Few dispute that the rapid development and approval of two mRNA vaccines against COVID-19 was an unmitigated success, or that the evolving field of gene therapy has delivered multiple successful treatments for a range of rare diseases. Still, genomic medicines is a field that’s ripe for further innovation, in areas ranging from the development of improved delivery vehicles to the streamlining of manufacturing. Three areas of genomic medicines could benefit greatly from an integrated approach to development: gene editing, gene therapy and mRNA medicines. All of these modalities hold great potential in the development of innovative therapies to treat cancer, autoimmune disease, rare inherited diseases and more. Engaging partners early in the development process is critical to assembling cutting-edge tools to enable efficient end-to-end development and manufacturing. Customizing gene editing for improved efficiency CRISPR gene editing is being developed as a treatment for a range of applications, including CAR-T cells for cancer, gene-edited stem cells to treat diabetes and ex-vivo gene editing to treat the blood disorder beta thalassemia. Integrated DNA Technologies (IDT)* can customize guide RNAs for common CRISPR research applications, as well as emerging gene-editing approaches like prime editing and research using the Cas13 enzyme. IDT also offers the Alt-R™ CRISPR-Cas9 System with the HiFi CRISPR-Cas9 nuclease, allowing for more precise gene editing with less of the off-target activity that can be a concern in gene editing. Aldevron offers additional research-grade and GMP versions (SpyFiTM Cas9 Nuclease), which can help accelerate experimental therapies. Aldevron has partnered with external partners as well and can offer a full library of novel CRISPR nucleases to qualified customers, such as Eureca-V™ Nuclease, the wild-type MAD7™ CRISPR Type-V nuclease from Inscripta. Improving the delivery of gene therapy Lipid nanoparticles (LNPs) are widely used to deliver mRNA, CRISPR components and other nucleic acids into cells, but developing the LNP chemistry and process is challenging. Precision NanoSystems (PNI) has advanced technologies that can ease the process of developing and manufacturing LNPs. Its GenVoy Delivery Platform includes a novel lipid library and LNP reagents optimized for key applications including vaccines, gene editing and cell therapies. And researchers can use PNI’s desktop NanoAssemblr® platform with NxGen™ microfluidics to make well-formed, stable LNPs using a production process that is scalable from small batch discovery research to GMP production. The viral vectors used to deliver gene and cell therapies are also in high demand, intensifying the need for technologies that can ease development and manufacturing. Aldevron offers a range of plasmids essential for the development of viral vectors. And with the help of some key partnerships, Danaher’s life sciences compan...
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