On November 8, 2024 NanoCell Therapeutics, Inc. ("NanoCell"), a company developing a non-viral, DNA-based in vivo gene therapy platform, reported animal proof of concept data from its lead program utilizing targeted lipid nanoparticle (tLNP) technology for in vivo CAR-T cell generation (Press release, NanoCell Therapeutics, NOV 8, 2024, View Source [SID1234648032]).

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"The studies demonstrated successful targeting and stable CAR expression in a human PBMC engrafted mouse model evaluating the company’s tLNP technology. These findings were initially shared at the 31st Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Rome last month. Additional data are being presented at the SITC (Free SITC Whitepaper) 39th Annual Meeting in Houston (Abstract #284)]"

These preclinical data demonstrate the potential of our cell-directed, lipid-enabled delivery platform," said Maurits Geerlings, CEO of NanoCell. "As we progress from platform validation, we are advancing our lead clinical construct, a dual-CAR CD19/CD22 targeting B cell malignancies and autoimmune diseases."

"Our proof of concept studies demonstrate that our cell-directed, lipid-enabled delivery system can achieve stable CAR expression in T cells in vivo," said Dr. Jacek Lubelski, Chief Technology Officer of NanoCell. "These results prove the mechanism of action for our non-viral vector technology approach."

Click here to view Abstract #284

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