MaRS Innovation Supports LAB150 Research on New Therapeutics for CF and Other Conditions

Vijaya Iyer, PhD avatar

by Vijaya Iyer, PhD |

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MaRS Innovation recently announced the launch of two new projects targeting the development of new therapeutics for cystic fibrosis (CF) and respiratory syncytial virus (RSV).

These projects are part of LAB150, MaRS Innovation’s joint venture with Evotec AG to support collaboration among scientists, investors, and pharmaceutical companies to accelerate development of new therapies.

“We are thrilled to be working with these exceptional scientific teams to tackle major unmet needs in both cystic fibrosis and RSV infection treatment,” Rafi Hofstein, PhD, said in a press release. Hofstein is co-founder of LAB150, and president and CEO of MaRS Innovation.

MaRS Innovation is a non-profit organization that supports the development and commercialization of promising scientific breakthroughs.

The CF project is based on research from The Hospital for Sick Children (SickKids) in Toronto, Canada. Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene are the cause of CF. The discovery of CFTR modulators have led to significant advances in the treatment of CF; however, not all CF patients respond to treatment with the CFTR modulators available.

The SickKids project aims to develop new modulators of key CF targets, or modifier genes, that can be used either as monotherapy or in combination with CFTR modulators.

Modifier genes regulate the activity of other genes, such as the faulty CFTR gene in CF. Several such modifier genes for CFTR have been identified. Targeting these modifier genes is a potential approach to restore CFTR function, and improve the patient’s health outcomes.

This project results from  decade of research by senior scientists like Lisa Strug, PhD, Johanna Rommens, and Roman Melnyk at SickKids.

“We are dedicated to identifying therapies that can improve the lives of individuals living with cystic fibrosis and other obstructive lung diseases,” said Strug, senior scientist in the Genetics & Genome Biology program at SickKids Research Institute. “Through this project, we hope to find targeted treatments that could benefit many patients and their families.”

The second project stems from research conducted about RSV at the University of Toronto, Canada. RSV is a leading cause of throat and lung infections in kids, and the virus is expert at invading the immune system.

Researchers at the University of Toronto have identified a receptor on lung cells — called nucleolin — that is used by RSV to establish a successful infection. The goal of the project is to target nucleolin and block its interaction with the virus, subsequently preventing its escape from the immune system.

“Our discovery and validation of nucleolin as a target for RSV infection opens up an entirely new approach for RSV treatment and prevention. We are very excited to be working with MaRS Innovation and Evotec through LAB150 to develop potent nucleolin-targeting compounds for clinical use in RSV infections,” said Richard Hegele, MD, PhD, professor at the laboratory Medicine & Pathobiology, University of Toronto.

With the addition of these two projects, LAB150 now oversees a total of four disease specific projects. The other two focus on glaucoma (eye conditions that damage the optic nerve), and a rare skin infection called Netherton syndrome.

“With four projects funded and more to come, LAB150 is now well established as a major mechanism to advance Canadian drug discovery projects,” Hofstein said.