FDA approves first in vivo gene insertion programme in infants

Cell and gene therapy

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency.

The Food and Drug Administration (FDA) Investigational New Drug (IND) clearance for ECUR-506 makes it the first in vivo gene insertion programme authorised for clinical investigation in infants in the US.

The OTC-HOPE study was previously cleared to begin in the UK by the Medicines & Healthcare Products Regulatory Agency (MHRA) and Australia by the Therapeutic Goods Administration (TGA).

“There is a significant need for clinical research and treatment options for newborns with severe, neonatal onset OTC deficiency,” said Gabriel Cohn, Chief Medical Officer of iECURE. “For many families whose children are diagnosed with neonatal onset OTC deficiency, liver transplant is the only curative option, but carries with it serious risks and requires considerable immunosuppressant therapy to prevent transplant rejection. ECUR-506 represents hope for potentially enabling children to produce functional OTC enzyme in the long term without the need for transplantation.”

A novel gene editing approach

The Phase I/II first-in-human study is designed primarily to assess the safety and tolerability of ECUR-506 following intravenous administration of a single dose.

Secondary objectives are to assess the pharmacokinetics and efficacy of ECUR-506. In addition, exploratory endpoints will assess disease-specific biologic markers, developmental milestones and quality of life.

The ECUR-506 programme represents the first time that the ARCUS nuclease has been used to provide an in vivo insertion point for a functional gene in the clinic.

“This milestone is the culmination of over eight years of pre-clinical research in my laboratory addressing gene editing strategies for severe rare liver metabolic diseases,” said James Wilson, Rose H Weiss Professor and Director, Orphan Disease Center; Professor in the Departments of Medicine and Pediatrics, Perelman School of Medicine; and Director of the Gene Therapy Program (GTP) at the University of Pennsylvania, whose laboratory developed ECUR-506 as a novel gene editing approach to treat OTC deficiency.

Diana Spencer, Senior Digital Content Editor, DDW

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