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04-20-22-gene-therapy-program-jesse-zhang
Penn's Gene Therapy Program on April 20, 2022. Credit: Jesse Zhang

Philadelphia-based gene-editing startup iECURE, which is partnered with Penn’s controversial Gene Therapy Program, recently raised $65 million to fund their clinical research.

iECURE, which was co-founded by GTP’s James Wilson, announced on Wednesday that the company has extended its Series A venture funding round at a time when other biotech companies are struggling with financing. A total of $115 million raised will support clinical testing on iECURE’s research programs, which seek to develop gene-insertion therapies to treat rare pediatric liver diseases.

Wilson, who serves as chief scientific advisor of iECURE and holds equity stakes in the company, is a Penn Medicine physician and professor who heads GTP.

In November 2021, The Daily Pennsylvanian uncovered GTP’s alleged toxic workplace environment based on the testimonies of 11 current and former employees. In April, internal reports claimed that top University officials manipulated an investigation into workplace abuse allegations at GTP in an attempt to protect its financial interests and Wilson.

iECURE CEO Joseph Truitt disagreed with the allegations about the workplace environment at the Wilson lab, describing iECURE’s collaboration with GTP as a positive one. 

“It’s been a very productive relationship with incredibly talented staff,” Truitt told BioPharma Dive.

The startup is investigating a drug candidate, called GTP-506, designed to treat infants with ornithine transcarbamylase deficiency. This rare genetic condition is characterized by an increased amount of ammonia in the blood. High levels of ammonia puts patients at risk for severe and permanent brain damage.

Current treatments for OTC deficiency include dialysis, diet restrictions, and liver transplants. However, there are currently no drugs specifically approved to treat OTC deficiency. iECURE’s drug candidate could potentially offer a new form of long-term treatment through in-vivo gene editing. With this technique, the drug can target a specific DNA site and insert a healthy copy of the mutated OTC gene into the patient’s chromosome.

iECURE launched in Philadelphia in September 2021 in partnership with GTP after having secured $50 million in Series A funding. It aims to develop and commercialize gene therapies that treat patients with inherited genetic diseases. In the past, the GTP lab studied genetic treatments for OTC deficiency and ARCUS, the gene-editing technology used in iECURE’s new drug.

This most recent round of financing for iECURE was led by investors Novo Holdings A/S and LYFE Capital.