VIPER, the lentiviral IPPER platform for drug discovery in trafficking diseases. IPPER-accelerated development of pharmacological correctors for cystic fibrosis

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Challenge: Drug discovery by high-throughput screening of candidate compounds faces challenges and needs the development and validation of specific platforms. An example of complex diseases that would benefit from such platforms is cystic fibrosis, a lung disease caused by genetics and aggravated by air pollution. An immediate response of bronchial epithelial cells to pollution is the downregulation of CFTR from the cell surface. This initiates a cascade of degenerative processes leading to the disease of cystic fibrosis.

Solution: IPPER is a platform developed within a collaboration between McGill University and LDC (Germany) funded by CQDM through the Québec-Germany program to establish a series of novel techniques and reagents to detect new compounds with endoplasmic reticulum (ER)-stress modifying characteristics. This new project is an excellent opportunity to apply the developed IPPER platform to a complex problem, such as acquired cystic fibrosis, and to demonstrate its multiple use. The main goal of this project is to develop and validate the Viral Integrated platform for the Pharmacology of the ER (VIPER), the lentiviral and enhanced version of IPPER, and prove its utility for drug discovery in trafficking diseases. The platform will identify and verify potential therapeutic compounds and their mechanism of action by direct assessment in patient cells under recreated air pollution stress.

Achievements/Impact: Beside the major benefits associated with the discovery of new candidate drugs to treat acquired cystic fibrosis, an important achievement will be the availability of a new tool, a patient cell-based platform in the development of novel pharmaceuticals. By benefitting from the multiple funders’ contributions and by coordinating the individual expertise of several renowned institutes, the project will strengthen these international partnerships that productively tackle global challenges. This will increase the capabilities of Traffick Therapeutics, a Montréal-based R&D company that specializes in the development of molecules with therapeutic potential up to the pre-clinical phase, to attract new investments and further expand its development.

Principal Investigator:
David Y. Thomas
McGill University
Co-Investigators:
Graeme Carlile
Gregor Jansen
McGill University
John Hanrahan 
Traffick Therapeutics 
Bert Klebl 
Lead Discovery Center (Germany) 
Ongoing Project
$3,071,147 / 3 years
Supported by CQDM through:
– MEI
And by co-funding partners:
– Traffick Therapeutics
– Lead Discovery Center
– Cystic Fibrosis Canada
– CIHR
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