Preclinical data in mice and non-human primates demonstrate iGPS particles efficiently and specifically deliver CAR molecules to T cells in vivo for durable tumor clearance, without typical toxicities and need for lymphodepleting chemotherapy



Kelonia plans to advance lead program in multiple myeloma towards the clinic



Data shared during oral presentation at ASGCT Annual Meeting

Kelonia Therapeutics, a biotech company revolutionizing in vivo gene delivery, today announced the results of preclinical research demonstrating that its in vivo Gene Placement System (iGPS™) technology efficiently delivered CAR molecules specifically to T cells at therapeutic dose levels in both mice and non-human primates (NHPs). Kelonia’s iGPS platform enables CAR T cell therapy without the need for lymphodepleting chemotherapy or time-consuming ex vivo manufacturing. These data demonstrate the potential for iGPS particles to be a highly effective, safe, “off-the-shelf” therapy for patients with multiple myeloma, which Kelonia is pursuing as its lead indication. Kelonia shared the data for the first time during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 26^th Annual Meeting in Los Angeles.

“In just over a year since launching, the extraordinary Kelonia team has shown the potential of our iGPS technology to provide precise, efficient, and safe CAR gene delivery directly to multiple myeloma patients intravenously and as an off-the-shelf therapy that doesn’t require preparative chemotherapy for potent CAR T cell activity,” said Kevin Friedman, Ph.D., Founder, President, and Chief Scientific Officer, Kelonia Therapeutics. “Cancer patients are waiting for an incredibly effective medicine without the severe toxicities or complicated manufacturing that have limited the accessibility and impact of existing CAR T cell therapies. Today, we’ve shown compelling preclinical evidence that achieving this transformative treatment for multiple myeloma is possible with Kelonia’s iGPS technology, and we look forward to sharing additional information as we progress towards the clinic.”

Highlights from the oral presentation (Abstract 90):

• Intravenous infusion of NHPs with surrogate iGPS particles expressing an anti-CD20 CAR in the absence of preparative chemotherapy resulted in potent CAR T cell activity across a 10x dose range. No clinical or biomarker evidence of toxicities, including cytokine release syndrome or neurotoxicities, was observed even at the highest dose level.
• Potent anti-tumor activity was observed with iGPS particles that expressed an anti-BCMA CAR in mouse models of multiple myeloma. CAR T cells generated in vivo with iGPS particles exhibited prolonged functional persistence and tumor control compared to standard, ex vivo CAR T cells.
• In both mice and NHPs, therapeutic dose levels of iGPS particles specifically transduced T cells and showed no evidence of unexpected “off-target” transduction, including progenitor cells or in vital and reproductive organs.

About Kelonia Therapeutics

Kelonia is pioneering a new wave of genetic medicines using its in vivo gene placement system (iGPS™). The company’s elegant, cutting-edge in vivo gene delivery technology uses an advanced lentiviral vector particle harboring envelope modifications to improve in vivo gene transfer efficiency and tropism molecules to facilitate tissue-specific delivery. Initially focused on developing transformational in vivo CAR T therapies for hematologic cancers, Kelonia is building a pipeline of genetic medicines for a range of diseases, with the bold goal of making genetic medicines accessible to every patient in need, when and where they need them. Learn more about Kelonia at www.keloniatx.com and follow us on LinkedIn and Twitter.

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