Research & Development
iECURE Raises USD 65m to Advance In Vivo Gene Editing Programs for the Treatment of Rare Pediatric Liver Diseases
5 December 2022 - - US-based gene editing company iECURE has closed a USD 65m series A-1 financing, the company said.

The series A-1 financing was co-led by Novo Holdings A/S and LYFE Capital with significant participation from existing investors Versant Ventures and OrbiMed Advisors.

This financing, coupled with the USD50 m raised in the prior series A financing announced in September 2021, will bring the company's total funds raised to USD 115m.

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.

Additionally, Tal Zaks, M.D., Ph.D., has been appointed to iECURE's board 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.

The company expects the proceeds from the series A-1 financing to enable the advancement of the company's lead program, GTP-506, including funding IND-enabling studies, starting clinical trials (subject to regulatory approval), and achieving early human data readouts.

In addition, the series A-1 financing is expected to fuel further progress on iECURE's portfolio of gene editing products for the treatment of patients with rare liver diseases, including citrullinemia type 1 (CTLN1) and phenylketonuria.

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. iECURE licensed the ARCUS nuclease for GTP-506 from Precision BioSciences.

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

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 Novo Nordisk Foundation's assets.

LYFE Capital is a global healthcare investment platform.

Precision BioSciences, Inc. is a clinical stage biotechnology company focused on improving life (NASDAQ: DTIL) with its novel and proprietary ARCUS genome editing platform.

The University of Pennsylvania and Dr. Wilson each hold equity interests in iECURE. Penn also receives significant sponsored research support from the company, and both Penn and Dr. Wilson stand to benefit from licensing revenues received from iECURE based on successful technology development and commercialization of the technologies licensed from Penn. Dr. Wilson serves as chief scientific Advisor for iECURE.
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