{"id":3422,"date":"2024-02-26T10:45:33","date_gmt":"2024-02-26T01:45:33","guid":{"rendered":"https:\/\/bionauts.jp\/?p=3422"},"modified":"2025-05-14T13:51:54","modified_gmt":"2025-05-14T04:51:54","slug":"parkinsons-disease-drug","status":"publish","type":"post","link":"https:\/\/bionauts.jp\/?p=3422","title":{"rendered":"Therapeutic Drugs for \u03b1-Synucleinopathy"},"content":{"rendered":"<h3>Advantages<\/h3>\n<ul>\n<li>A single dose is expected to produce long-term effects (more than one year).<\/li>\n<li>Maintains the physiological function of \u03b1-Syn.<\/li>\n<li>Effective for both sporadic and hereditary cases.<\/li>\n<\/ul>\n<h3>Current Stage and Key Data<\/h3>\n<p><u>Preclinical research stage<\/u><\/p>\n<ul>\n<li>Identification of multiple SSOs that efficiently induce <em>SNCA<\/em> splice switching (promotion of low-aggregation variants).<\/li>\n<li>Intracerebroventricular administration (once immediately after birth) of the SSOs to Parkinson&#8217;s disease model mice (human <em>SNCA<\/em> E46K BAC Tg +\/-) induced sustained splice switching and reduction of aggregating \u03b1-Syn protein in the cerebral cortex and brainstem (13 months after administration).<\/li>\n<li>Administration of the SSO reduced motor dysfunction measured by grip strength, balance beam test, and open field test in Parkinson&#8217;s disease model mice (+\/+) (36 weeks old).<\/li>\n<li>Histological evaluations showed improvement in degeneration of dopaminergic neurons in the striatum (putamen) (13 months after administration).<\/li>\n<\/ul>\n<table style=\"border-collapse: collapse; width: 39.8333%; height: 5px;\">\n<tbody>\n<tr>\n<td style=\"width: 100%;\"><a href=\"http:\/\/bionauts.jp\/wp-content\/uploads\/2024\/02\/BK-04061a.png\"><img decoding=\"async\" loading=\"lazy\" class=\"alignleft size-full wp-image-4530\" src=\"http:\/\/bionauts.jp\/wp-content\/uploads\/2024\/02\/BK-04061a.png\" alt=\"\" width=\"240\" height=\"178\" \/><\/a><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<table style=\"border-collapse: collapse; width: 100%;\">\n<tbody>\n<tr>\n<td style=\"width: 100%;\"><a href=\"http:\/\/bionauts.jp\/wp-content\/uploads\/2024\/02\/BK-04061b.png\"><img decoding=\"async\" loading=\"lazy\" class=\"alignleft size-full wp-image-4531\" src=\"http:\/\/bionauts.jp\/wp-content\/uploads\/2024\/02\/BK-04061b.png\" alt=\"\" width=\"984\" height=\"265\" srcset=\"https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/02\/BK-04061b.png 984w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/02\/BK-04061b-300x81.png 300w, https:\/\/bionauts.jp\/wp-content\/uploads\/2024\/02\/BK-04061b-768x207.png 768w\" sizes=\"(max-width: 709px) 85vw, (max-width: 909px) 67vw, (max-width: 1362px) 62vw, 840px\" \/><\/a><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n<h3>Partnering Model<\/h3>\n<p><u>Seeking an exclusive license partner company<\/u><\/p>\n<ul>\n<li>Examples of potential partners: Biotech\/pharmaceutical companies focusing on neurodegenerative diseases such as Parkinson&#8217;s disease and\/or nucleic acid medicines.<\/li>\n<\/ul>\n<h3>Background<\/h3>\n<p>\u03b1-Synucleinopathies, such as Parkinson&#8217;s disease, dementia with Lewy bodies, and multiple system atrophy, are neurodegenerative diseases characterized by the abnormal accumulation of \u03b1-Syn aggregates. Research on antisense oligonucleotides (ASOs) targeting <em>SNCA<\/em> knockdown has been reported. However, a major limitation is the need for repeated administration (e.g., via lumbar puncture) due to the insufficient duration of efficacy. In addition, \u03b1-Syn is thought to be involved in regulating synaptic function, raising concern that its knockdown may inhibit neurophysiological processes.<\/p>\n<h3>Principal Investigator<\/h3>\n<p>Masahisa KATSUNO (Department of Neurology, Nagoya University Graduate School of Medicine, Tokai Higher Education and Research System)<\/p>\n<h3>Reference<\/h3>\n<ul>\n<li>Paper: Unpublished yet<\/li>\n<li>Patent: PCT International Patent Application pending (WO2025\/063185)<\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n<p>Project No. BK-04061<\/p>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Splice-switching antisense oligonucleotides (SSOs) targeting the \u03b1-synuclein (SNCA) gene suppress \u03b1-synuclein (\u03b1-Syn) aggregation.<\/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\/3422"}],"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=3422"}],"version-history":[{"count":3,"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/posts\/3422\/revisions"}],"predecessor-version":[{"id":4532,"href":"https:\/\/bionauts.jp\/index.php?rest_route=\/wp\/v2\/posts\/3422\/revisions\/4532"}],"wp:attachment":[{"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=3422"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=3422"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=3422"},{"taxonomy":"categories_of_inventions","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories_of_inventions&post=3422"},{"taxonomy":"disease_categories___indication","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fdisease_categories___indication&post=3422"},{"taxonomy":"asset_type","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fasset_type&post=3422"},{"taxonomy":"development_phase","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fdevelopment_phase&post=3422"},{"taxonomy":"medicine","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fmedicine&post=3422"},{"taxonomy":"other_than_medicine","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fother_than_medicine&post=3422"},{"taxonomy":"material","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fmaterial&post=3422"},{"taxonomy":"engineering","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fengineering&post=3422"},{"taxonomy":"software_it","embeddable":true,"href":"https:\/\/bionauts.jp\/index.php?rest_route=%2Fwp%2Fv2%2Fsoftware_it&post=3422"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}