Illustration of DNA helices undergoing gene or base editing on a blue background
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iECURE, a gene editing startup focused on liver disorders, this week announced a $65 million Series A-1 financing. The company is collaborating with the University of Pennsylvania’s Gene Therapy Program (GTP) led by James M. Wilson, MD, PhD, who led the gene-therapy trial that ended with teenager Jesse Gelsinger’s death in 1999. This event let to a multi-year investigation and congressional hearings.

But Wilson rebounded from the Gelsinger tragedy to fuel a powerhouse of gene therapy discovery. At least 40 companies are using AAVs that fall under Wilson’s patents, accounting for nearly 100 drug development programs.

iECURE’s lead treatment is GTP-506, which relies on the delivery of twin adeno-associated virus (AAV) capsids carrying different payloads. Those are an ARCUS nuclease vector (GTP-506A) targeting gene editing in the PCSK9 gene locus and a therapeutic donor vector (GTP-506D) that inserts the OTC gene to provide the correction. iECURE licensed the ARCUS nuclease for GTP-506 from Precision BioSciences.

The lead indication is ornithine transcarbamylase (OTC) deficiency, a rare genetic condition characterized high levels of ammonia in the blood that cause severe and permanent brain damage. The severe form of the condition emerges shortly after birth and the only treatment for early onset severe OTC deficiency is a liver transplant. iECURE says GTP-506 could provide long-term treatment through in-vivo gene editing.

The FDA has granted both Orphan Drug Designation and Rare Pediatric Designation to GTP-506 for OTC deficiency.

The financing was co-led by Novo Holdings A/S and LYFE Capital with participation from existing investors Versant Ventures and OrbiMed Advisors. This latest financing, coupled with the $50 million raised in the prior Series A financing announced in September 2021, will bring the company’s total funds raised to $115 million.

“In the last year, we have made significant progress in both advancing development of our lead program for neonatal onset OTC and building a world-class team with an extensive track record in developing and commercializing novel therapies,” said Joseph Truitt, Chief Executive Officer of iECURE. “We believe that this funding will enable us to execute all the tasks necessary to begin clinical development of what could be the first mutation-agnostic in vivo gene insertion therapeutic program.”

The company expects the proceeds from the Series A-1 financing to enable the advancement of the GTP-506, including funding IND-enabling studies, starting clinical trials (subject to regulatory approval), and achieving early human data readouts. In addition, the financing is expected to fuel further progress on iECURE’s portfolio of gene editing products for the treatment of patients with other rare liver diseases, including citrullinemia type 1 and phenylketonuria.

“iECURE, through its collaboration with the University of Pennsylvania Gene Therapy Program, has generated impressive pre-clinical data demonstrating durable gene integration in non-human primates with its cutting-edge approach to mutation-agnostic in vivo gene editing. We are excited to work closely with the company as they the move their programs into the clinic,” said Ray Camahort, PhD, Partner at Novo Ventures.

The death of Jesse Gelsinger is not the only setback Wilson has faced in his long career at Penn, which started in 1993 when he was recruited to lead the newly founded Institute for Human Gene Therapy there. Earlier this year, leaked reports alleged that top officials from the University of Pennsylvania “manipulated” an investigation into workplace abuse charges in Wilson’s Gene Therapy Program to protect the University and Wilson.

As part of iCURE’s Series A-1 financing, Ray Camahort, PhD, Partner at Novo Ventures and Derek Yuan, PhD, Managing Director at LYFE Capital will join iECURE’s Board of Directors. Additionally, Tal Zaks, MD, PhD, has been appointed to iECURE’s board as OrbiMed’s new representative on the Board of Directors. Stephen Squinto, PhD, will continue to serve on the Board of Directors as an independent member.

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