{"id":2243,"date":"2023-07-18T20:17:00","date_gmt":"2023-07-19T00:17:00","guid":{"rendered":"https:\/\/cqdm.org\/en\/news-and-events\/development-of-pharmacological-correctors-for-alpha-1-antitrypsin-deficiency-atd-using-viper\/"},"modified":"2024-09-25T16:25:31","modified_gmt":"2024-09-25T20:25:31","slug":"development-of-pharmacological-correctors-for-alpha-1-antitrypsin-deficiency-atd-using-viper","status":"publish","type":"post","link":"https:\/\/cqdm.org\/en\/news-and-events\/development-of-pharmacological-correctors-for-alpha-1-antitrypsin-deficiency-atd-using-viper\/","title":{"rendered":"Development of pharmacological correctors for Alpha-1 antitrypsin deficiency (ATD) using VIPER"},"content":{"rendered":"\n

Challenge:<\/strong> Drug\u00a0discovery by\u00a0high-throughput screening of candidate compounds\u00a0is usually limited in efficacy\u00a0as their\u00a0functional potential is not assessed\u00a0in\u00a0relevant biological systems, such as\u00a0patient\u00a0cells,\u00a0during this first selection; new platforms are needed that\u00a0enable functional testing\u00a0within specific biological systems.\u00a0An example of\u00a0protein trafficking\u00a0that would benefit\u00a0from\u00a0such platforms\u00a0is\u00a0Alpha-1 antitrypsin deficiency (ATD),\u00a0an orphan disease, whose\u00a0behaviour\u00a0in the endoplasmic reticulum (ER) is complex,\u00a0rendering\u00a0the design of\u00a0therapeutic candidates\u00a0difficult.<\/p>\n\n\n\n

Solution:<\/strong> The\u00a0primary goal of this project is to validate the utility of IPPER,\u00a0a platform developed\u00a0within\u00a0a collaboration\u00a0funded by CQDM\u00a0through the Quebec-Germany program\u00a0between Pr. Thomas (McGill)\u00a0and LDC (Germany),\u00a0in drug discovery of therapeutics for protein\u00a0trafficking diseases,\u00a0in particular for\u00a0Alpha-1 Antitrypsin Deficiency. This challenge is an excellent\u00a0opportunity to show the power of IPPER to resolve a major problem in cell-based high throughput\u00a0screening.\u00a0The team will\u00a0validate\u00a0VIPER\u00a0reporter platform, an enhanced version of IPPER platform, as a versatile tool to aid the development of\u00a0novel\u00a0therapies, while providing additional tools,\u00a0standard\u00a0operating\u00a0procedures\u00a0and lead compounds to correct ATD.\u00a0More specifically, the project will lead to the identification of candidate molecules,\u00a0preselected\u00a0by\u00a0their ability to activate\u00a0the Alpha-1 antitrypsin pathway,\u00a0the validation\u00a0of these\u00a0candidate compounds\u00a0and the elucidation of their mechanism of action.<\/p>\n\n\n\n

Achievements\/Impact:<\/strong> Beside\u00a0the\u00a0major\u00a0benefits associated with the discovery of new\u00a0compounds\u00a0of interest for\u00a0ATD\u00a0patients, a\u00a0critical\u00a0achievement\u00a0is\u00a0the\u00a0availability\u00a0of a\u00a0patient cell-based\u00a0platform that\u00a0can\u00a0identify\u00a0drug candidates\u00a0with therapeutic potential in specific\u00a0cellular\u00a0systems,\u00a0such as endoplasmic reticulum-associated\u00a0\u00a0will be offered\u00a0as a technical service for the\u00a0team\u2019s\u00a0in-house projects, for pharmaceutical companies and contract research organizations.\u00a0This\u00a0proof-of-principle\u00a0project will allow\u00a0Traffick Therapeutics\u00a0(TTI), a\u00a0Montr\u00e9al-based R&D company\u00a0with expertise\u00a0in translational respiratory disease research,\u00a0to attract new investors\u00a0and further expand its development. In\u00a0addition,\u00a0this project will strengthen partnerships between several\u00a0internationally-renowned\u00a0institutes.<\/p>\n\n\n\n

Principal Investigator:<\/strong>
David Y. Thomas
<\/strong>McGill University<\/td><\/tr>
Co-Investigators:<\/strong>
John Hanrahan<\/strong>\u00a0
Traffick Therapeutics\u00a0
Eric Chevet<\/strong>\u00a0
INSERM\u00a0(France)\u00a0
Bert\u00a0Klebl<\/strong>\u00a0
Lead\u00a0Discovery Center\u00a0(Germany)\u00a0<\/td><\/tr>
Ongoing <\/strong>Project<\/strong><\/td><\/tr>
$2,027,577\u00a0\/ 3 years<\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/td><\/tr>
Supported by CQDM through:<\/strong>
<\/strong><\/strong><\/strong><\/strong>– Pfizer Canada
– MEI<\/td><\/tr>
And by co-funding partners:<\/strong>
– Traffick Therapeutics
– Lead Discovery Center
– INSERM
– CIHR
– FRQS<\/td><\/tr><\/tbody><\/table><\/figure>\n","protected":false},"excerpt":{"rendered":"

Challenge: Drug\u00a0discovery by\u00a0high-throughput screening of candidate compounds\u00a0is usually limited in efficacy\u00a0as their\u00a0functional potential is not assessed\u00a0in\u00a0relevant biological systems, such as\u00a0patient\u00a0cells,\u00a0during this first selection; new platforms are needed that\u00a0enable functional testing\u00a0within specific biological systems.\u00a0An example of\u00a0protein trafficking\u00a0that would benefit\u00a0from\u00a0such platforms\u00a0is\u00a0Alpha-1 antitrypsin deficiency (ATD),\u00a0an orphan disease, whose\u00a0behaviour\u00a0in the endoplasmic reticulum (ER) is complex,\u00a0rendering\u00a0the design of\u00a0therapeutic candidates\u00a0difficult. Solution:…<\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[1],"tags":[],"class_list":["post-2243","post","type-post","status-publish","format-standard","hentry","category-our-news"],"acf":[],"_links":{"self":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts\/2243","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/comments?post=2243"}],"version-history":[{"count":1,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts\/2243\/revisions"}],"predecessor-version":[{"id":4780,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts\/2243\/revisions\/4780"}],"wp:attachment":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media?parent=2243"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/categories?post=2243"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/tags?post=2243"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}