Cell therapy started out as highly individualized, with treatments that take a patient’s own immune cells and engineer them in the lab. Efforts are being made to develop established cell therapies, but these therapies made from donor cells are not personalized and may trigger an immune response. Kelonia Therapeutics proposes an alternative: an off-the-shelf cell therapy that is also personalized to the patient. The biotech company aims to provide both functions through technology that enables cell engineering to occur in patients.
“We’re solving a problem that no one else can currently solve — gene delivery in vivo,” said Kevin Friedman, president and chief scientific officer of Kelonia.
The Cambridge, Massachusetts-based startup recently emerge from invisibility The $50 million financing brings its research closer to a clinical trial. The Series A round included participation from Alta Partners, Horizon Ventures and Venrock.
Cells used in cell therapy are engineered with lentiviruses that deliver genetic payloads. In CAR T therapy, this genetic payload causes cells to express a receptor that targets a protein on cancer cells. The multi-step process of making these therapies is expensive, laborious and time-consuming. It can take a month or more to convert a patient’s immune cells into CAR T therapy.
Kelonia builds on work done using lentiviruses. The problem with current lentiviral technology, Friedman said, is that the virus binds to receptors that are expressed on a large number of tissues. This means that some of them can go to unintended targets, leading to toxic effects. Kelonia’s technology enables targeted delivery. The technique modifies the envelope of the lentivirus, making it “off-target,” Friedman said. The virus is then decorated with antibodies that redirect the virus. These viruses are injected into the body, carrying their genetic material into the desired cells. The cells then express therapeutic proteins, such as cancer-targeting receptors. Kelonia’s approach avoids the multi-step manufacturing process required by currently available cell therapies, Friedman said.
“We’re eliminating all of that,” Friedman said. “We’re eliminating that and finally getting a personalized medicine.”
Kelonia’s detargeting technology comes from the MIT lab of bioengineering professor Michael Birnbaum, the startup’s co-founder.He is one of the authors of the research paper post Detargeting and redirection of lentivirus-like particles was described last month in Nature Methods.
Kelonia is advancing with two partners to help the biotech company develop new treatments. Antibody specialist Adimab will discover antibodies that decorate the lentiviral envelope, directing it to the desired cells. Lentiviral particles will be produced by ElevateBio at its facility. Kelonia will co-develop the manufacturing process with ElevateBio. Freedman said the partnership with ElevateBio will shorten the typically long lead times for transferring technology from a process discovery lab to a GMP facility.
The first CAR T therapy to hit the market was for blood cancers. Kelonia, which also has blood cancer as its first indication, aims to use its technology to provide a ready-made CAR that avoids the adverse effects associated with current treatments. These risks include brain toxicity and a dangerous immune response called cytokine release syndrome. Friedman believes that Kelonia’s method of producing cancer-fighting therapies in patients should be safer, but the company certainly needs to prove it in clinical trials.
Friedman declined to provide a timeline for starting human testing, but said that by bringing the technology to the clinic quickly, Kelonia will move beyond ex vivo cell therapy approaches while generating clinical data to learn more about the platform Lay the foundation. therapy. New indications can be realized simply by changing the cargo and designing lentiviruses for delivery to different tissues. In addition to CARs, Friedman said, Kelonia technology could also be applied to T-cell receptors or a range of gene cargoes. Beyond cancer, the biotech company aims to develop its technology for delivery to nerve, muscle and kidney tissue.
In addition to the manufacturing advantages, Friedman said, Kelonia’s technology offers the potential to improve the accessibility of these drugs. No complicated manufacturing process is required, and Kelonia will not need to be administered only in major medical centers. It can also be offered in community hospitals, “with incredible clinical benefit for patients no matter where they are,” Friedman said.
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