Hemophilia Gene Therapy Market

Hemophilia Gene Therapy Market is valued at USD 331.4 Million in 2021 and expected to reach USD 4092.6 Million by 2028 with a CAGR of 43.2% over the forecast period. 

Global Hemophilia Gene Therapy Market: Global Size, Trends, Competitive, Historical & Forecast Analysis, 2022-2028- Increasing prevalence of haemophilia, rising number of research activities for genetic mutations and growing awareness about haemophilia gene therapy treatments are major factors anticipated to drive the growth of the Global Market. 

Scope– 

Hemophilia is a genetic rare disorder in which blood doesn’t clot in the systematic way because the person affected cannot make enough blood-clotting proteins. As patients cannot stop bleeding after injury as these people missing the proteins. Haemophilia gene therapy is the therapy for these patients in which the clotting factor that’s missing in patients introduced through virus containing a copy of the dna that encodes for the clotting factor. The patients also given treatment with the virus; patients should begin producing their own clotting factor normally. 

The Covid-19 pandemic has also affected the growth of global hemophilia gene therapy market. As the Covid cases increasing, all healthcare and research was focused on Covid-19 and the research activities regarding the other diseases were kept on hold. For instance; as per National Centre of Biotechnology Information (NCBI) there was 63% decrease in medical/nursing clinic consultation activity compared to the same period in 2019 due to public health measures executed in March 2020 to reduce COVID-19 spread. Covid-19 pandemic caused a huge impact on hemophilia healthcare delivery which negatively impacted the market.  

The global hemophilia gene therapy market is segmented into on the basis of type, and region & country level. The regions covered in global market report are North America, Europe, Asia-Pacific, Latin America and Rest of the World. On the basis of country level, the market of global market is subdivided into U.S., Mexico, Canada, U.K., France, Germany, Italy, China, India, Japan, South East Asia, Middle East Asia (UAE, Egypt, Saudi Arabia) GCC, Africa, etc. 

Key Players- 

Some of the key players for global hemophilia gene therapy market are -

• herapeutics
• Animal Health
• Sangamo Therapeutics Inc
• BioMarin Pharmaceuticals Inc.
• Spark Therapeutics
• Pfizer Inc.
• UniQure NV
• Ultragenyx Pharmaceutical
• Shire
• Freeline Therapeutics
• Zoetis
• CSL Limited
• Bioverativ
• Novo Nordisk
• Sanofi S.A.
• others

Segmentation-

By Type

• Haemophilia A 
• Haemophilia B 

By Region Analysis

North America

• U.S.
• Canada

Europe

• Germany
• France
• U.K.
• Italy
• Spain
• Netherland
• Turkey
• Switzerland
• Belgium
• Rest of Europe

Asia-Pacific

• China
• South Korea
• Japan
• India
• Australia
• Philippines
• Singapore
• Malaysia
• Thailand
• Indonesia
• Rest Of APAC

Latin America

• Brazil
• Mexico
• Colombia
• Argentina
• Peru
• Rest of South America

Middle East and Africa

• Saudi Arabia
• UAE
• Egypt
• South Africa
• Rest Of MEA

Increasing Prevalence of Haemophilia, Rising Number of Research Activities in the Field of Genetic Mutations and Growing Awareness about Haemophilia Gene Therapy Treatments are Major Factors Driving the Market Growth  

One of the major factors driving the growth of the hemophilia gene therapy market is increasing prevalence of hemophilia. Increasing number of patients with hemophilia is one of the major factors driving the demand for hemophilia gene therapy. For instance; hemophilia affected above 400,000 people globally, as per National Hemophilia Foundation factsheet 2018. In addition, rising research and strong product pipeline in genetic mutations is also expected to boost the market growth. For instance; updated preliminary data for the ongoing phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A announced by Spark Therapeutics in 2018 and Interventional study for Efmoroctocog Alfa Injection to treat haemophilia A was carried by the League of Clinical Research for Efmoroctocog Alfa Injection to treat haemophilia A.  

Moreover, growing awareness about hemophilia gene therapy is also supplementing the market growth as people are now more about the dna therapies and its benefits. However, side affects caused by plasma derived products may hinder the growth of the market. In spite of that rising number of research and development activities in the field this field may offer more opportunities for the further growth of the global market.  

The North America is Expected to Dominate Global Hemophile Gene Therapy Market 

The North America is expected to dominate the global hemophilia gene therapy due to the presence of major players, increasing number people having hemophilia patients and availability of advanced technologies and growing awareness among the people in this region. For instance; as per Center for Disease Control and Prevention; Hemophilia A affected one in 5,000 male births and 400 babies are born with hemophilia A each year in U.S. In addition, availability of advanced technologies is also boosting the market growth in North America. For instance; updated preliminary data for the ongoing phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A announced by Spark Therapeutics in 2018. Moreover, growing awareness about the disease and treatment for hemophilia such as hemophilia gene therapy through education is also augmenting the growth of the market in this region.  

Asia Pacific is expected to be the second largest region in the global hemophilia gene therapy market due to the improving healthcare facilities, rising awareness about hemophilia and growing investments by government in this region. Healthcare facilities are increasing in developing countries such as India and China which is providing growth to the market. In addition, rising awareness about hemophilia also adding growth to the market. For instance; in 2016 Hemophilia Federation India (HFI) organized an event to spread awareness about the disease and to help patients suffering from this terrible disease.  

News- 

First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN (valoctocogene roxaparvovec), Approved by European Commission (EC) 

On August 22nd, 2022; The European Commission (EC) had granted conditional marketing authorization (CMA) to ROCTAVIAN (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients. This was the first approved gene therapy for hemophilia A. In this treatment functional dna is delivered a functional gene that is designed to allow the body to produce Factor VIII on its own without the need for continued hemophilia prophylaxis.  

Hemophilia B Gene Therapy Shown Promise in Clinical Trial 

On July 22nd, 2022; The safety and efficacy of a liver-directed gene therapy to restore factor IX levels long-term in hemophilia B patients was tested at the University College London (UCL), the Royal Free Hospital, and Freeline Therapeutics. The researchers estimating the adeno-associated virus (AAV) based gene treatment candidate, called FLT180a, for the treatment of patients with severe and moderately severe hemophilia B.  

Report Analysis	Details
Historical data	2018 - 2021
Forecast Period	2021 - 2028
Market Size in 2021:	USD 331.4 Million
Base year considered	2021
Forecast Period CAGR %:	43.2%
Market Size Expected in 2028:	USD 4092.6 Million
Tables, Charts & Figures:	175
Pages	200
Hemophilia Gene Therap Players	herapeutics, Animal Health, Sangamo Therapeutics Inc, BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire, and Freeline Therapeutics, Zoetis, CSL Limited , Bioverativ, Novo Nordisk, Sanofi S.A. and others.
Segments Covered	By Type
Regional Analysis	North America, U.S., Mexico, Canada, Europe, UK, France, Germany, Italy, Asia Pacific, China, Japan, India, Southeast Asia, South America, Brazil, Argentina, Columbia, The Middle East and Africa, GCC, Africa, Rest of the Middle East and Africa