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An Integrated Approach to Developing Next-Generation Genomic Medicines August 5,2022.

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 companies work with gene therapy manufacturers to improve the efficiency of their manufacturing processes.


For example, in 2019, Aldevron teamed up with Nature Technology Corporation (NTC), which offers NanoplasmidTM vector technology and antibiotic-free RNA-OUTTM selectable technology, both of which improve the efficiency of viral-vector manufacturing.

On the consumables and equipment front for viral vector manufacturing, Pall Corporation and Cytiva have partnered with Andelyn Biosciences. Andelyn provides feedback to Pall and Cytiva on the performance of consumables, equipment and software and on production yields. Pall and Cytiva can then take that information to make changes in real time to improve customers’ manufacturing processes.


Powering the next generation of mRNA

Encouraged by the success of mRNA vaccines to fight COVID-19, several companies are developing mRNA medicines to prevent other infectious diseases and treat a range of diseases. These also require plasmid DNA backbones.


For example, Aldevron works hand-in-hand with biopharma innovators to provide customized mRNA, as well as high-quality enzymes and linearized plasmid DNA for mRNA synthesis. It’s a one-stop shop for critical reagents, quality control assays, purification processes and other services that can support mRNA development from discovery to clinical testing.


The LNPs used to deliver mRNA used to be made by hand in a cumbersome process, but with PNI’s NanoAssemblr® platform, developers now have an automated solution for making stable LNPs quickly. It’s so fast that one of PNI’s customers reported cutting 10 months from the development time of every drug in its pipeline.


A major bottleneck in mRNA is getting the scale right for commercial manufacturing. This can be a challenge, because the quantities of mRNA can vary widely depending on factors like indication and potency of the medicine. Some products—the COVID vaccines, for example—require enormous production capacity to meet global demand, while others may only need production amounts in the milligrams.


Once mRNA therapeutics are ready for large-scale production, Cytiva can step in with a range of solutions. Cytiva’s FlexFactory™ and KUBio™ are flexible manufacturing systems that can be tailored and delivered to customers. FlexFactory and KUBio can be installed in new or existing manufacturing settings, enabling rapid changes in mRNA production. Cytiva also offers a range of mRNA purification, encapsulation, and aseptic filling solutions.

To fully realize the potential of genomic medicines, developers must embrace new and integrated methods of improving development and manufacturing. Such efforts will ensure a bright future for mRNA, gene therapy, gene editing, and—most importantly—for the millions (or billions) of patients who stand to benefit from these next-generation technologies.

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