Kelonia Therapeutics, a biotechnology company working on in vivo gene delivery, has announced the results of preclinical research demonstrating that its in vivo Gene Placement System (iGPS) technology efficiently delivers specific CAR molecules to T cells at therapeutic dose levels in mice and non-primates. human (NHP).
The Kelonia iGPS platform enables CAR T-cell therapy without the need for lymphodepleting chemotherapy or time-consuming ex vivo manufacturing. These data demonstrate the potential of iGPS particles to become a highly effective, safe, “off-the-shelf” therapy for patients with multiple myeloma, which Kelonia is pursuing as its primary indication.
Kelonia shared the data during an oral presentation at the 26th annual meeting of the American Society of Gene & Cell Therapy (ASGCT) in Los Angeles.
“In just one year since launch, the outstanding Kelonia team has demonstrated the potential of our iGPS technology to provide precise, efficient and safe delivery of the CAR gene directly to multiple myeloma patients intravenously and as a ready-to-use therapy that does not require preparative chemotherapy. for strong CAR T cell activity,” said Kevin Friedman, founder, president, and chief scientific officer at Kelonia Therapeutics.
“Cancer patients are waiting for highly effective drugs without severe toxicity or manufacturing complexities that limit the accessibility and impact of existing CAR T cell therapies. Today, we have shown convincing 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 toward the clinic.”
Intravenous infusion of NHPs with surrogate iGPS particles expressing anti-CD20 CAR in the absence of preparative chemotherapy resulted in robust CAR T-cell activity over the 10x dose range. No evidence of clinical toxicity or biomarkers, including cytokine release syndromes or neurotoxicity, was observed even at the highest dosage levels.
Strong anti-tumor activity was observed with iGPS particles expressing anti-BCMA CAR in a mouse model 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 mice and NHP, therapeutic dose levels of iGPS particles specifically transduced T cells and showed no evidence of unexpected “off-target” transduction, including progenitor cells or vital and reproductive organs.
There are other companies working on multiple myeloma: earlier this year, GPCR Therapeutics launched a multiple myeloma trial. And last year, Oncopeptides received a grant for the project to engage NK (natural killer) cells in multiple myeloma.