Sickle cell disease is a genetic disorder and is the most common inherited genetic disorder. People with the sickle cell disease have abnormal shaped red blood cells that get blocked in the small blood vessels. This leads to a restricted flow of blood and oxygen in the body. Sickle cell disease is potentially a fatal disease as a patient may undergo vital organ failure, painful crisis, and other complications.
Sickle cell disease is broadly categorized as sickle cell disease, sickle cell disorder, and sickle cell anemia. The sickle cell disorder includes patient with positive sickle preparation smear. Sickle cell disease is where the patient faces serious morbidity such as vas-occlusive crises or organ failure. Sickle cell anemia is for patients who are homozygous for hemoglobin S.
Sickle Cell Disease Market: Dynamics
The main driver driving the sickle cell treatment market is the increasing number of new molecules in the pipeline and also the molecules which the government regulatory bodies are approving. Sickle cell disease treatment has given a new area of expansion for market players already in the sickle cell treatment market and for those who are trying to establish a new area in this market.
Modus therapeutics has been granted two U.S. patents for its drug Sevuparin, for the treatment of sickle cell disease. The molecule has been granted a patent for its manufacturing process and other for the treatment of sickle cell disease.
Moreover, researchers have moved ahead of treating sickle cell disease by antibiotics to the genome level. Researchers from Stanford School of Medicine successfully corrected the genes carrying the gene for sickle cell disease and transplanted those with healthy cells. By using the Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) technique, researchers replaced the sickle cell disease cell genes with the one which did not carry that gene.
The prenatal diagnosis using CVS has opened a window of opportunity for a couple expecting their child. The test gives information about the hemoglobin genotype of the unborn child. CVS and amniocentesis are both invasive procedure used for the identification of the presence of sickle cell disease DNA.
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Sickle Cell Disease Market: Region-wise Outlook
North America dominated the sickle cell disease treatment market owing to the various insurance coverage policies and also the research undertakings by institutes and sickle cell treatment industry players. Medicare recently announced that it will cover most of the clinical trials which use stem cell transplantation for the treatment of sickle cell disease. Before the Medicare’s decision, adult patients found it difficult to use the chemo-free treatment as it was very expensive.
Apart from government support, the amount of approved treatment options for sickle cell disease is also increasing. The FDA recently accepted Emmaus Life Science NDA review for L-glutamine. Once accepted, this would be the first orally administered treatment option for children with sickle cell disease.
Asia-Pacific is the fastest growing region as various industry players funding for the stem cell research and also researchers bringing in new technologies for DNA analysis. Infosys donated
USD 1.11 million for stem cell research in India. The donation would be used for developing gene therapies for disorders such as sickle cell disease and hemophilia.
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Sickle Cell Disease Market: Market Players
The company already existing in the sickle cell disease treatment market are boosting their hematology pipeline by acquiring startups. Novartis is one such company who recently acquired Selexys Pharmaceuticals for USD 665 million. The deal is expected to help Novartis expand its product offerings as blood disease have high prices due to increasing demand.
Some of the sickle cell disease treatment providers are Gamida Cell, Biogen, Genetix Pharmaceuticals Inc., Alnylam Pharmaceuticals, Inc., Sangamo Biosciences, Acceleron Pharma, Global Blood Therapeutics Inc., Anthera, Mast Therapeutics, Inc. and Emmaus Medical, Inc.
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