Zorawar Chandi’s Parents Have Raised Nearly $1.4 Million Toward a $5 Million Gene Therapy Ontario Called ‘Experimental’

Nearly $1.4 million has been raised through a GoFundMe campaign for 2.5-year-old Zorawar Singh Chandi of Brampton, Ontario, whose parents hope to take him to Boston Children’s Hospital for a gene therapy that the province refused to pay for. Ontario’s health ministry turned down their funding request in January 2026 and called the treatment “experimental,” according to CP24. His father, Mandeep Chandi, estimates the treatment will cost about $5 million.

The Diagnosis Behind the Fundraiser

Zorawar has aromatic L-amino acid decarboxylase deficiency, known as AADC deficiency. CP24 reported that his parents, Mandeep Chandi and Ramandeep Kaur, noticed developmental delays when he was about two months old, and he was diagnosed at seven months.

The condition affects an enzyme the body needs to make dopamine and serotonin. MedlinePlus, the U.S. National Library of Medicine’s genetics guide, says symptoms usually begin in the first six months of life and can include feeding problems, weak muscle tone, sleep trouble, delayed development and episodes of the eyes rolling upward. It reports about 350 cases in the medical literature.

The National Organization for Rare Disorders adds that children may look typical at birth and that medications can manage symptoms, though responses vary and finding a regimen that works can be difficult.

CP24 described what that looks like for Zorawar. His eyes stay rolled upward for up to 30 minutes several times a week. He eats a liquid diet because chewing is hard, and he is often irritable. According to the family’s application, his developmental level matches that of a one-month-old.

What the Treatment Is and Where It Stands

The therapy his parents are seeking is Kebilidi, which the U.S. Food and Drug Administration approved on November 14, 2024. The agency called it the first FDA-approved gene therapy for AADC deficiency. It is given as four infusions during one surgical session into a brain region involved in motor control, and it must be given at a medical center that specializes in pediatric stereotactic neurosurgery. The approval covers adults and children, and the supporting study enrolled 13 children.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in the agency’s announcement that it offers “innovative treatment options for rare diseases that are otherwise difficult to manage.” Nicole Verdun, director of the Office of Therapeutic Products in the same center, noted that AADC deficiency “can cause a range of debilitating symptoms, including life-threatening complications.”

NORD notes that a version of the therapy, eladocagene exuparvovec under the name Upstaza, is approved in the European Union and the United Kingdom for people 18 months and older with severe disease. In Canada, the picture is different. CP24 reported that Health Canada has not received a regulatory submission for Kebilidi, and that the decision to file one rests with the manufacturer, PTC Therapeutics. The company did not respond to CTV News Toronto. Boston Children’s Hospital told the outlet that clinical studies are ongoing and it is too early to call the treatment a cure, though it appears to correct the root cause of the disorder.

How Ontario Reached “Experimental”

The family’s paper trail, as laid out by CP24, runs through most of a year:

• August 13, 2025: the family doctor asked for prior approval of full payment for insured out-of-country services.
• October 2025: the ministry asked for more information.
• January 2026: the ministry denied the application, saying the treatment did not meet the requirements of the Health Insurance Act and calling it “experimental.”
• February 2026: the family appealed to the Health Services Appeal and Review Board, which also denied the appeal.

Ontario’s own guidance for doctors explains the standard. On its out-of-country OHIP page, the province says OHIP funds care outside Canada only with prior approval, and that a service must not be “experimental or for research or for a survey.” It must also be accepted in Ontario as appropriate for the same medical circumstances and not available in Ontario as an identical or equal procedure, except when a delay would cause death or medically significant irreversible tissue damage.

CP24 also reported that Regulation 552 under the Health Insurance Act was amended on October 6, 2026, narrowing when out-of-country funding can be approved. Lawyer Lonny Rosen of Rosen Sunshine LLP told the outlet that funding now requires four conditions: the treatment is performed in Ontario, the drug and its administration are publicly funded in Ontario, access to the drug is temporarily unavailable, and travel abroad is necessary to avoid death or medically significant irreversible tissue damage. The ministry would not comment on Zorawar’s case, citing patient confidentiality.

A Family Counting on Strangers

With public funding refused, the family turned to a crowdfunding campaign meant to cover the treatment, medical assessments, travel and follow-up care. CP24 said it had raised nearly $1.4 million when the story was published on November 13, 2026, which leaves a large gap against the roughly $5 million estimate.

Mandeep Chandi told the outlet that the family has “no plan B right now.” He said the treatment is not affordable for a middle-class family, and that the family waited about two years and is “still left with no hope.” He asked the public for help.

What Happens Next Is Still Open

As of CP24’s report, no date for treatment had been announced, and the outlet did not say whether Boston Children’s has accepted Zorawar as a patient or scheduled him. The family’s appeal to the Health Services Appeal and Review Board has already been denied, and the ministry has declined to discuss the case.

What remains public is the gap between two descriptions of the same therapy. In the United States, the FDA has approved it. In Ontario, the ministry classed it as experimental when it reviewed this family’s request. Zorawar’s parents are asking strangers to cover the difference.

Another family’s long fundraising road to a gene therapy ended differently: Eliza O’Neill’s parents raised $2 million for her rare disease, and the FDA later approved the therapy.

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This article was produced with AI assistance and edited for accuracy against the sources linked above.

Image: Sikander Iqbal – CC BY-SA 4.0/Wikimedia Commons