Principal Investigator:<\/strong><\/p>\n\n\n\n David Y. Thomas Co-investigators<\/strong><\/p>\n\n\n\n John Hanrahan<\/strong>\u00a0 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 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 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","protected":false},"featured_media":3832,"template":"","project-category":[94],"class_list":["post-2952","project","type-project","status-publish","has-post-thumbnail","hentry","project-category-platforms-and-databases"],"acf":[],"_links":{"self":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project\/2952","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project"}],"about":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/types\/project"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media\/3832"}],"wp:attachment":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media?parent=2952"}],"wp:term":[{"taxonomy":"project-category","embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project-category?post=2952"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}
<\/strong>McGill University<\/p>\n<\/div>\n\n\n\n
Traffick Therapeutics\u00a0
Eric Chevet<\/strong>\u00a0
INSERM\u00a0(France)\u00a0
Bert\u00a0Klebl<\/strong>\u00a0
Lead\u00a0Discovery Center\u00a0(Germany)\u00a0<\/p>\n<\/div>\n<\/div>\n\n\n\n
\n\n\n\nOngoing\u00a0<\/strong><\/strong>Project<\/strong><\/strong> of $2,027,577<\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong> over 3 years<\/strong><\/strong><\/h2>\n\n\n\n
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\n\n\n\nChallenge<\/h2>\n\n\n\n
Solution<\/strong><\/h2>\n\n\n\n
Achievements\/Impact<\/strong><\/h2>\n\n\n\n