“We used to think that our fate was in our stars, but now we know that, in large measure, our fate is in our genes, “quotes James Watson.
This fate that Watson is talking about is contained in our genes, and deals with a new technique, GENE THERAPY.
Gene therapy is an innovative way to fix genetic problem from the source. By adding a corrected copy of a defective gene, gene therapy promises to help diseased tissues and organs work properly. This approach is different from traditional drug-based approaches, which may treat symptoms but not the underlying genetic problems.
The treatment sounds magical, but the complexity of designing and delivering arises. The safety issues slowed the progress, following events like death of Jess Gelsinger, who died in 1999 just weeks after receiving an experimental gene therapy in a clinical trial being conducted by the University of Pennsylvania.
Although gene therapy is a promising treatment option for a number of diseases, the technique remains risky and is still under study to make sure that it will be safe and effective.
Also some genetic disorders are rare in nature affecting 1 in million. For such patients, Gene therapy can be a miracle but high cost of developing a treatment can make it unappealing for the pharmaceutical companies. With a limited number of patients to recover those expenses from, developers may never earn money from treating such rare genetic disorders. And some patients may never be able to afford them.
The first milestone in gene therapy, Gendicine, developed by Shenzhen SiBiono GeneTech was approved in China in 2003 for the treatment of head and neck cancers and the biggest watershed moment was Glybera, a gene therapy for lipoprotein lipase deficiency, launched in Germany at a price of more than 1 million euros ($1.1 million).
Meanwhile, companies like bluebird bio and Spark Therapeutics generated impressive clinical data with programmes of their own.
The success has generated renewed interest from both private and public equity investors as well as pharmaceutical companies, such as Bayer and Sanofi, which have willingly provided much-needed resources to companies working in the field in the form of financing and partnerships, helping to drive what could turn out to be a virtuous circle of success.
DelveInsight Gene Therapy Reports
DelveInsight’s Reports have already established a reputation of offering the affordable and comprehensive industry coverage and “on-the-ground” analysis in virtually every region of the world. These reports provide complete information for over 300 gene therapies which are in the pipeline for various therapy areas like; Oncology, Genitourinary, Dermatology, Central nervous system, Genetic Disorders, Hematological disorders, Metabolic disorders, Ophthalmology, Cardiovascular disease, Respiratory , Immunology, and many more…
DelveInsight’s Gene Therapy Reports cover the entire gene therapy market scenario including technology assessments, licensing opportunities, collaborations, market trends, pipeline coverage and competitive landscape. The report essentially provides DelveInsight’s proprietary market and pipeline analytics which identifies the front runners of all therapeutic areas. It also identifies the potential market movers and future regulatory landscape. These reports are designed to provide the clients with the means to out produce their competitors by developing a product that makes history.
For more info on Gene Therapy Reports for various Therapeutic areas contact us at: email@example.com.
By Rashi Aggarwal, Associate Analyst, DelveInsight
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The Haemophilia Market is driven by clotting factor concentrates. Besides hemophilia A and B, and those with von Willebrand disease (VWD), is also sometimes treated with clotting factor.
Currently about 20,000 people have hemophilia in the US; about 80% of them have hemophilia A, and 3,000 to 4,000 have hemophilia B. About 400 US babies with hemophilia are born each year. Von Willebrand disease is the most common bleeding disorder, affecting about 1% of the population.
In the Haemophilia world; diseases are cured by factors like Hemophilia A patients use factor VIII (FVIII), Hemophilia B patients use factor IX (FIX) and VWD patients use factor VIII with VWF (VWF/FVIII complex).
The Recombinant hemophilia market is set to take over the market place with the long acting treatments in the major markets; the US, France, Germany, Italy, Spain, UK and Japan by the beginning in the US in 2014 and the European countries (5EU) in 2015. The launch of Biogen Idec and Sobi’s long-acting therapies for hemophilia A and B will affect the position of Baxter, Bayer and Pfizer’s market- leading therapies. These long acting therapies will give a significant increase in sales to the hemophilia market.
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Compare the global sales of 2013 and 2012 of the top ten blockbuster drugs which is losing patent this year. Teva Pharmaceutical’s Copaxone is the most affected drugs in terms of sales as the sales of 2013 has increased to 4.328 billions bearing the highest loss. Least affected Drug is AbbVie’s Trilipix which has faced the decrease in sales from 1.098 billions to 303 millions.
Table: Global Sales of Blockbuster Drugs for 2012 and 2013
|Brand Name||Global Sales 2012 USD (b)||Global Sales 2013