iECURE Raises $65 Million to Advance In Vivo Gene Editing Programs for the Treatment of Rare Pediatric Liver Diseases

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PHILADELPHIA–(BUSINESS WIRE)–iECUREa gene-editing company focused on the development of mutation-agnostic empathize gene insertion, or knock-in, editing therapies for the treatment of liver disease with significant unmet need, today announced the completion of a $65 million Series A-1 financing. The Series A-1 financing was jointly led by Novo Holdings A/S and LYFE Capital with significant participation from existing investors Versant Ventures and OrbiMed Advisors.

This financing, combined with the $50 million raised in the previous Series A financing announced in September 2021, will bring the company’s total amount raised to $115 million. As part of the Series A-1 financing, Ray Camahort, Ph.D., Partner at Novo Ventures and Derek Yuan, Ph.D., Managing Director at LYFE Capital will join iECURE’s Board of Directors. In addition, Tal Zaks, MD, Ph.D., has been appointed to iECURE’s Board of Directors as OrbiMed’s new representative on the Board of Directors. Stephen Squinto, Ph.D., will continue to serve on the Board of Directors as an independent member.

“Over the past year, we have made significant progress in both the progressive development of our leading OTC neonatal onset program 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 this funding will enable us to perform all the tasks necessary to initiate clinical development of what is the first mutation-agnostic empathize therapeutic program for gene insertion.”

The Company expects the proceeds from the Series A-1 funding to enable the advancement of the Company’s lead program, GTP-506, including funding IND-supporting studies, initiating clinical trials (subject to regulatory approval) and achieving early readouts of human data. In addition, the Series A-1 funding is expected to further advance iECURE’s portfolio of gene editing products for the treatment of patients with rare liver diseases, including citrullinemia type 1 (CTLN1) and phenylketonuria (PKU).

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

About GTP-506

iECURE’s gene editing approach for its initial programs, including OTC deficiency, is based on the delivery of twin adeno-associated virus (AAV) capsids with different payloads. GTP-506 consists of 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. iECURE has licensed the ARCUS nuclease for GTP-506 to Precision BioSciences.1 The cut in the PCSK9 site serves as the insertion site for the therapeutic gene and provides a potential pathway to permanent expression of a healthy gene. The FDA has granted GTP-506 both orphan drug and rare pediatric status for the treatment of OTC deficiency.

About OTC Deficiency

OTC deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for ammonia detoxification. Individuals with OTC deficiency can build up excessive levels of ammonia in their blood, potentially leading to devastating consequences, including cumulative and irreversible neurological damage, coma, and death. The severe form of the condition develops 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 or preclude the risk of life-threatening symptoms or crises.

About Novo Holdings A/S

Novo Holdings A/S is a private limited liability company wholly owned by the Novo Nordisk Foundation. It is the holding company of the Novo Group, comprising Novo Nordisk A/S and Novozymes A/S, and is responsible for managing the assets of the Novo Nordisk Foundation. Novo Holdings is recognized as a leading international life science investor, with a focus on long-term value creation. As a life science investor, Novo Holdings provides seed and venture capital to development stage companies and takes significant ownership positions in growing and established companies. Novo Holdings also manages a broad portfolio of diversified financial assets. More information: www.novoholdings.dk.

About LYFE Capital

LYFE Capital is a global pioneering healthcare investment platform. We believe that “Healthcare knows no boundaries” and that every new choice we make has the potential to advance healthcare and solve everyone’s unmet needs. LYFE Capital leverages our expertise and global resources to invest in and create value for healthcare companies around the world. Our experienced team has extensive knowledge of healthcare around the world. Management teams value our credibility as we guide them to maximize their potential in a dynamic global marketplace. More information: https://www.lyfecapital.com/.

About iECURE

iECURE is a gene editing company focused on developing therapies using mutation-agnostic empathize gene insertion, or knock-in, editing for the treatment of liver disease with significant unmet need. We believe our approach has the potential to replace and restore the function of a dysfunctional gene by engaging a healthy copy, regardless of the mutation, to provide sustained gene expression and long-term, potentially curative, therapeutic benefit. Our management team has extensive experience in conducting global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. We intend to leverage our team’s strengths in research and development strategy to identify what we believe is the most appropriate target and modality for our product candidates to target certain liver diseases. We are partnering with the University of Pennsylvania Gene Therapy Program (GTP), led by James M. Wilson, MD, Ph.D., to leverage GTP’s world-class translational expertise and infrastructure, which has helped generate our first pipeline of potential product candidates . For more information visit https://iecure.com and continued LinkedIn.

About Precision BioSciences & ARCUS

Precision BioSciences Inc. is a clinical-stage biotechnology company focused on improving lives (Nasdaq: DTIL) with its new and patented ARCUS® genome editing platform. ARCUS is a highly accurate and versatile genome editing platform designed with therapeutic safety, delivery and control in mind. Using ARCUS, the company’s pipeline consists of multiple ex vivo “turnkey” CAR T immunotherapy clinical candidates and several empathize candidates for gene editing designed to cure genetic and infectious diseases for which no adequate treatments exist. For more information on Precision BioSciences, visit www.precisionbiosciences.com.

Penn’s financial disclosure

The University of Pennsylvania (Penn) and Dr. Wilson each have equity interests in iECURE. Penn also receives significant sponsored research support from the company, and both Penn and Dr. Wilson will benefit from licensing revenues received from iECURE based on successful technology development and commercialization of the technologies licensed to Penn. Dr. Wilson serves as Chief Scientific Advisor to iECURE.

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1 iECURE has licensed the ARCUS® nuclease from Precision BioSciences for four gene insertion programs including OTC, CTLN1 and PKU.

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