[vc_row el_class=”breadcrumbs”][vc_column][vc_column_text][\/vc_column_text][\/vc_column][\/vc_row][vc_section el_class=”start-ups”][vc_row][vc_column width=”1\/2″][vc_column_text el_class=”management-section”]MANAGEMENT:<\/span>\u00a0Sheila Mikhail
\nDUKE INVENTOR:<\/span>\u00a0Dwight Koeberl, Priya Kishnani
\nactustherapeutics.com<\/a>[\/vc_column_text][vc_column_text]Actus Therapeutics, a privately held portfolio company of Asklepios BioPharmaceutical, Inc. (AskBio<\/a>), was incorporated in 2017 to develop new therapies for rare diseases including Pompe disease and epilepsy. Founded by Sheila Mikhail and Phillipe Moullier, Actus will initially pursue gene therapies for Pompe disease based on technology licensed from Dwight Koeberl\u2019s laboratory at Duke University.[\/vc_column_text][\/vc_column][vc_column width=”1\/2″][vc_single_image image=”294″ img_size=”full”][\/vc_column][\/vc_row][vc_row css=”.vc_custom_1535394535521{padding-top: 15px !important;}”][vc_column][vc_column_text]The Company\u2019s goal is to use targeted, low-dose gene therapies to address this glycogen storage disorder. AskBio\u2019s double strand adeno-associated virus vectors (AAV) have the potential to provide treatment at a lower effective dose and with more cell type specificity and less potential systemic effects than other AVV.[\/vc_column_text][vc_column_text]<\/p>\n

Pompe Disease<\/h3>\n

Pompe Disease is an inherited rare disorder (< 1:40,000 births) caused by the deficiency of acid-alpha-glucosidase (GAA) in muscle. This deficiency results in the accumulation of glycogen in organs and tissues, especially muscles, which can impair their ability to function normally. While enzyme replacement has shown promise in patients with infantile-onset Pompe disease, no curative therapy is available. More information on Pompe disease is available at:\u00a0https:\/\/ghr.nlm.nih.gov\/condition\/pompe-disease<\/a>.[\/vc_column_text][\/vc_column][\/vc_row][vc_row][vc_column][vc_separator][\/vc_column][\/vc_row][\/vc_section]<\/p>\n<\/div>","protected":false},"excerpt":{"rendered":"

Developing new therapies for rare diseases including Pompe disease & epilepsy<\/p>\n","protected":false},"author":26,"featured_media":298,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"inline_featured_image":false,"_exactmetrics_skip_tracking":false,"_exactmetrics_sitenote_active":false,"_exactmetrics_sitenote_note":"","_exactmetrics_sitenote_category":0,"footnotes":""},"categories":[4],"tags":[],"class_list":["post-162","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-startups"],"acf":[],"_links":{"self":[{"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/posts\/162","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/users\/26"}],"replies":[{"embeddable":true,"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/comments?post=162"}],"version-history":[{"count":11,"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/posts\/162\/revisions"}],"predecessor-version":[{"id":849,"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/posts\/162\/revisions\/849"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/media\/298"}],"wp:attachment":[{"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/media?parent=162"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/categories?post=162"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/numbers.otc.duke.edu\/2018-report\/wp-json\/wp\/v2\/tags?post=162"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}