A Jim Wilson start-up will conduct the first FDA-approved tests of a gene therapy in infants
The start-up from University of Pennsylvania scientist Jim Wilson raised $65 million in late 2022 to help develop the treatment and conduct clinical trials. A company, iECURE, founded by University of Pennsylvania scientist Jim Wilson, has received FDA approval to test a gene therapy in babies for a rare liver disease for the first time in the US. The company is developing treatments based on research conducted by Wilson, director of the University of Penn's gene therapy program. The treatment is designed to treat babies whose livers are unable to produce a crucial enzyme and can lead to coma within a day or two of birth, resulting in brain damage and death. The new approach involves delivering the gene from a different type of virus that does not trigger the immune system, a delivery method Wilson has licensed for use in several other drugs. Both Penn and Wilson have an unspecified financial interest in the company.
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Published : a month ago by Harold Brubaker in Business Health
A company developing treatments based on research by Jim Wilson, director of the University of Pennsylvania's gene therapy program, has received FDA approval to test a gene therapy in babies for a rare liver disease. Read more
A company started by University of Pennsylvania scientist Jim Wilson has received FDA approval to test a form of gene therapy in infants for the first time in the United States, the company said Thursday.
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 7 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.
Topics: FDA, Startups