Hemophilia Gene Therapy Market

Hemophilia Gene Therapy Market Outlook (2022-2032)

Hemophilia is a bleeding disorder usually seen as an inherited disorder in which the blood is unable to clot neatly. These patients are likely to face spontaneous bleeding and may follow injuries. Hemophilia Gene therapy is the introduction of a healthier gene into the body of the patient which replaces the damaged gene. Due to progress and technological advancement, this therapy is soon expected to be a possibility.

Hemophilia is one disease that is expected to be a target for treatment with such therapy. People suffering from hemophilia have difficulty and lack in forming blood clots which brings them at huge risk of uncontrolled bleeding during minor injuries and internal bleeding into muscles and joints.

The global market for hemophilia therapy is at present valued at USD 330.9 Million in 2022. The market is anticipated to reach a market size of USD 12,337 Million by end of the forecast period i.e. by 2032. The market is likely to grow with a CAGR of 43.6% in the forecast period.

Report Attributes	Details
Market size (2022 A)	USD 330.9 Million
Base Year for Estimation	2022
Forecast duration for this research	2022-2032
Forecasted Market value (2032 F)	USD 12,337 Million
Global Market Growth rate (2022-2032 E)	CAGR of 43.6%
Key Companies Profiled	F. Hoffmann-La Roche AG Pfizer Inc. BioMarin Pharmaceuticals uniQure Shire PLC Sangamo Therapeutics

Market dynamics: Drivers and Restraints

Patients suffering from hemophilia A have a mutation for factor VIII in the gene and patients suffering from hemophilia B have a mutation due to the absence of clotting factor IX. The final goal of hemophilia gene therapy is the restoration of a corrected gene for the rest of the life of the patient which is challenging and yet not accomplished. This treatment has a low brink of success.

The investment in research for hemophilia treatment is also due to the need for treating prolonged external and internal bleeding issues. Such bleeding can cause many disorders. This has driven the demand for gene therapy in blood clot treatment. The upcoming period is likely to experience advancement in gene editing which is likely to bring promising results in hemophilia treatments in the forecast period.

There is no approved Hemophilia Gene therapy presently however the treatment involves injections that clot the proteins, these proteins are expensive and cost about 1 million per year. Manufacturers and scientists with continuous Research and development are making efforts for the successful treatment of Hemophilia by Gene therapy.

The hemophilia gene therapy uses viruses as a vector however the problem is the vector is not large enough which can carry all the genes the other problem occurs during the insertion of the vector as the vector is a virus there is a possibility that it can interfere with the immune system and react to it. Moreover, the therapy is more likely to interact with the immune system if the doses are increased. Scientists and the biopharmaceutical industries are trying their best in order to cure Hemophilia A and B through gene therapy.

Segment wise Insights

The market is segmented into Hemophilia type A and type B on the basis of indication. The global market will witness advancement due to the rising population suffering from hemophilia. According to the national institute of hemophilia, approximately 20,000 people in U.S. and 400,000 worldwide are suffering from hemophilia.

The National hemophilia foundation is awarding grants to further support the research for such therapy which will in turn help in the treatment of hemophilia. The biopharmaceutical manufacturers and research centers are working together to understand the genetics of hemophilia Manufacturers such as Roche have recently acquired Spark Therapeutics for its long-term investment hemophilia A gene therapy market. Also, many drugs for hemophilia gene therapy are in clinical trials.

The continuous investment and research by the manufacturers are expected to improve the market in the coming future. Also, therapy assures to address the unmet needs by one-time administration which will further improve its severity. However, the arrival of Hemophilia Gene therapy is a concern over its affordability and accessibility.

Regional Insights

Geographically, the global market is split into regions viz. North America, Latin America, Middle East & Africa, Asia Pacific, Western Europe, and Eastern Europe. North America’s this market is expected to grow because of the evolution and progression in technology and advancements to improve patients’ health.

However, the willingness of payers and the government to arrange funding or insurance coverage for Hemophilia Gene therapy is not well established. If the manufacturers that bring therapy to the market have conventional and older hemophilia therapies within their product portfolio their consideration to offer gene therapy for a low price may lack as the new technology would disrupt their present market.

Recent trends

“The biotech innovation to lead hemophilia treatment”

The therapy intends to use a modified virus that induces the gene copy which encodes the clotting factor which is missing in the patients. The recombinant factor concentrates are used in DNA technology to treat, inactivate or remove the viruses which are blood borne.

Biomarkers and gene editing are key technological inventions that are helping biotech work in hemophilia treatment development. Gene editing is aimed at the replacement of harmful genes with mutated genes. The next generation of gene mutations and sequencing has improved specialization.

Competitive landscape

Genetic research is focused on by leading companies in order to keep a competitive advantage over other firms. The vendor’s capacity to produce promising results is a key factor that determines their market stay and is likely to drive the market demand in the forecast duration.

There are numerous players in the industry that are increasingly tough competition and every firm is trying to keep its dominant position. The well-established companies making large growth and major share in the market. Collaborations, mergers, and acquisitions are commonly adopted strategies by market players.

These tactics help players to explore untapped business expansion capacities in hemophilia treatment. The market exists with many players. Also, partnerships, mergers and acquisitions, different business ventures, and government initiatives are proving to be a boon to the market. Many firms are trying to enhance the success rate.

Hemophilia Gene Therapy Market: Segmentation

• By Indication :

  • Hemophilia A
  • Hemophilia B

• By Region :

  • North America
  • Latin America
  • Europe
  • CIS/Russia
  • Japan
  • Asia-Pacific excluding Japan
  • Middle East & Africa