{"id":49964,"date":"2022-10-24T11:01:59","date_gmt":"2022-10-24T09:01:59","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/sandhoff-disease-drug-research-report-2022-comprehensive-insights-about-5-companies-and-5-pipeline-drugs-featuring-intrabio-sanofi-polaryx-therapeutics-azafaros-researchandmarkets-com\/"},"modified":"2022-10-24T11:01:59","modified_gmt":"2022-10-24T09:01:59","slug":"sandhoff-disease-drug-research-report-2022-comprehensive-insights-about-5-companies-and-5-pipeline-drugs-featuring-intrabio-sanofi-polaryx-therapeutics-azafaros-researchandmarkets-com","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/sandhoff-disease-drug-research-report-2022-comprehensive-insights-about-5-companies-and-5-pipeline-drugs-featuring-intrabio-sanofi-polaryx-therapeutics-azafaros-researchandmarkets-com\/","title":{"rendered":"Sandhoff Disease Drug Research Report 2022: Comprehensive Insights About 5+ Companies and 5+ Pipeline Drugs Featuring IntraBio, Sanofi, Polaryx Therapeutics, &amp; Azafaros &#8211; ResearchAndMarkets.com"},"content":{"rendered":"<div>\n<p>DUBLIN&#8211;(BUSINESS WIRE)&#8211;The <a target=\"_blank\" href=\"https:\/\/www.researchandmarkets.com\/reports\/5456992\/sandhoff-disease-pipeline-insight-2022?utm_source=BW&amp;utm_medium=PressRelease&amp;utm_code=dsvl5w&amp;utm_campaign=1760352+-+Sandhoff+Disease+Drug+Research+Report+2022%3A+Comprehensive+Insights+About+5%2B+Companies+and+5%2B+Pipeline+Drugs+Featuring+IntraBio%2C+Sanofi%2C+Polaryx+Therapeutics%2C+%26+Azafaros&amp;utm_exec=chdo54prd\" rel=\"nofollow noopener\" shape=\"rect\">&#8220;Sandhoff disease &#8211; Pipeline Insight, 2022&#8221;<\/a> drug pipelines has been added to <strong>ResearchAndMarkets.com&#8217;s<\/strong> offering.\n<\/p>\n<p><a href=\"https:\/\/mms.businesswire.com\/media\/20221024005431\/en\/1611078\/4\/logo.jpg\"><img decoding=\"async\" src=\"https:\/\/mms.businesswire.com\/media\/20221024005431\/en\/1611078\/21\/logo.jpg\"><\/a><\/p>\n<p>\nThe &#8220;Sandhoff disease &#8211; Pipeline Insight, 2022,&#8221; report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Sandhoff disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.\n<\/p>\n<p>\nThe assessment part of the report embraces, in depth Sandhoff disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Sandhoff disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.\n<\/p>\n<p>\n<strong>Report Highlights<\/strong>\n<\/p>\n<p>\nThe companies and academics are working to assess challenges and seek opportunities that could influence Sandhoff disease R&amp;D. The therapies under development are focused on novel approaches to treat\/improve Sandhoff disease.\n<\/p>\n<p>\n<strong>Sandhoff disease Emerging Drugs Chapters<\/strong>\n<\/p>\n<p>\nThis segment of the Sandhoff disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.\n<\/p>\n<p>\n<strong>Sandhoff disease Emerging Drugs<\/strong>\n<\/p>\n<p>\n<strong>IB1001: IntraBio<\/strong>\n<\/p>\n<p>\nIB1001, N-acetyl-L-leucine, is an orally administered modified amino acid. In vivo studies have identified N-acetyl-L-leucine to be the active isomer of N-acetyl-DL-leucine and can restore neuronal function and protect against\/delay disease progression in multiple neurological circuits of the brain.\n<\/p>\n<p>\nThe mechanism of N-acetyl-L-leucine is known to be multi-modal, including altered glucose and antioxidant metabolism, reduced lysosomal storage, and the reduction of neuroinflammation in the cerebellum, leading to the attenuation of cell death. The company is conducting a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).\n<\/p>\n<p>\n<strong>Venglustat: Sanofi<\/strong>\n<\/p>\n<p>\nVenglustat is a novel, oral investigational therapy that has the potential to slow the progression of certain diseases by inhibiting abnormal glycosphingolipid (GSL) accumulation. GSLs are cellular building blocks whose abnormal accumulation is implicated in several rare diseases, responsible for both cell dysfunction and disease progression. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Sandhoff Disease.\n<\/p>\n<p>\n<strong>Sandhoff disease: Therapeutic Assessment<\/strong>\n<\/p>\n<p>\nThis segment of the report provides insights about the different Sandhoff disease drugs segregated based on following parameters that define the scope of the report, such as:\n<\/p>\n<p>\n<strong>Major Players in Sandhoff disease<\/strong>\n<\/p>\n<p>\nThere are approx. 5+ key companies which are developing the therapies for Sandhoff disease. The companies which have their Sandhoff disease drug candidates in the most advanced stage, i.e. phase III include, Sanofi.\n<\/p>\n<p>\n<strong>Phases <\/strong>\n<\/p>\n<p>\n<strong>The report covers around 5+ products under different phases of clinical development like<\/strong>\n<\/p>\n<ul>\n<li>\nLate stage products (Phase III)\n<\/li>\n<li>\nMid-stage products (Phase II)\n<\/li>\n<li>\nEarly-stage product (Phase I) along with the details of\n<\/li>\n<li>\nPre-clinical and Discovery stage candidates\n<\/li>\n<li>\nDiscontinued &amp; Inactive candidates\n<\/li>\n<li>\nRoute of Administration\n<\/li>\n<\/ul>\n<p>\n<strong>Products have been categorized under various ROAs such as<\/strong>\n<\/p>\n<ul>\n<li>\nOral\n<\/li>\n<li>\nParenteral\n<\/li>\n<li>\nintravenous\n<\/li>\n<li>\nSubcutaneous\n<\/li>\n<li>\nTopical.\n<\/li>\n<li>\nMolecule Type\n<\/li>\n<\/ul>\n<p>\n<strong>Products have been categorized under various Molecule types such as<\/strong>\n<\/p>\n<ul>\n<li>\nMonoclonal Antibody\n<\/li>\n<li>\nPeptides\n<\/li>\n<li>\nPolymer\n<\/li>\n<li>\nSmall molecule\n<\/li>\n<li>\nGene therapy\n<\/li>\n<li>\nProduct Type\n<\/li>\n<\/ul>\n<p>\n<strong>Sandhoff disease Report Insights<\/strong>\n<\/p>\n<ul>\n<li>\nSandhoff disease Pipeline Analysis\n<\/li>\n<li>\nTherapeutic Assessment\n<\/li>\n<li>\nUnmet Needs\n<\/li>\n<li>\nImpact of Drugs\n<\/li>\n<\/ul>\n<p>\n<strong>Sandhoff disease Report Assessment<\/strong>\n<\/p>\n<ul>\n<li>\nPipeline Product Profiles\n<\/li>\n<li>\nTherapeutic Assessment\n<\/li>\n<li>\nPipeline Assessment\n<\/li>\n<li>\nInactive drugs assessment\n<\/li>\n<li>\nUnmet Needs\n<\/li>\n<\/ul>\n<p>\n<strong>Key Players<\/strong>\n<\/p>\n<ul>\n<li>\nIntraBio\n<\/li>\n<li>\nSanofi\n<\/li>\n<li>\nPolaryx Therapeutics\n<\/li>\n<li>\nAzafaros\n<\/li>\n<\/ul>\n<p>\n<strong>Key Products<\/strong>\n<\/p>\n<ul>\n<li>\nIB1001\n<\/li>\n<li>\nVenglustat\n<\/li>\n<li>\nPLX 300\n<\/li>\n<li>\nPLX-200\n<\/li>\n<li>\nAZ 3102\n<\/li>\n<\/ul>\n<p>\nFor more information about this drug pipelines report visit <a target=\"_blank\" href=\"https:\/\/www.researchandmarkets.com\/reports\/5456992\/sandhoff-disease-pipeline-insight-2022?utm_source=BW&amp;utm_medium=PressRelease&amp;utm_code=dsvl5w&amp;utm_campaign=1760352+-+Sandhoff+Disease+Drug+Research+Report+2022%3A+Comprehensive+Insights+About+5%2B+Companies+and+5%2B+Pipeline+Drugs+Featuring+IntraBio%2C+Sanofi%2C+Polaryx+Therapeutics%2C+%26+Azafaros&amp;utm_exec=chdo54prd\" rel=\"nofollow noopener\" shape=\"rect\">https:\/\/www.researchandmarkets.com\/r\/hukomz<\/a>\n<\/p>\n<p> <b>Contacts<\/b> <\/p>\n<p>\nResearchAndMarkets.com<br \/>\n<br \/>Laura Wood, Senior Press Manager<br \/>\n<br \/><a target=\"_blank\" href=\"ma&#105;&#108;&#116;&#x6f;&#x3a;&#x70;&#x72;es&#115;&#64;&#114;&#x65;&#x73;&#x65;&#x61;rc&#104;&#97;&#110;&#x64;&#x6d;&#x61;&#x72;ke&#116;&#115;&#46;&#x63;&#x6f;&#x6d;\" rel=\"nofollow noopener\" shape=\"rect\">&#x70;&#x72;&#101;&#115;&#115;&#64;&#x72;&#x65;&#x73;&#101;&#97;rc&#x68;&#x61;&#x6e;&#100;&#109;a&#x72;&#x6b;&#x65;&#116;&#115;&#46;c&#x6f;&#x6d;<\/a><\/p>\n<p>For E.S.T Office Hours Call 1-917-300-0470<br \/>\n<br \/>For U.S.\/ CAN Toll Free Call 1-800-526-8630<br \/>\n<br \/>For GMT Office Hours Call +353-1-416-8900\n<\/p>\n<\/div>\n","protected":false},"excerpt":{"rendered":"<p>DUBLIN&#8211;(BUSINESS WIRE)&#8211;The &#8220;Sandhoff disease &#8211; Pipeline Insight, 2022&#8221; drug pipelines has been added to ResearchAndMarkets.com&#8217;s offering. The &#8220;Sandhoff disease &#8211; Pipeline Insight, 2022,&#8221; report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Sandhoff disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the &#8230; <span class=\"more\"><a class=\"more-link\" href=\"https:\/\/pharma-trend.com\/en\/sandhoff-disease-drug-research-report-2022-comprehensive-insights-about-5-companies-and-5-pipeline-drugs-featuring-intrabio-sanofi-polaryx-therapeutics-azafaros-researchandmarkets-com\/\">[Read more&#8230;]<\/a><\/span><\/p>\n","protected":false},"author":4,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[13],"tags":[],"class_list":{"0":"entry","1":"post","2":"publish","3":"author-business","4":"post-49964","6":"format-standard","7":"category-industry"},"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.5 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Sandhoff Disease Drug Research Report 2022: Comprehensive Insights About 5+ Companies and 5+ Pipeline Drugs Featuring IntraBio, Sanofi, Polaryx Therapeutics, &amp; Azafaros - ResearchAndMarkets.com - Pharma Trend<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/pharma-trend.com\/en\/sandhoff-disease-drug-research-report-2022-comprehensive-insights-about-5-companies-and-5-pipeline-drugs-featuring-intrabio-sanofi-polaryx-therapeutics-azafaros-researchandmarkets-com\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Sandhoff Disease Drug Research Report 2022: Comprehensive Insights About 5+ Companies and 5+ Pipeline Drugs Featuring IntraBio, Sanofi, Polaryx Therapeutics, &amp; Azafaros - ResearchAndMarkets.com - Pharma Trend\" \/>\n<meta property=\"og:description\" content=\"DUBLIN&#8211;(BUSINESS WIRE)&#8211;The &#8220;Sandhoff disease &#8211; Pipeline Insight, 2022&#8221; drug pipelines has been added to ResearchAndMarkets.com&#8217;s offering. The &#8220;Sandhoff disease &#8211; Pipeline Insight, 2022,&#8221; report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Sandhoff disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the ... 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