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.
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PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.
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