On May 2, 2023 Kelonia Therapeutics, a biotech company revolutionizing in vivo gene delivery, reported that it will share initial preclinical data on its in vivo Gene Placement System (iGPS) technology during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting, occurring May 16-20, 2023, in Los Angeles (Press release, Kelonia Therapeutics, MAY 2, 2023, View Source [SID1234630872]).
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The preclinical data presented will demonstrate that Kelonia’s iGPS technology uniquely enables safe and efficient in vivo generation of highly efficacious CAR T cells. In a humanized mouse model of multiple myeloma treatment, when an anti-BCMA CAR transgene was delivered directly in vivo using iGPS particles, low intravenous dose levels resulted in complete initial tumor regression comparable to ex vivo-manufactured CAR T cells. However, functional CAR T cell persistence after iGPS particle treatment resulted in significantly longer tumor control compared to ex vivo-generated CAR T cells. Despite efficient in vivo CAR gene transfer to T cells, no concerning or unexpected "off-target" transduction was observed including in vital, progenitor or reproductive organs.
"CAR T cells have been transformative for patients suffering from lethal cancers. But manufacturing unique CAR T cells for every patient has limited access to this incredible medicine. The preclinical data we’ll present at ASGCT (Free ASGCT Whitepaper) demonstrates the potential for potent, durable and safe CAR T cells generated by our iGPS in vivo delivery technology, without the requirement for preparative chemotherapy," said Kevin Friedman, Ph.D., Founder, President, and Chief Scientific Officer, Kelonia Therapeutics. "This is the first step toward our vision of solving the central challenge of genetic medicines: democratizing and enabling access when and where patients need them. We look forward to sharing detailed data at the conference."
Details for the oral presentation are as follows:
Abstract Title: Potent in vivo transduction by iGPS particles generates CAR T cells with durable anti-tumor activity
Presenter: Shannon Grande Contrastano, Ph.D., Senior Director, Preclinical Sciences, Kelonia Therapeutics
Session Date/Time: Wednesday, May 17, 2023: 3:45-5:30 PM PDT
Abstract number: 90