{"id":3439,"date":"2024-03-05T16:16:10","date_gmt":"2024-03-05T07:16:10","guid":{"rendered":"https:\/\/bionauts.jp\/?p=3439"},"modified":"2024-03-07T10:59:48","modified_gmt":"2024-03-07T01:59:48","slug":"polycystic-kidney-disease-treatment-drug","status":"publish","type":"post","link":"https:\/\/bionauts.jp\/?p=3439","title":{"rendered":"Polycystic Kidney Disease Treatment Drug"},"content":{"rendered":"<h3>Advantages<\/h3>\n<ul>\n<li>Unique artificially modified nucleic acid ASO accumulates in the kidneys when administered subcutaneously to animal models.<\/li>\n<li>Improvement of renal function through multiple mechanisms of action (suppression of cyst formation, suppression of renal fibrosis, improvement of vascular endothelial disorder).<\/li>\n<\/ul>\n<h3>Background and Technology<\/h3>\n<p>ADPKD is a type of cystic kidney disease and is a congenital disease caused by a single gene PKD1\/2 mutation with autosomal dominant inheritance. The incidence is 1 in 3,000 people, and it is said to be the most common single gene mutation disease. Cysts form in the liver and kidneys, resulting in kidney enlargement, and approximately half of patients in their late 60s require maintenance dialysis. The world&#8217;s only vasopressin V2 receptor antagonist has been approved as a specific treatment for slowing the progression of the disease, but its effectiveness is still not sufficient.<\/p>\n<p>We identified a therapeutic target molecule for ADPKD, developed a stabilized ASO using a unique artificially modified nucleic acid to knockdown the molecule, and confirmed its effectiveness.<\/p>\n<table style=\"border-collapse: collapse; width: 109.167%; height: 167px;\">\n<tbody>\n<tr style=\"height: 157px;\">\n<td style=\"width: 584px; height: 157px;\"><a href=\"http:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726a-2.png\"><img decoding=\"async\" loading=\"lazy\" class=\"alignleft size-large wp-image-3452\" src=\"http:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726a-2-1024x179.png\" alt=\"\" width=\"840\" height=\"147\" srcset=\"https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726a-2-1024x179.png 1024w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726a-2-300x53.png 300w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726a-2-768x135.png 768w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726a-2-1536x269.png 1536w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726a-2-2048x359.png 2048w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726a-2-1200x210.png 1200w\" sizes=\"(max-width: 709px) 85vw, (max-width: 909px) 67vw, (max-width: 1362px) 62vw, 840px\" \/><\/a><\/td>\n<\/tr>\n<tr style=\"height: 11px;\">\n<td style=\"width: 584px; height: 10px;\"><a href=\"http:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726b-3.png\"><img decoding=\"async\" loading=\"lazy\" class=\"alignleft size-large wp-image-3454\" src=\"http:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726b-3-1024x364.png\" alt=\"\" width=\"840\" height=\"299\" srcset=\"https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726b-3-1024x364.png 1024w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726b-3-300x107.png 300w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726b-3-768x273.png 768w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726b-3-1536x546.png 1536w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726b-3-2048x727.png 2048w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/03\/BK-04726b-3-1200x426.png 1200w\" sizes=\"(max-width: 709px) 85vw, (max-width: 909px) 67vw, (max-width: 1362px) 62vw, 840px\" \/><\/a><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<p><span style=\"font-size: 23px; font-weight: 900;\">Reference<\/span><\/p>\n<ul>\n<li>Patent pending (Unpublished yet)<\/li>\n<\/ul>\n<h3>Principal Investigator<\/h3>\n<p>Noritoshi KATO \uff08Department of Nephrology, Nagoya University Hospital\uff09<\/p>\n<h3>Current Stage and Next Step<\/h3>\n<ul>\n<li>\u00a0Our original artificially modified nucleic acid ASO accumulated in the kidney when administered subcutaneously to cystic kidney model animals and showed a stable knockdown effect in vivo. The therapeutic effects showed inhibition of cyst formation, inhibition of renal fibrosis, improvement of markers related to improvement of vascular endothelial damage, inhibition of renal hypertrophy, and improvement of renal function. In addition, the unique artificially modified nucleic acid showed a more stable effect on suppressing renal hypertrophy than LNA, a well-known artificially modified nucleic acid.<\/li>\n<li>Currently analyzing the mechanism of action <em>in vitro<\/em>.<\/li>\n<li>We are looking for collaborative partner companies for non-clinical and clinical development of ASO therapeutics for ADPKD.<\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n<p>Project No. BK-04726<\/p>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Therapeutic target molecules and antisense oligonucleic acid (ASO) therapeutic agents for autosomal dominant polycystic kidney disease (ADPKD).<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_mi_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0},"categories":[1],"tags":[],"categories_of_inventions":[46],"disease_categories___indication":[],"asset_type":[],"development_phase":[],"medicine":[105,108],"other_than_medicine":[],"material":[],"engineering":[],"software_it":[],"aioseo_notices":[],"_links":{"self":[{"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/posts\/3439"}],"collection":[{"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=3439"}],"version-history":[{"count":8,"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/posts\/3439\/revisions"}],"predecessor-version":[{"id":3456,"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/posts\/3439\/revisions\/3456"}],"wp:attachment":[{"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=3439"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=3439"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=3439"},{"taxonomy":"categories_of_inventions","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories_of_inventions&post=3439"},{"taxonomy":"disease_categories___indication","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fdisease_categories___indication&post=3439"},{"taxonomy":"asset_type","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fasset_type&post=3439"},{"taxonomy":"development_phase","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fdevelopment_phase&post=3439"},{"taxonomy":"medicine","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fmedicine&post=3439"},{"taxonomy":"other_than_medicine","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fother_than_medicine&post=3439"},{"taxonomy":"material","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fmaterial&post=3439"},{"taxonomy":"engineering","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fengineering&post=3439"},{"taxonomy":"software_it","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fsoftware_it&post=3439"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}