GLOBAL HEMOPHILIA GENE THERAPY MARKET 2019 BY COMPANY, REGIONS, TYPE AND APPLICATION, FORECAST TO 2024

Feb 2019 116

Delivery of the Report will take 2-3 working days once order is placed.

Synopsis

Hemophilia is a rare bleeding disorder in which the blood does not clot normally. Hemophilia is a monogenic disease (a disease that is caused by a genetic defect in a single gene). There are two types of hemophilia caused by mutations in genes that encode protein factors which help the blood clot and stop bleeding when blood vessels are injured. Individuals with hemophilia experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to joint disease such as arthritis. The most frequent forms of hemophilia affect males.

Scope of the Report:
About 80% of them have hemophilia A, which affects the clotting factor VIII. The second most common form, hemophilia B, is due to a deficiency of the clotting factor IX. Several biotechs are racing to launch the first gene therapy for hemophilia. Currently, uniQure in the Netherlands and Spark Therapeutics in the US have the most advanced programs. Spark scored a victory in December when it presented Phase I/II for its candidate SPK-9001. The gene therapy was able to reduce annual bleeding episodes by 97%, as compared to its competitor uniQure’s candidate, AMT-060. However, uniQure has fought back. The company added a modification in its gene therapy that is known to increase clotting activity by 8- to 9- fold. This improved version has already been cleared to start a Phase III clinical trial in both Europe and the US.
Hemophilia B has traditionally been the main focus of biotechs, since most big pharma efforts were focused on the bigger hemophilia A market. In addition, applying gene therapy to hemophilia A is more challenging; The gene coding for the factor IX protein missing in hemophilia B is simply smaller than that for factor VIII missing in hemophilia A, and therefore easier to fit in the viral vectors used for gene delivery. US-based BioMarin is leading the development of a gene therapy. To overcome the size limit, the company has deleted a region from the factor VIII protein that is not necessary for clotting.
Hemophilia treatment is currently in the pre-clinical stage. And the multiple treatments that are underway might significantly improve the quality of life of patients with hemophilia, by getting rid of frequent infusions and hospital visits, and transitioning patients from severe to mild hemophilia.
The global Hemophilia Gene Therapy market is valued at xx million USD in 2018 and is expected to reach xx million USD by the end of 2024, growing at a CAGR of xx% between 2019 and 2024.
The Asia-Pacific will occupy for more market share in following years, especially in China, also fast growing India and Southeast Asia regions.
North America, especially The United States, will still play an important role which cannot be ignored. Any changes from United States might affect the development trend of Hemophilia Gene Therapy.
Europe also play important roles in global market, with market size of xx million USD in 2019 and will be xx million USD in 2024, with a CAGR of xx%.
This report studies the Hemophilia Gene Therapy market status and outlook of Global and major regions, from angles of players, countries, product types and end industries; this report analyzes the top players in global market, and splits the Hemophilia Gene Therapy market by product type and applications/end industries.

Market Segment by Companies, this report covers
- Spark Therapeutics
- Ultragenyx
- Shire PLC
- Sangamo Therapeutics
- Bioverativ
- BioMarin
- uniQure
- Freeline Therapeutics

Market Segment by Regions, regional analysis covers
North America (United States, Canada and Mexico)
Europe (Germany, France, UK, Russia and Italy)
Asia-Pacific (China, Japan, Korea, India and Southeast Asia)
South America (Brazil, Argentina, Colombia)
Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Market Segment by Type, covers
- Hemophilia A
- Hemophilia B

Market Segment by Applications, can be divided into
- Hemophilia A Gene Therapy
- Hemophilia B Gene Therapy

Table of Contents

Related reports :

(Jul 19)

Autologous cell therapy refers to the injection or delivery of human tissue or cells to the same person to treat pathological conditions. Fo...

(Jul 19)

Real-time PCR or quantitative PCR is a well-established technology that has become the tool of choice for the rapid, sensitive quantificatio...

(Jul 19)

Interferons (IFNs) are a group of signaling proteins made and released by host cells in response to the presence of several pathogens, such ...

(Jun 19)

Scope of the Report: The global Cryotherapy market is valued at xx million USD in 2018 and is expected to reach xx million USD by the...

(Jun 19)

Silage inoculants are additives containing anaerobic lactic acid bacteria (LAB) that are used to manipulate and enhance fermentation in hayl...

(Jun 19)

Scope of the Report: The worldwide market for Plasmid is expected to grow at a CAGR of roughly over the next five years, will reach m...

GLOBAL HEMOPHILIA GENE THERAPY MARKET 2019 BY COMPANY, REGIONS, TYPE AND APPLICATION, FORECAST TO 2024

Global Info Research

Share this report on:

Subscription Option


Using our subscription option, you get access to Market Research Reports and industry data of Biotechnology market as per your needs. Get the best of Biotechnology research reports by utilizing your research budgets in an optimum way. Contact for details

Contact for reports

*

Avail 15% Discount, Use JULY15 Coupon Code

We use cookies to deliver the best possible experience on our website.
By continuing to use this site, or closing this box, you consent to our use of cookies. To learn more, visit our Privacy Policy