Home / Health / Ontario Denies Funding for Life-Saving Gene Therapy
Ontario Denies Funding for Life-Saving Gene Therapy
13 Nov
Summary
- Family crowdfunding millions for toddler's rare AADC deficiency treatment in the US.
- Ontario government denied funding, citing treatment as experimental.
- Kebilidi gene therapy, not approved in Canada, costs millions.
A Brampton family faces a daunting financial challenge as they seek millions for their two-and-a-half-year-old son Zorawar's treatment in the United States. Zorawar was diagnosed with Aromatic L-Amino Acid Decarboxylase (AADC) deficiency, a rare genetic disorder affecting approximately one in 1.3 million people. The Ontario government denied their request for out-of-country funding for Kebilidi, an FDA-approved gene therapy, citing it as experimental.
The family's doctor first applied for funding in August 2025, emphasizing Zorawar's severe developmental delays. The Ministry of Health denied the application in January 2026, a decision later upheld by the Health Services Appeal and Review Board. Kebilidi, a one-time gene therapy treatment administered directly to the brain, costs approximately $5 million. While Boston Children's Hospital notes promising results in ongoing studies, the treatment has not yet received Health Canada approval.
Recent amendments to provincial regulations, which came into effect retroactively to October 1, 2011, have further narrowed the criteria for out-of-country funding. These changes require treatments to be performed in Ontario and publicly funded there, conditions not met by Kebilidi. The family has started a GoFundMe, which has raised nearly $1.4 million towards the treatment, travel, and follow-up care.
