A company started by University of Pennsylvania scientist Jim Wilson has received FDA approval to test a form of gene editing in infants for the first time in the United States, the company said this week.
The Plymouth Meeting company, iECURE, is developing a treatment for babies whose livers are unable to make a crucial enzyme.
Infants born 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.
This is the same disease that Wilson was studying in a high-profile test that resulted in a patient death in 1999. The patient in that case, Jesse Gelsinger, had a mild form of the disease. The 18-year-old died after his body rejected the virus used to deliver the treatment.
In Wilson’s new approach with iECURE, 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.
The treatment had previously been approved for testing in the United Kingdom and Australia, iECURE said. The company is enrolling boys up to 9 months old.
“This milestone is the culmination of over 8 years of pre-clinical research in my laboratory addressing gene editing strategies for severe rare liver metabolic diseases,” Wilson said in a news release.
Founded in 2022, iECURE raised $65 million from venture capitalists in late 2022. That was during a period of waning investor enthusiasm for cell and gene therapy companies.
Both Penn and Wilson have an unspecified financial interest in iECURE.
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