{"id":10682,"date":"2020-09-03T18:13:58","date_gmt":"2020-09-03T12:43:58","guid":{"rendered":"https:\/\/www.delveinsight.com\/blog\/?p=10682"},"modified":"2021-07-24T12:58:05","modified_gmt":"2021-07-24T07:28:05","slug":"recent-pharma-happenings-for-amylyx-orca-lyell-ionis","status":"publish","type":"post","link":"https:\/\/www.delveinsight.com\/blog\/recent-pharma-happenings-for-amylyx-orca-lyell-ionis","title":{"rendered":"Amylyx&#8217;s neuron-protecting drug slows ALS decline; Antisense drugs could help fight ALS and dementia; CAR-T research updates; Orca and Lyell announces research partnership"},"content":{"rendered":"<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_76 counter-hierarchy ez-toc-counter ez-toc-white ez-toc-container-direction\">\n<p class=\"ez-toc-title\" style=\"cursor:inherit\">Table of Contents<\/p>\n<label for=\"ez-toc-cssicon-toggle-item-69f1b53595a91\" class=\"ez-toc-cssicon-toggle-label\"><span class=\"\"><span class=\"eztoc-hide\" style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #999;color:#999\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #999;color:#999\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 .5-.1.7-.3.2-.2.3-.5.3-.7s-.1-.5-.3-.7zM5.8 14.7l6.2 6.3 6.2-6.3c.2-.2.3-.5.3-.7s-.1-.5-.3-.7c-.2-.2-.4-.3-.7-.3h-11c-.3 0-.5.1-.7.3-.2.2-.3.5-.3.7s.1.5.3.7z\"\/><\/svg><\/span><\/span><\/label><input type=\"checkbox\"  id=\"ez-toc-cssicon-toggle-item-69f1b53595a91\"  aria-label=\"Toggle\" \/><nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/www.delveinsight.com\/blog\/recent-pharma-happenings-for-amylyx-orca-lyell-ionis\/#Amylyxs_neuron-protecting_drug_slows_ALS_decline_in_the_late-phase_study\" >Amylyx&#8217;s neuron-protecting drug slows ALS decline in the late-phase study<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/www.delveinsight.com\/blog\/recent-pharma-happenings-for-amylyx-orca-lyell-ionis\/#The_antisense_drugs_of_Ionis_could_help_fight_ALS_and_dementia\" >The antisense drugs of Ionis could help fight ALS and dementia<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/www.delveinsight.com\/blog\/recent-pharma-happenings-for-amylyx-orca-lyell-ionis\/#Virus_combo_makes_solid_tumors_vulnerable_to_CAR-T_attack\" >Virus combo makes solid tumors vulnerable to CAR-T attack&nbsp;<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/www.delveinsight.com\/blog\/recent-pharma-happenings-for-amylyx-orca-lyell-ionis\/#Orca_Bio_Lyell_Immunopharma_pen_new_deal_to_open_up_CAR-T_for_solid_tumors\" >Orca Bio, Lyell Immunopharma pen new deal to open up CAR-T for solid tumors<\/a><\/li><\/ul><\/nav><\/div>\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Amylyxs_neuron-protecting_drug_slows_ALS_decline_in_the_late-phase_study\"><\/span><strong>Amylyx&#8217;s neuron-protecting drug slows ALS decline in the late-phase study<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>A novel combination of compounds has displayed that it can help decrease <a href=\"https:\/\/www.delveinsight.com\/report-store\/amyotrophic-lateral-sclerosis-als-market\"><strong>amyotrophic lateral sclerosis (ALS)<\/strong><\/a> by supporting the brain&#8217;s neurons&#8217; inner workings.<\/p>\n\n\n\n<p>After six months of treatment, AMX0035 of Amylyx Pharmaceuticals demonstrated a significant benefit compared to placebo, delaying the fast-acting neurodegenerative disease that robs the patient of their ability to talk, swallow, and eventually move and breathe.<\/p>\n\n\n\n<p>Using a standard clinical metric for gauging patients&#8217; abilities to walk, dress, and feed themselves, the company&#8217;s phase 2\/3 study showed that participants taking AMX0035 lost fewer points over time.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"The_antisense_drugs_of_Ionis_could_help_fight_ALS_and_dementia\"><\/span><strong>The antisense drugs of Ionis could help fight ALS and dementia<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p><strong><a href=\"https:\/\/www.delveinsight.com\/report-store\/frontotemporal-dementia-market\">Frontotemporal dementia (FTD)<\/a><\/strong> and <strong><a href=\"https:\/\/www.delveinsight.com\/report-store\/amyotrophic-lateral-sclerosis-als-epidemiology-forecast\">amyotrophic lateral sclerosis (ALS)<\/a><\/strong> are two fatal neurodegenerative diseases marked by different clinical signs. However, they are believed to be caused by a common underlying mechanism that is a mutation in the C9orf72 gene, which leads to the toxic accumulation of the TDP-43 protein.<\/p>\n\n\n\n<p>Now, the scientists at Mayo Clinic and the University of Pennsylvania provide further insight into the problem by throwing light on the function of another protein called poly (GR) in TDP-43 aggregation.<\/p>\n\n\n\n<p>The researchers found that drugs called antisense oligonucleotides developed by Ionis Pharmaceuticals reduced TDP-43 and neurodegeneration in mice with mutated C9orf72, as reported in a new study published in Science Translational Medicine.&nbsp;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Virus_combo_makes_solid_tumors_vulnerable_to_CAR-T_attack\"><\/span><strong>Virus combo makes solid tumors vulnerable to CAR-T attack&nbsp;<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>FDA-approved <strong><a href=\"https:\/\/www.delveinsight.com\/report-store\/car-t-cell-therapy-for-acute-lymphoblastic-leukemia-market\">CAR-T cell therapies<\/a><\/strong> like Kymriah of Novartis&#8217;, target CD19, an antigen that is widely expressed in some blood cancers. However, these therapies are not useful for solid tumors treatment, most of which express like a wide variety of proteins that they lack one target. The solid tumors create an environment that is so hostile, making it difficult for CAR-T cells to survive, expand, and ward off cancer.<\/p>\n\n\n\n<p>The scientists at the City of Hope informed that they have come up with a potential strategy for making CD19-targeted CAR-T cells function in solid tumors, and it involves an oncolytic virus.<\/p>\n\n\n\n<p>The City of Hope team engineered an oncolytic virus to create a truncated version of CD19 in solid tumor cells. Their idea was that the antigen would travel to the tumor cells&#8217; surface, making them targetable with CD19-directed <strong><a href=\"https:\/\/www.delveinsight.com\/report-store\/cart-related-neurotoxicity-nt-epidemiology-forecast\">CAR-T cell therapies<\/a><\/strong>. The combination worked in human cancer cells and mouse models.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><span class=\"ez-toc-section\" id=\"Orca_Bio_Lyell_Immunopharma_pen_new_deal_to_open_up_CAR-T_for_solid_tumors\"><\/span><strong>Orca Bio, Lyell Immunopharma pen new deal to open up CAR-T for solid tumors<\/strong><span class=\"ez-toc-section-end\"><\/span><\/h2>\n\n\n\n<p>Two Californian biotechs collaborate to break the barrier from blood to solid tumors in cell therapy. While being a significant hit in blood cancers for some patients, CAR-T has struggled in tumors, with several poor trial results over the years diminishing hopes it could translate its efficacy into a broader range of oncology targets.<\/p>\n\n\n\n<p>Orca Bio and Lyell Immunopharma partner up and sign an R &amp; D agreement combining two partners&#8217; technology to generate potentially synergistic therapeutic solutions for solid tumors.<\/p>\n\n\n\n<p>A small but growing number of academic researchers and biotechs like Novartis are also getting in on the act, trying to tweak the use of cell therapies to work on tumors of the brain, kidney, and other targets.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Amylyx&#8217;s neuron-protecting drug slows ALS decline in the late-phase study A novel combination of compounds has displayed that it can help decrease amyotrophic lateral sclerosis (ALS) by supporting the brain&#8217;s neurons&#8217; inner workings. After six months of treatment, AMX0035 of Amylyx Pharmaceuticals demonstrated a significant benefit compared to placebo, delaying the fast-acting neurodegenerative disease that [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":10684,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_editorskit_title_hidden":false,"_editorskit_reading_time":0,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","advgb_blocks_editor_width":"","advgb_blocks_columns_visual_guide":"","footnotes":""},"categories":[32],"tags":[18716,1262,18718,420,18717],"industry":[17225],"therapeutic_areas":[17235,17245,17228],"class_list":["post-10682","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-notizia","tag-amylyx","tag-latest-news","tag-lyellpharma-news","tag-news","tag-orca","industry-pharmaceutical","therapeutic_areas-cell-and-gene-therapy","therapeutic_areas-neurology","therapeutic_areas-oncology"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v25.8 (Yoast SEO v25.8) - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>AMX0035 for ALS; Ionis antisense drug; CAR-T research; Orca&#039;s new deal<\/title>\n<meta name=\"description\" content=\"Amylyx&#039;s neuron-protecting drug slows ALS decline in the late-phase study. 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Recent Pharma, Healthcare and Biotech Happenings<\/a>"],"unlinked":["<span class=\"advgb-post-tax-term\">Notizia - Recent Pharma, Healthcare and Biotech Happenings<\/span>"]},"tags":{"linked":["<a href=\"https:\/\/www.delveinsight.com\/blog\/notizia\/\" class=\"advgb-post-tax-term\">Amylyx<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/notizia\/\" class=\"advgb-post-tax-term\">latest news<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/notizia\/\" class=\"advgb-post-tax-term\">Lyellpharma news<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/notizia\/\" class=\"advgb-post-tax-term\">news<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/notizia\/\" class=\"advgb-post-tax-term\">Orca<\/a>"],"unlinked":["<span class=\"advgb-post-tax-term\">Amylyx<\/span>","<span class=\"advgb-post-tax-term\">latest news<\/span>","<span class=\"advgb-post-tax-term\">Lyellpharma news<\/span>","<span class=\"advgb-post-tax-term\">news<\/span>","<span class=\"advgb-post-tax-term\">Orca<\/span>"]}},"comment_count":"0","relative_dates":{"created":"Posted 6 years ago","modified":"Updated 5 years ago"},"absolute_dates":{"created":"Posted on Sep 3, 2020","modified":"Updated on Jul 24, 2021"},"absolute_dates_time":{"created":"Posted on Sep 3, 2020 6:13 pm","modified":"Updated on Jul 24, 2021 12:58 pm"},"featured_img_caption":"recent-pharma-happenings-for-amylyx-orca-lyell-ionis","series_order":"","_links":{"self":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/10682","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/comments?post=10682"}],"version-history":[{"count":0,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/10682\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/media\/10684"}],"wp:attachment":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/media?parent=10682"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/categories?post=10682"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/tags?post=10682"},{"taxonomy":"industry","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/industry?post=10682"},{"taxonomy":"therapeutic_areas","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/therapeutic_areas?post=10682"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}