Treatment uses immune cells engineered in body, rather than lab
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Kernal Biologics has been awarded up to $48 million in funding to accelerate the development of KR-402, a next-generation CAR T-cell therapy for multiple sclerosis (MS) and certain blood cancers.
The funds come from the Advanced Research Projects Agency for Health’s (ARPA-H) EMBODY program, which aims to support the development of more affordable immune therapies where immune cells are transformed inside the body (in vivo) rather than in a lab. Kernal is developing one of these therapies.
CAR-T cell therapies involve collecting a patient’s immune T-cells, engineering them in a lab to boost their ability to recognize and destroy disease-causing cells, and reinfusing them back into the patient’s bloodstream. While highly effective, these therapies are expensive to produce and take time to manufacture. They are also available only at specialized centers.
“Manufacturing [current] CAR-T therapies is a complex and expensive process,” Burak Yilmaz, president of Kernal Bio, said in a company press release. “However, with our proprietary platform, there is a potential of reducing the cost of manufacturing in vivo CAR T-cell therapies by as much as 100-fold.”
Current therapy also requires chemotherapy drugs, which “carry significant toxicity, making these therapies viable for just a small group of patients,” Yilmaz said. “We believe that with our technology and the support of our partners and ARPA-H, we can greatly transform access to this category of therapies.”
“We’re honored to join the elite cohort of ARPA-H awardees,” said Yusuf Erkul, MD, cofounder and CEO of Kernal. “Current CAR-T therapies heralded a true revolution in cancer treatment. Yet, they have their limitations, including a three-week vein-to-vein turnaround time … and side effects such as cytokine release syndrome or secondary T-cell malignancies. At Kernal Bio, we believe that we have the tools to evolve the CAR-T modality towards in vivo therapies.”
MS is caused by the immune system mistakenly attacking healthy parts of the brain and spinal cord. B-cells, which normally produce antibodies to fight off infections, are key immune cells involved in this attack, and are also involved in certain blood cancers.
By targeting and depleting harmful B-cells, KR-402 aims to both suppress the autoimmune attacks that drive MS and eliminate the malignant cells in B-cell blood cancers.
The next-generation therapy uses Kernal’s mRNA 2.0 platform, where a messenger RNA (mRNA) molecule containing the instructions for making a chimeric antigen receptor (CAR) that targets B-cells is delivered to patients.
An mRNA molecule is one cells naturally make when reading genetic instructions that is then used as a template to make proteins. In KR-402, the mRNA molecule is inserted inside lipid nanoparticles that are highly specific to T-cells, ensuring that the therapy is selectively delivered to those cells. A second key feature in KR-402’s design is that the treatment is active only inside those cells, further reducing the potential for affecting other cells.
Because KR-402 works directly inside the body, it eliminates two major steps required for conventional CAR-T therapies: the collection and reinfusion of engineered T-cells, and the use of chemotherapy to deplete white blood cells before treatment to support CAR T-cell expansion and survival. The company expects this to reduce costs and improve the experience for patients.
The $48 million grant will be used to create mRNA molecules for in-body CAR-T therapies, develop new manufacturing strategies for these therapies, and create preclinical models to evaluate the treatments’ safety and effectiveness.
Kernal said it will collaborate with researchers at the Stanford University School of Medicine, Dana-Farber Cancer Institute, and the Jackson Laboratory to conduct the studies.
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