Penn gene-editing spinout iEcure was granted rare pediatric disease designation from the Food and Drug Administration for their lead product.
iEcure is developing the new drug candidate, GTP-506, as a treatment for ornithine transcarbamylase deficiency, a rare genetic condition caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. This condition can lead to irreversible neurological impairment, seizures, coma, and death.
Currently, according to iEcure, the only treatment for early onset, severe ornithine transcarbamylase deficiency is a liver transplant. Available medical therapies do not correct the disease and do not eliminate the risk of life-threatening symptoms or crises.
The gene-editing approach being taken by iEcure could potentially solve this problem. The process involves making a cut in genomic DNA, and then inserting a healthy copy of the disease-causing gene into the chromosome.
Joe Truitt, CEO of iEcure, said the company expects to file an investigational new drug application for GTP-506 with the FDA in mid-2023. If approved, iEcure will be able to begin human clinical testing of the experimental gene therapy to see if it can effectively treat early onset, severe ornithine transcarbamylase deficiency.
“Receiving Rare Pediatric Disease Designation for GTP-506 highlights the dire need for new treatment options for this devastating pediatric disease,” sai Truitt.
iEcure was founded by Dr. Jim Wilson, director of Penn’s gene therapy program, and pharmaceutical industry veterans Stephen Squinto, co-founder of Alexion Pharmaceuticals, and the late Dr. Tadataka “Tachi” Yamada, a former executive at Takeda Pharmaceuticals and GlaxoSmithKline. That trio also co-founded Passage Bio, another Philadelphia gene therapy company that went public last year. Tom Woiwode, managing director at Versant Ventures, is also an iEcure co-founder.
To learn more about iEcure and its FDA designation for GTP-506, read the full article in the Philadelphia Business Journal here.