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

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 for ornithine transcarbamylase deficiency, a rare condition caused by a genetic defect in a liver enzyme responsible for the detoxification of ammonia. Read more here

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 their lead product candidate, GTP-506, developed in partnership with Penn’s Gene Therapy Program.  GTP-506 is 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.

Shortly after receiving its first designation, iEcure was also awarded orphan drug designation for GTP-506. “The FDA’s decision to grant both Orphan Drug Designation and Rare Pediatric Disease Designation for our investigational gene editing therapy aligns with our mission to provide treatments for patients where few if any options exist and highlights the urgency of developing a treatment for pediatric patients with OTC deficiency, a serious and life-threatening liver disease,” said Joe Truitt, CEO of iEcure.

Read about iEcure’s first FDA designation for GTP-506 here and its second designation here.

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