the data during an oral presentation at the upcoming International Society on Thrombosis and Haemostasis (ISTH) 2022 Congress (July 9-13). Six-year and five-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts, respectively, demonstrated sustained hemostatic efficacy of valoctocogene roxaparvovec.
In February 2022, Jiangsu Gensciences lnc., biopharmaceutical Company, develop drugs that focus on underserved illnesses like hemophilia, metabolic diseases, and anti-tumor fields carried study on ADVATE. ADVATE is a medicine used to replace clotting factor (factor VIII or antihemophilic factor) that is missing in people with hemophilia A (also called “classic” hemophilia). The study completion date is estimated to be on September 2022.
Browse 16 Market Data Tables and 21 Figures spread through 110 Pages and in-depth TOC on “Global Hemophilia Gene Therapy Market”- Forecast to 2030, Global Hemophilia Gene Therapy Market, by Hemophilia Type (Hemophilia A and Hemophilia B), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa)
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Moreover, increasing research and development activities for development of the treatment of Hemophilia is expected to drive the global hemophilia gene therapy market. For instance, in April 2021, Zhengzhou Gensciences Inc, Biopharmaceutical Company in collaboration with Jiangsu Gensciences lnc., Biopharmaceutical Company, carried out clinical trial study for FRSW107. The study focused on evaluation of efficacy and safety of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in adolescent and adult patients with hemophilia A.
Key Takeaways of the Global Hemophilia Gene Therapy Market:
- The global hemophilia gene therapy market is expected to exhibit a CAGR of 43.6% during the forecast period due to the increasing adoption of inorganic strategies such as acquisition to develop treatment for Hemophilia. For instance, in August 2017, REGENXBIO Inc., Biotechnology Company and Dimension Therapeutics, Inc., Biotechnology Company, entered into a definitive agreement under which REGENXBIO will acquire Dimension in an all-stock transaction for an implied value of approximately US$ 3.41 per share. The boards of directors of both companies have unanimously approved the transaction. In addition, REGENXBIO will acquire DTX201 for the treatment of hemophilia A. DTX201 is designed to use REGENXBIO’s NAV Technology to deliver a copy of the Factor VIII gene to liver cells, and is partnered through a global development and commercialization collaboration with Bayer.
- Among regions, North America is estimated to account for the largest market share in the global hemophilia gene therapy market over the forecast period, owing to the increasing prevalence of the Hemophilia in that region. For instance, in September 2020, according to the data published by the National Center for Biotechnology Information, the estimated prevalence of hemophilia in the U.S. is 12 cases per 100,000 U.S. males for hemophilia A and 3.7 cases per 100,000 U.S. males for hemophilia B.
- Major players operating in the global hemophilia gene therapy market include BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC, Sangamo Therapeutics, Inc., and Freeline Therapeutics
