iECURE emerges with $50M to search for ‘holy grail’ and ‘untapped frontier’ of gene editing – FierceBiotech

iECURE is emerging from stealth with $50 million and a mission to go after the "holy grail" of gene editing with an in vivo approach that is mutation agnostic.

The biotech will use the funds for a slate of gene insertion programs for liver disorders out of the lab of gene therapy pioneerJim Wilson, M.D., Ph.D. at the University of Pennsylvania.

Versant Ventures, which has backed gene-editing companiesCRISPR Therapeutics and Graphite Bio,is launching iECURE alongside OrbiMed Advisors. The series A gives the biotech access to up to 13 programs out of Wilson's Gene Therapy Program at Penn.

Wilson's lab will conduct the preclinical, toxicology and manufacturing supportand then iECURE will sign the regulatory filings and move the assets through the clinic and onto the market, CEO JosephTruitt told Fierce Biotech.

Its a much more mature company than you would typically start off with if you were just licensing some technology from an academic center," Truitt said. By getting access to Wilson's team, iECURE doesn't have to recruit dozens of employees, the CEO said.

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Wilson's lab has already shown long-term evidence of genome editing in non-human primates, which is important because translating into species larger than mice is often difficult, said Tom Woiwode, Ph.D., iECURE chairman and Versant managing director, in a joint interview with Truitt and Wilson.

Wilson has thought of the liver as a great target for gene therapy since the late-1980's, when he was in the lab as a post-doc, because "so many diseases have their root in the liver [and] it's accessible to biologics." Wilson is also director of the Orphan Disease Center and a professor in medicine and pediatrics at Penn's School of Medicine.

iECURE and Wilson's lab are approaching the "holy grail of genome editing" by using an in vivo approach that inserts genes inside organs, inside the human body, Woiwode said. In another adventurous analogy,Woiwode said the company is also going after the "untapped frontier of gene editing" by using a mutation agnostic approach. This is critical given many genetic diseases have multiple, "sometimes hundreds," of individual mutations. That many targets would not lend well to creating therapies at scale, the chairman added.

The series A will help iECURE bankroll the first three indications in the program of up to 13, Truitt said. More indications down the pipeline are still under evaluation. The financing is slated to take iECURE through the early part of 2023, the CEO said.

The first program goes after familial hypercholesterolemia, or high cholesterol caused by abnormalities in the gene PCSK9. The treatment reduced PCSK9 protein levels by 85% and LDL cholesterol by 56% in nonhuman primates for three years, Wilson's lab reported in Molecular Therapy in February.

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The lab has been working with Precision Biosciences on that asset, which is ready to enter the clinic.As part of iECURE's deal with Wilson's lab, iECURE will conduct the phase 1 study and then return the program to Precision, Truitt said.

The next two indications in the pipeline are the rare metabolic disorders phenylketonuria and ornithine transcarbamylase deficiency, Truitt said.

The gene-editing technology used by iECURE and Wilson's lab is delivered viaadeno-associated virus, or AAV. Toxicity and safety issues have arisen with AAV gene therapies, which resulted in an FDA advisory committee meeting last week to discuss and make recommendations on animal and human testing with these therapies.

Wilson took part in two panels during thetwo-day meeting and said he doesn't see the results of that meeting having an impact on iECURE.

"In terms of the work with IECURE, there really isnt anything that I heard at the committee that impacts on our preclinical plan, our clinical plan or our discussions with health authorities," Wilson said.

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iECURE emerges with $50M to search for 'holy grail' and 'untapped frontier' of gene editing - FierceBiotech