Two decades after a patient died in his gene-therapy trial, Penn’s Jim Wilson has a new approach to be tested in babies

A startup that Wilson co-founded has received $65 million in new venture capital financing, the company said Wednesday.

More than two decades after a patient with liver disease died in his gene-therapy trial, University of Pennsylvania scientist Jim Wilson has a new approach for tackling the same disease, this time in babies.

On Wednesday, a biotech start-up that plans to test the drug.

The concept remains the same: Inject patients with genetic instructions to make a crucial liver enzyme that they are unable to make themselves.

Gelsinger had a mild form of the disease, called OTC deficiency, but babies with a severe form of the illness can lapse into a coma within a day or two of birth, their brains damaged by a buildup of ammonia. Some die soon thereafter; the rest have little recourse beyond a liver transplant.

Yet when Gelsinger was injected with the corrective gene, packaged inside a virus, his immune system unexpectedly rebelled, and he died within a week.

In Wilson’s new approach, the gene is delivered with a different type of virus that does not trigger the immune system — a delivery method that he already has licensed for use in several other drugs. Among them is for a fatal childhood disease called spinal muscle atrophy.

The new gene therapy for OTC deficiency is being tested by iECURE, a Plymouth Meeting-based start-up that Wilson cofounded in 2020. Wilson, who serves as iECURE’s chief scientific adviser, also owns a stake in the venture, as does Penn.

After securing the go-ahead from the FDA, the plan is to treat a small group of infants by the end of 2023, chief executive officer Joseph Truitt said. Based on Wilson’s preliminary studies in his lab at Penn.

The bulk of the new $65 million investment will go toward testing the treatment for OTC deficiency, though some will be used to develop treatments for two other liver diseases, Truitt said.

The funding is especially significant given that venture capitalists lately have been lukewarm on the biotech sector, said Supriya Munshaw, a faculty member at Johns Hopkins Carey Business School who studies investment in the life sciences.

It’s a good vote of confidence that they were able to raise this much money in the current market,” she said. “There’s lots of hesitation in gene and cell therapies, which specifically have cooled down over the last few years.”

The financing was led by venture capital firms Novo Holdings A/S and LYFE Capital, with “significant participation” from Versant Ventures and OrbiMed Advisors, which previously funded iECURE with an initial $50 million.