Dr. Xi Huang’s team developed a designer peptide that specifically kills glioblastoma (GBM) cells by targeting a tumor-specific ion channel complex, offering new potential for targeted therapy and immunotherapy combination.

The Pearson Lab developed a platform to discover small molecules that contract disease-causing repeat expansions like CAG tracts, offering a strategy to slow or reverse progression in over 70 neurodegenerative and neuromuscular diseases.

SickKids researchers developed novel bile acid variants that are retained in the gut, inhibit the TcdB toxin, and prevent recurrent Clostridioides difficile infection in mouse models, offering a promising oral alternative to current therapies.

Researchers at SickKids and the University of Manitoba have developed HexM, a stable hybrid enzyme with therapeutic potential for Tay-Sachs and Sandhoff diseases, which are currently untreatable neurodegenerative disorders caused by β-hexosaminidase deficiencies.

The Huang Lab has identified KCNB2 as a key regulator of medulloblastoma growth, showing that its deficiency reduces tumor burden, prolongs survival, and enhances anti-SHH therapy efficacy in preclinical models.

The ImmunoTargeting Vaccine (ITV) is a modular, adjuvant-free subunit vaccine platform that enhances immune responses by targeting HLA-DR, enabling rapid, cost-effective development with broad strain coverage and improved thermostability for global accessibility.

The Coles and Maynes labs at SickKids are developing novel heart failure therapeutics by targeting the ILK–parvin complex within the integrin-based mechanotransduction pathway, aiming to reverse cardiac fibrosis and dysfunction at their root.

The Post lab has developed a patent-protected method to differentiate human stem cells into ALMs, which can be delivered directly to the lungs to modulate the immune environment and potentially treat multiple lung diseases simultaneously.

Researchers at SickKids have found that niclosamide, an FDA-approved drug, safely inhibits all three C. difficile toxins—including a highly virulent variant—without harming host cells or gut microbiota, offering a promising strategy to prevent disease and recurrence.

SickKids researchers identified CFTR potentiators that prevent mucositis in bronchial cells exposed to tobacco smoke, offering potential as a disease-modifying therapy for chronic obstructive pulmonary disorder.