{"id":6614,"date":"2019-11-27T17:13:54","date_gmt":"2019-11-27T11:43:54","guid":{"rendered":"https:\/\/www.delveinsight.com\/blog\/?p=6614"},"modified":"2023-02-10T12:39:53","modified_gmt":"2023-02-10T07:09:53","slug":"sickle-cell-disease-upcoming-therapy-approach","status":"publish","type":"post","link":"https:\/\/www.delveinsight.com\/blog\/sickle-cell-disease-upcoming-therapy-approach","title":{"rendered":"\ufeffNovel treatment approval promises a better future for Sickle cell disease patients"},"content":{"rendered":"\n

Sickle cell disease is a haematological,\ngenetic disorder that affects millions of people worldwide.<\/strong><\/p>\n\n\n\n

According to the NIH, \u201cSickle\ncell disease is the most common inherited blood disorder in the United States,\naffecting 70,000 to 80,000 Americans\u2019\u2019<\/em>. The estimates are somewhat similar to\nthat of CDC\u2019s according to which SCD afflicts approximately 100,000 Americans.<\/p>\n\n\n\n

Sickle cell disease preponderance<\/strong><\/a> is more or less equal in both the genders. <\/strong><\/p>\n\n\n\n

Interestingly enough, Japan has almost nil prevalence of sickle cell disease.<\/em><\/p>\n\n\n\n

The disease affects 1 in 500\nAfrican Americans and 1 in 1,000 to 1,400 Hispanic Americans. <\/strong><\/p>\n\n\n\n

Sickle cell disease is a result of a single genetic mutation. Characterized by the abnormality in haemoglobin sickle (HbsS), it results in the formation of haemoglobin \u03b2, i.e. polymerization. It is inherited in autosomal recessive form and occurs due to homozygosity for HbS (HbSS). <\/p>\n\n\n\n

Oxbryta<\/strong><\/h4>\n\n\n\n

Sickle cell disease treatment approaches<\/strong> chiefly help in easing the symptoms and are curative in nature. However, recent approvals of therapies like that of Global Blood Therapeutics (GBT), are focusing on treating the disease from its root. <\/p>\n\n\n\n

Oxbryta<\/strong> (Voxelotor), has\nrecently received a green signal from the US FDA, is a tablet taken orally once-a-day.\nIt blocks the polymerization of the mutant haemoglobin thus increasing its\naffinity to oxygen. The new therapy, as revealed by the company, will cost USD\n125,000 annually to the Sickle cell disease patients. <\/p>\n\n\n\n

Other pharma companies across the globe, like GBT Therapeutics<\/strong>, Pfizer<\/strong>, AstraZeneca<\/strong>, Ironwood<\/strong> Pharmaceuticals, Imara Therapeutics<\/strong>, and many others, are involved in developing therapies for Sickle Cell Disease.<\/p>\n\n\n\n

Launch of emerging therapies<\/a>, such as Rivipansel<\/strong> (Pfizer), IMR-687<\/strong> (Imara Therapeutics), Ticagrelor<\/strong> (AstraZeneca), and other compelling treatments, are expected to further increase the Sickle Cell Disease market<\/a> in upcoming years.<\/p>\n\n\n\n

Other novel gene-edited therapies are also under development for permanent cure of Sickle cell disease<\/a>. For instance, Vertex Pharma’s CTX001, an investigational ex vivo CRISPR gene-edited therapy is currently under Phase I\/II stage of development in collaboration with CRISPR Therapeutics. The candidate has already received FDA Fast Track Designation early this year.<\/p>\n\n\n\n

Novel and innovative therapeutic approach<\/a> for treating Sickle Cell Disease, including new medications, advances in transplantation, and gene therapies<\/a> are being explored, which are expected to drive the Sickle cell disease treatment market during the forecast period significantly. <\/p>\n","protected":false},"excerpt":{"rendered":"

Sickle cell disease is a haematological, genetic disorder that affects millions of people worldwide. According to the NIH, \u201cSickle cell disease is the most common inherited blood disorder in the United States, affecting 70,000 to 80,000 Americans\u2019\u2019. The estimates are somewhat similar to that of CDC\u2019s according to which SCD afflicts approximately 100,000 Americans. Sickle […]<\/p>\n","protected":false},"author":1,"featured_media":6620,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_editorskit_title_hidden":false,"_editorskit_reading_time":1,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","advgb_blocks_editor_width":"","advgb_blocks_columns_visual_guide":"","footnotes":""},"categories":[17434,41],"tags":[96,17846,17845,460,536,7102,17843,7101,17844],"industry":[17225],"therapeutic_areas":[17238,17233],"class_list":["post-6614","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-facts-and-figures","category-snippets","tag-astrazeneca","tag-imara-therapeutics","tag-oxbryta","tag-pfizer","tag-sickle-cell-disease","tag-sickle-cell-disease-market","tag-sickle-cell-disease-patients","tag-sickle-cell-disease-therapy","tag-sickle-cell-disease-treatment-approaches","industry-pharmaceutical","therapeutic_areas-genetic-disorders","therapeutic_areas-hematological-disorders"],"acf":[],"yoast_head":"\nSickle Cell Disease \ufeffTreatment | Oxbryta, Rivipansel, IMR-687<\/title>\n<meta name=\"description\" content=\"Sickle cell disease therapy approaches chiefly help in easing the symptoms and are curative in nature. 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