Precision Biosciences Inks Gene Editing Deal With iECURE

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Durham-based Precision Biosciences has inked a deal with gene editing company iECURE to advance one of Precision’s drug candidates into Phase 1 clinical studies. The agreement also gives iECURE a license to develop four gene insertion programs that are based on Precision’s proprietary ARCUS gene-editing platform.

In return, Precision – which specializes in gene editing – will receive an equity stake in iECURE and is eligible for milestone and royalty payments on sales of products developed with the ARCUS technology.

ARCUS was discovered and developed by Precision scientists. It uses DNA-cutting enzymes – or nucleases – to insert, remove or repair the DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-Crel, which evolved in a certain type of algae.

Under the agreement, iECURE plans to file a Phase 1 clinical trial application as early as 2022 for Precision’s PBGENE-PCSK9 candidate to treat familial hypercholesterolemia (FH). Precision will retain the rights to PBGENE-PCSK9. FH is a genetic disorder, resulting from a defect on chromosome 19, that causes very high levels of LDL (bad) cholesterol.

In addition, Precision has granted iECURE a license to use its PCSK9-directed ARCUS nuclease to develop treatments for four other rare genetic diseases. The initial focus will be on liver disorders.

Precision Biosciences entrance
Precision Biosciences headquarters. -- Precision photo

“We founded iECURE with the aim of focusing on genetic diseases with significant unmet need that we could target in a mutation-agnostic manner,” said the company’s CEO Joe Truitt. “After evaluating different gene editing technologies and platforms, we believe gene editing with ARCUS, including use of the uniquely designed ARCUS nuclease as a gene insertion tool targeting the PCSK9 gene, will help us rapidly advance several candidates to the clinic with the potential to deliver on the promise of highly efficient, specific, and safe insertion.”

Matt Kane, CEO and co-founder of Precision, said “gene editing promises to fundamentally reshape the treatment landscape across numerous therapeutic categories. We are excited to announce a new collaboration with iECURE, which we expect to help us expedite clinical validation of the ARCUS platform for both gene knockout and gene insertion.”

Precision said it expects three of its preclinical programs to advance to investigational new drug (IND)/ clinical trial application (CTA) status in the next three years:

  • PBGENE-PCSK9 for familial hypercholesterolemia (by iECURE).

  • PBGENE-PH1 for primary hyperoxaluria (PH) type 1. PH is a group of rare genetic metabolic disorders characterized by the accumulation of a substance known as oxalate in the kidneys and other organ systems.

  • PBGENE-HBV for chronic hepatitis B.

About Precision Biosciences

Precision is a clinical-stage biotechnology company that was spun out of Duke University in 2006 and went public in March 2009. Its shares are listed on the Nasdaq Global Select Market under the ticker symbol “DTIL,” shorthand for the company’s marketing tagline, “Dedicated to improving life.”

Precision has close to 225 employees. Its pipeline includes several “off-the-shelf” CAR T immunotherapy clinical and in vivo gene-correction therapy candidates. They are targeted at curing genetic and infectious diseases for which no adequate treatments exist.

About iECURE

iECURE is a newly emerging in vivo gene editing company focused on curing devastating diseases with high unmet need. The company was co-founded by University of Pennsylvania gene therapy pioneer James M. Wilson, M.D., Ph.D. The Penn spinout works in close partnership with the school’s Gene Therapy Program.

iECURE announced September 9 that it had secured $50 million in Series A financing to advance its gene editing and in vivo delivery pipeline.

Bryant Haskins, NCBiotech Writer
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