The mRNA Monthly—July 2024 Newsletter


Phase 2b Autoimmune CAR-T Win

Congratulations to Cartesian Therapeutics on their Phase 2b autoimmune CAR-T trial success—shifting the intensity of the cell therapy paradigm! This milestone is a testament to their innovation. It’s exciting to see mRNA as part of that approach, stirring hope for those suffering from autoimmunity.

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Accelerating LNP Development for mRNA Delivery Using AGILE 

In a world where the potential of mRNA medicines is partially constrained by the ability to functionally deliver mRNA payloads to the right cells, this study shows that artificial intelligence, in the form of deep-learning, can accelerate the discovery of novel lipid nanoparticle chemistries. Further, the approach helps to predict the biological activity of these chemistries which will reduce the reliance on animal studies and speed up the discovery of new LNPs, potentially unlocking new disease targets for mRNA. 

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mRNA Vaccine for Alzheimer’s Disease 

This pilot study compares the immunogenicity of potential pDNA to mRNA therapeutic vaccines for Alzheimer’s Disease in mice and non-human primates. The mRNA fares quite well to a clinical pDNA vaccine, creating hope that mRNA may one day play a part in protecting patients suffering from neurodegenerative disease. Vernal is honored to have played a small but important part in this research. 

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GSK Stepping Up: mRNA Vaccinations 

GSK, a British pharmaceutical and biotechnology company, headquartered in London, continues to be enthusiastic about mRNA vaccines. 

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New Initiative: mRNA Vaccine Development Against H5N1

The World Health Organization (WHO) and the Medicines Patent Pool (MPP) have launched a new initiative to advance mRNA vaccine development against human avian influenza (H5N1). This project aims to enhance pandemic preparedness by developing vaccine candidates and transferring the technology to low- and middle-income countries. This effort reflects the WHO’s commitment to equitable access to health technologies and strengthening global response capabilities for future pandemics.

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FDA Guidance: Platform Technology Designation Program for Drug Development 

The FDA’s new guidance clarifies eligible Platform Technologies must adhere to the statutory definition and require consistent CMC processes. Applicants can recycle features across products, while those lacking assets can leverage CDMOs like Vernal Biosciences for standardized processes. Vernal’s CEO, Christian Cobaugh, Senior Director Commercial & Technical Operations, Grant Henderson and guest contributor, Khaled Yamout share their thoughts in this blog article.

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When Does mRNA Purity Matter?  

We want to hear your opinion. When does mRNA purity matter? Answer this open-ended question however you interpret it by submitting this form. 

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