Astellas is penning a deal with Kelonia Therapeutics to develop in vivo cell therapies to treat cancer, but with a twist.

Not only would the treatments be off-the-shelf, meaning that they would not have to be tailored to each patient as current CAR-T therapies are, but the two companies plan to engineer them to be “convertible” — the CAR-T cells could be turned on and off using an antibody.

Via its subsidiary Xyphos Biosciences, Astellas will pay Kelonia $40 million upfront with another potential $35 million for a second program. All in all, the deal could be worth nearly $800 million, the two companies announced Thursday evening.

The plan is to develop an in vivo CAR-T program using Kelonia’s delivery technology and Xyphos’ convertible CAR-T platform. The science is still in early stages, and Astellas and Kelonia declined to comment on when a program may enter human trials.

Kelonia, a startup born from research done at Stanford and MIT, launched from stealth in 2022 with $50 million. The biotech company is working on a system to deliver genetic medicines directly into the body via engineered lentiviral vectors. Kelonia CEO Kevin Friedman led the early development of what is now Abecma at bluebird bio. The multiple myeloma CAR-T cell therapy, which is now in the hands of bluebird spinout 2seventy bio and Bristol Myers Squibb, is engineered with lentiviral vectors, but outside of the body.

With in vivo delivery, the instructions for making a CAR would be delivered directly to T cells, using the body’s own machinery to create the cancer-fighting therapies. Friedman said the targeting method the delivery system uses “is like a UPS delivery, where it targets specific cells, specific addresses of interest — here being T cells.”

And in this case, the instructions would be for a convertible CAR that could be switched on or off.

Xyphos president Gary Starling said the goal of the switch is to make a safer CAR-T therapy compared to conventional approaches. Rather than having CAR-T cells that are always on, the convertible approach would enable the CAR-T therapy to only act when the specific antibodies are also dosed.

The FDA has been reviewing the risk of secondary cancers with cell therapies, though the agency’s cell and gene therapy leaders and experts in the field maintain that the benefits of these cancer treatments far outweigh their safety risk. But FDA officials have suggested that the risk may stem from the use of viral vectors in engineering the treatments.

“We believe that as a gene delivery tool, lentiviruses are really second to none when it comes to safety. We only need to treat once and they cause long-lasting durable CAR-T cells,” Friedman said when asked about the risk.

Others in the field are also developing convertible or in vivo CAR-T treatments. AbbVie and Calibr, part of Scripps Research Institute, recently expanded a pact around what they call their switchable CAR-T platform.

AbbVie also has a deal with Umoja Biopharma on in vivo CAR-T therapies. Capstan, Mustang Bio and others are also developing in vivo treatments, though none have entered clinical studies. Umoja has said that it hopes to enter the clinic this year.


About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Related News

Related Services