iEcure raises $65M to advance its gene-editing technology and Receives Pediatric Disease Designation from FDA 

Penn spinout iEcure raised $65 million to advance its in vivo gene-editing technology aimed at treating rare pediatric liver diseases. The Series A-1 equity financing round for iEcure was co-led by Novo Holdings and Lyfe Capital. Existing investors Versant Ventures and OrbiMed Advisors also participated in the round.

The company plans to use the new funding to complete pre-clinical work, initiate clinical trials and generate clinical data from a Phase 1/2 study of its lead investigational product, GTP-506, developed in partnership with Penn’s Gene Therapy Program.  GTP-506 is a treatment for ornithine transcarbamylase deficiency, a rare condition caused by a genetic defect in a liver enzyme responsible for the detoxification of ammonia.  This condition can lead to irreversible neurological impairment, seizures, coma, and death.

Another milestone for iEcure took place in August 2022, when the company was granted a rare pediatric disease designation from the Food and Drug Administration for GTP-506.

Read more about the Series A-1 financing round here.  Read about iEcure’s first FDA designation for GTP-506 here and its second designation here.

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