23 August 2022 - - The US Food and Drug Administration has granted Rare Pediatric Disease Designation to US-based gene editing company iECURE's lead product candidate GTP-506 for the treatment of Ornithine Transcarbamylase deficiency, a rare genetic condition that can lead to irreversible neurological impairment, seizures, coma and death, in a pediatric population.

The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States.

The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations.

Under this program, a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product or sold to another sponsor for priority review of their marketing application.

iECURE's approach to gene editing for its initial programs, including OTC deficiency, relies on the delivery of twin adeno-associated virus capsids carrying different payloads.

GTP-506 comprises two vectors, an ARCUS nuclease vector (GTP-506A) targeting gene editing in the well-characterized PCSK9 gene locus and a therapeutic donor vector (GTP-506D) that inserts the OTC gene to provide the desired genetic correction.

The cut in the PCSK9 site serves as the insertion site for the therapeutic gene, providing a potential path to permanent expression of a healthy gene.

OTC deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia.

Individuals with OTC deficiency can build up excessive levels of ammonia in their blood, potentially resulting in devastating consequences, including cumulative and irreversible neurological damage, coma and death.

The severe form of the condition emerges shortly after birth and is more common in boys than girls.

The only treatment for early onset severe OTC deficiency is a liver transplant. Currently available medical therapies do not correct the disease, and do not eliminate the risk of life-threatening symptoms or crises.

iECURE is a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need.

The company is collaborating with the University of Pennsylvania's Gene Therapy Program, or GTP, led by James M. Wilson, M.D., Ph.D., to utilize GTP's translational expertise and infrastructure, which has helped generate our initial pipeline of potential product candidates.

The University of Pennsylvania and Dr. Wilson hold equity interests in iECURE.

Penn also receives significant sponsored research support from the company, and both Penn and Dr. Wilson benefit from licensing revenues received from iECURE based on technology development and commercialization of the technologies licensed from Penn.

Dr. Wilson serves as chief scientific Advisor for iECURE.