iECURE Partners to Develop Next Generation Liver-Targeted Lipid Nanoparticles with the University of Pennsylvania


Expands indications for iECURE focused on liver to 15

PHILADELPHIA CREAM – (COMMERCIAL THREAD) – iECURE, an agnostic mutation in vivo gene-editing company striving to cure devastating diseases with significant unmet needs, today announced that it has entered into an exclusive agreement with the University of Pennsylvania (Penn) to develop lipid nanoparticles (LNP ) new generation for liver gene editing applications. This LNP technology originated from the laboratory of Michael Mitchell, Ph.D.

“Our focus on gene insertion approaches to correct liver disorders is based on the most robust delivery of gene-editing enzymes for application at your fingertips,” said Joseph Truitt, CEO of iECURE . “Having the adaptability to move beyond AAV to provide the gene editing payload is important, particularly when we are exploring the indications for adults or if using an editing technology of alternative genes provides more solid data. With this in mind, this collaboration with Dr. Mitchell’s lab is essential as we define our strategic path for liver-focused gene editing therapies.

iECURE will hold certain exclusive rights to gene editing in the liver with LNP technology developed in collaboration with Dr Mitchell. In addition, the agreement brings the number of potential pipeline programs to be developed by iECURE with Penn from 13 to 15.

Dr Mitchell is the Skirkanich Assistant Professor of Innovation in the Department of Bioengineering, Faculty of Engineering and Applied Sciences, University of Pennsylvania. Dr. Mitchell’s lab is a world leader in the discovery and development of biomaterials to overcome biological barriers to drug delivery, with a particular focus on the development of next-generation LNP for the delivery of mRNA, d ‘SiRNA and gene editing therapies. The lab collaborated for several years with the Penn Gene Therapy Program (GTP), led by James M. Wilson, MD, Ph.D .; this collaboration will continue within the framework of the Penn-iECURE partnership. Prior to joining Penn in 2018, Dr Mitchell was a Postdoctoral Fellow at the National Institutes of Health (NIH) in the lab of Robert S. Langer, Sc.D., of the Massachusetts Institute of Technology.

“As gene editing comes to the fore for correcting genetic diseases, scientists will discover new editing technologies that may require other means of delivery,” Dr. Mitchell noted. “Our lab has been working on the development of non-viral vectors for delivering nucleic acids, and we find that LNP technologies work very well to provide a range of gene editing technologies. We are delighted to collaborate with the iECURE team.

IECURE’s initial internal development programs are based on the use of endonuclease-based gene editing technology delivered via AAV for rare neonatal indications. Subsequent pipeline candidates may use LNPs for the delivery of other gene editing technologies.

Financial Information: The University of Pennsylvania and Dr. Wilson hold interests in iECURE. University of Pennsylvania Dr Wilson and Dr Mitchell receive company sponsored research support and will be entitled to receive licensing income from iECURE based on the successful technology development and commercialization of the technology under Licence. Dr Mitchell is Scientific Advisor for iECURE.

About iECURE

iECURE is a mutation agnostic in vivo gene-editing company striving to cure devastating diseases with significant unmet needs. We are advancing our pipeline in close partnership with the world-class translation engine of the University of Pennsylvania’s Gene Therapy Program. Using in vivo In editing, our methods focus on inserting functional genes into patient genomes, providing long-term stable expression of these genes. With the proven track record of our team, along with the deep expertise of the University of Pennsylvania and the driving force behind translational genetic medicine, we believe it is now possible to turn the tide of these devastating diseases. For more information visit www.iECURE.com and follow LinkedIn.

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