Haemophilia Gene Therapy Market Size to Reach USD 5165.26 Million by 2030 – The Rising Prevalence of Haemophilia to Propel Growth: The Brainy Insights

The pooling of resources rendered by big pharmaceutical giants, biotechnology companies and government institutions with increased government funding towards medicine has all opened up newer opportunities in the haemophilia gene therapy market. The North American region emerged as the largest global haemophilia gene therapy market, with a 43.31% market revenue share in 2022.

Newark, March 13, 2023 (GLOBE NEWSWIRE) — The Brainy Insights estimates that the USD 350 million in 2022 haemophilia gene therapy market will reach USD 5165.26 million by 2030. Modern medicine is highly advanced, given the extensive knowledge that the world has now acquired about life sciences over the past few years. Earlier, the medical community had to deal with diseases that affected large-scale populations or caused pandemics. Influenza, smallpox, coronaviruses and other diseases/infections were the priority. As healthcare improved globally, such diseases have become endemic, and the deaths from these diseases have decreased substantially. Now the focus has turned to treating or improving patient outcomes in chronic and rare diseases. Healthcare has modernized with automation, digitization and technological advancements worldwide. The emphasis on offering the optimum healthcare to every patient has paved the way for novel medicines, therapies and treatments. The increasing research and development in genetics have opened up promising and possible treatments that can improve patient outcomes. Government and private players are collaborating to invest more in researching genetic engineering and editing. Therefore, the promising trials with several gene therapies have prompted more government and private investment into the same, thereby contributing to the global haemophilia gene therapy market.

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Key Insight of the haemophilia gene therapy Market

North America to account for the largest market size during the forecast period.

North America is expected to have the largest market share in the haemophilia gene therapy market during the forecast period. One of the main factors influencing the market is the existence of a sizable population with haemophilia. Early detection of haemophilia is made possible by the established healthcare system’s sophisticated and comprehensive diagnostics network, which also permits speedy access to further therapies. The condition’s increasing incidence in middle-aged people will also help the market expand. A key secondary factor supporting early diagnosis and treatment of infant haemophilia is well-established paediatric care. Large pharmaceutical and biotechnology businesses are driving gene therapy research and development. Scientific advances made possible by a strong scientific community and a robust research infrastructure have opened the door to using gene treatments to treat various genetic diseases, including haemophilia.

During the forecast period, the haemophilia A segment will augment the haemophilia gene therapy market.

The indication segment is divided into haemophilia A and haemophilia B. The haemophilia A segment dominated the market with a market share of around 75% in 2022. Haemophilia A is a bleeding disorder caused due to the absence of factor VII in the genes of the baby.

Advancement in market

December 2022 – Self-amplifying messenger ribonucleic acid (sa-mRNA) vaccine research, development, production, and international marketing have been completed by CSL Seqirus and Arcturus Therapeutics. According to the agreement, CSL will get access to the sa-mRNA vaccine platform technology of Arcturus. The corporations revealed their strategic alliance in November. CSL agreed to give Arcturus a USD 200 million upfront payment after this contract. Arcturus is entitled to development milestone payments of over USD 1.3 billion and commercial milestone payments of around USD 3 billion. For the treatment of respiratory viral diseases like influenza, COVID-19, and others, CSL will be granted an exclusive licence to the mRNA technology. The business will also receive a non-exclusive licence with the opportunity to become exclusive in multi-pathogen pandemic preparedness. Future product sales will also result in royalty payments or profit sharing for Arcturus.

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Market Dynamics

Driver: increasing haemophilia globally

Even though international healthcare organizations classify haemophilia as an uncommon disease or condition, there has been a worrying increase in the number of haemophilia patients worldwide. All demographic groups, including undeveloped, developed, and developing nations, have seen an equally alarming increase in haemophilia. The current medical developments allow haemophilia, a hereditary disorder, to be managed rather than treated. Haemophilia gene therapy is a transformative procedure that can permanently end patients’ pain and enhance their quality of life. The use of haemophilia gene therapy is projected to increase because of increased healthcare funding and government programmes to provide treatment for rare diseases. Therefore, the increasing prevalence of haemophilia will drive the global haemophilia gene therapy market.

Restraint: expensive haemophilia gene therapy

Understanding the origins of an illness or inherited condition requires substantial investigation. Millions are invested in figuring out the disease and then creating potential treatments. However, not all research or development efforts will inevitably produce fruitful outcomes. Successful ones have invested a significant sum of money. Gene therapy research and development are time- and money-consuming processes. The medical sciences of genetic engineering and gene editing are very new, in the early phases of development, and not widely available. Because of this, there aren’t many players in the field of gene therapy. Therefore, the money invested in research must be recouped to create better treatment alternatives. Due to the small number of market participants and their strong market power, these players can demand higher fees for these medicines. The cost of haemophilia gene therapy will constrain market expansion.

Opportunities: rising research and development expenditure

As technology develops, scientists can delve deeply into genetics and take advantage of its potential to address current healthcare issues. Numerous scientific discoveries and the advancement of scientific methods have produced encouraging results in treating illnesses that were earlier considered incurable. Large pharmaceutical corporations, biotechnology firms, and governmental organizations are working together to develop cutting-edge medications, treatments, and medical equipment. The pooling of resources provided by these partnerships and rising government investment in healthcare have all created more potential in the market for haemophilia gene therapy.

Challenge: lack of sufficient data about haemophilia gene therapy

About 400,000 people worldwide suffer from the rare illness known as haemophilia. The general population is unaware of the condition, its causes, or its symptoms. Given the paucity of information about the disease, the lack of appropriate government messaging, and the inadequate diagnostics infrastructure in most nations, a sizeable section of the patient population, is ignorant of their condition. Additionally, several commercially available medications are used to control the illness; these medications are effective enough for patients to avoid switching to an expensive form of gene therapy. Gene therapy is conveniently more expensive than drugs. Additionally, haemophilia gene therapy has not yet proven to be a significantly effective treatment option, which makes the market’s anticipated growth difficult to predict.

Report Scope

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Some of the major players operating in the haemophilia gene therapy market are:

• BioMarin Pharmaceuticals Inc.
• F Hoffmann-La Roche AG
• Freeline Therapeutics
• Pfizer
• Sangamo Therapeutics Inc.
• Shire PLC
• Spark Therapeutics
• Ultragenyx Pharmaceutical
• uniQure NV
• Zoetis

Key Segments cover in the market:

By Indication

• Haemophilia A
• Haemophilia B

By Region

• North America (U.S., Canada, Mexico)
• Europe (Germany, France, the U.K., Italy, Spain, Rest of Europe)
• Asia-Pacific (China, Japan, India, Rest of APAC)
• South America (Brazil and the Rest of South America)
• The Middle East and Africa (UAE, South Africa, Rest of MEA)

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About the report:

The market is analyzed based on value (USD Million). All the segments have been analyzed worldwide, regional, and country basis. The study includes the analysis of more than 30 countries for each part. The report analyzes driving factors, opportunities, restraints, and challenges for gaining critical insight into the market. The study includes porter’s five forces model, attractiveness analysis, Product analysis, supply, and demand analysis, competitor position grid analysis, distribution, and marketing channels analysis.

About The Brainy Insights:

The Brainy Insights is a market research company, aimed at providing actionable insights through data analytics to companies to improve their business acumen. We have a robust forecasting and estimation model to meet the clients’ objectives of high-quality output within a short span of time. We provide both customized (clients’ specific) and syndicate reports. Our repository of syndicate reports is diverse across all the categories and sub-categories across domains. Our customized solutions are tailored to meet the clients’ requirement whether they are looking to expand or planning to launch a new product in the global market.

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