07:47pm Sunday 20 October 2019

New partnership aims to discover treatment to correct CF gene mutation in patients

Through this collaboration, an academic research group and a pharmaceutical company will work together with the goal of developing and commercialising a CF drug through targeted drug screening. This joint effort builds upon SickKids’ long history of CF research including the discovery of the mutated gene in Cystic Fibrosis (CFTR) in 1989.

“This is a unique partnership that will bring together SickKids’ ground-breaking history of CF research and GSK’s drug discovery expertise to develop new medicines that could have a huge impact on the CF population,” says Christine Bear, SickKids Project Lead, Co-Director of the SickKids CF Centre, Senior Scientist in Molecular Structure & Function at SickKids and Professor in Physiology at the University of Toronto.

Cystic Fibrosis is a genetic disorder caused by a mutation in the CFTR gene. More than 1,900 mutations in the CFTR gene have been identified, however nearly 90 per cent of individuals with CF in Canada carry at least one copy of the most common CF-causing mutation, delta F508.

According to Bear, the CF medicines currently available treat only the symptoms of the disease. This collaboration is focused on discovering a medicine that will correct the delta-F508 mutant protein and potentially stop or slow the progression of disease for the majority of CF patients.

“This partnership can potentially enable us to accelerate the discovery of new CF drugs and dramatically improve quality of life for many CF patients,” says Dr. Janet Rossant, Chief of Research at SickKids and University Professor in the Departments of Molecular Genetics, Obstetrics and Gynaecology at the University of Toronto.

The alliance is part of GSK’s Discovery Partnerships with Academia (DPAc) initiative, designed to bring together the complementary skill sets of GSK and individual academic groups in the search for new medicines.

“This collaboration marks the first DPAc program outside the US and UK and we are excited to be working in Canada with SickKids, whose legacy in CF research is globally recognized,” said Pearl Huang, Vice President, DPAc.  

“We are proud to have this opportunity to partner with SickKids through our DPAc initiative, to share expertise that will provide new healthcare solutions to Canadians and people around the world,” said Rav Kumar, Vice-President, Regulatory & Development Operations of GSK Canada.  

Under the terms of the agreement, SickKids will receive success-based financial support from GSK linked to milestone events, as well as royalties on sales from any product that is successfully commercialised out of the collaboration.

About The Hospital for Sick Children
The Hospital for Sick Children (SickKids) is recognized as one of the world’s foremost paediatric health-care institutions and is Canada’s leading centre dedicated to advancing children’s health through the integration of patient care, research and education. Founded in 1875 and affiliated with the University of Toronto, SickKids is one of Canada’s most research-intensive hospitals and has generated discoveries that have helped children globally.  Its mission is to provide the best in complex and specialized family-centred care; pioneer scientific and clinical advancements; share expertise; foster an academic environment that nurtures health-care professionals; and champion an accessible, comprehensive and sustainable child health system.  SickKids is proud of its vision of Healthier Children. A Better World.™ For more information, please visit www.sickkids.ca

About SickKids Centre for Research and Learning 

The SickKids Centre for Research and Learning will bring together researchers from different scientific disciplines and a variety of clinical perspectives, to accelerate discoveries, new knowledge and their application to child health — a different concept from traditional research building designs. The facility will physically connect SickKids science, discovery and learning activities to its clinical operations.  Designed by award-winning architects Diamond + Schmitt Inc. and HDR Inc. with a goal to achieve LEED® Gold Certification for sustainable design, the Centre will create an architectural landmark as the eastern gateway to Toronto’s Discovery District. The SickKids Centre for Research and Learning is funded by a grant from the Canada Foundation for Innovation, the Government of Ontario, philanthropist Peter Gilgan and community support for the ongoing fundraising campaign. For more information, please visit www.sickkidsfoundation.com/bepartofit.

For more information, please contact:

Polly Thompson
The Hospital for Sick Children
Phone: 416-813-7654 ext. 2059
email: polly.thompson@sickkids.ca

Matet Nebres
The Hospital for Sick Children
Phone: 416-813-6380
email: matet.nebres@sickkids.ca

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