Several biotech companies, including Moderna, are exploring the use of mRNA infusions to enhance the body’s immune cells for fighting cancer and other diseases, offering a novel approach to CAR-T cell therapies. Instead of the labor-intensive process of extracting, engineering, and reinfusing a patient’s immune cells, mRNA-based therapies aim to streamline the process while providing similar benefits. This innovative approach involves engineering cells directly within the body, potentially simplifying treatment and reducing side effects. Companies like Carisma Therapeutics, Capstan Therapeutics, and Orna Therapeutics are presenting promising data at medical conferences, though multiple doses may be needed due to the short lifespan of mRNA. Human trials are planned for the future, with potential applications in cancer and autoimmune disease treatment.
Carisma Therapeutics is exploring an innovative approach to cancer treatment by using mRNA-infused lipid nanoparticles in collaboration with Moderna. They have developed engineered macrophages (CAR-M) to target solid tumors, capitalizing on these cells’ ability to infiltrate tumor environments. In a mouse study on metastatic pancreatic cancer, CAR-M therapy effectively reduced cancer spread and controlled metastasis in the livers and lungs. This “Trojan horse” approach could revolutionize cancer treatment. The Carisma-Moderna partnership may lead to milestone payments of around $250 million for up to twelve targets, though human trials’ timing remains undisclosed.
CAR-T cell therapies have demonstrated remarkable success in treating blood cancers like leukemia and lymphoma, but they pose challenges related to cost and accessibility, hindering their expansion into other diseases such as autoimmune conditions. Haig Aghajanian, Capstan’s head of research and co-founder, highlights the access issue, particularly for larger patient populations. Many efforts have aimed to develop off-the-shelf CAR-T therapies using generic stem cells, but success has been limited, with several therapies struggling to provide long-term cancer control. Capstan presented promising early results at a conference, suggesting their treatment could halt tumor growth in mice. The company, born from previous research involving prominent scientists like Carl June, Bruce Levine, and Drew Weissman, the Nobel Prize laureate for his work on mRNA, is developing therapies for cancer and autoimmune diseases, leveraging antibodies in lipid nanoparticles to target specific T cells in the spleen. Clinical trial timelines remain undisclosed.
Orna Therapeutics is pioneering circular RNA, a variation of mRNA, to extend CAR protein expression, potentially reducing cytokine release syndrome risks. In a mouse study, bi-weekly infusions of their therapy maintained nearly undetectable leukemia levels over eight weeks. Orna’s approach is likened to recombinant protein therapy, providing transient effects. Rodent and monkey studies are ongoing, with the company’s first clinical trial for blood cancers scheduled in 2024, aiming for dosing every two to three weeks. Meanwhile, other companies like Umoja Biopharma are exploring in vivo CAR-T approaches using viral vectors, with plans to test blood cancers and gynecologic tumors in the coming years.
Check out our mRNA service to expedite your vaccine research
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
Skyline Therapeutics Receives FDA Orphan Drug Designation for Gene Therapy for Retinitis Pigmentosa
BOSTON & SHANGHAI, September 2, 2024 – Skyline Therapeutics, a gene therapy company focused on innovative treatments for rare and severe diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SKG1108....
Novavax Receives Emergency Use Authorization for Updated Covid-19 Vaccine
Novavax has received emergency use authorization (EUA) from the U.S. Food and Drug Administration (FDA) for its updated Covid-19 vaccine, NVX-CoV2705, joining other vaccine manufacturers in addressing evolving virus variants. The EUA permits the use of Novavax's...
Turn Biotechnologies Reports Historic Skin Cell Rejuvenation Breakthroughs at ESDR this Week
Company will present data from its successful rejuvenation of fibroblasts and keratinocytes to influential gathering of international dermatologists MOUNTAIN VIEW, Calif., Sept. 4, 2024 /PRNewswire/ -- Turn Biotechnologies, a developer of novel mRNA medicines and...
H5 influenza vaccines: What needs to be done to reduce the risk of a pandemic
As the global threat of H5N1 influenza looms with outbreaks across species and continents including the U.S., three international vaccine and public health experts say it is time to fully resource and support a robust strategy to address this and future potential...
Related Services
