Plasmid DNA (pDNA) is an extrachromosomal, covalently closed circular nucleic acid molecule. This molecule is used widely by bacteria to carry genes that confer different properties and replicates independently of chromosomal replication. Plasmid DNA has already found extensive use in molecular microbiology, where it can be used as a tool to transport genetic information. Additionally, pDNA has the further advantage of being easy to manipulate, enabling the production of different molecules in a range of host organisms, from prokaryotes to eukaryotes.

The discovery of plasmids in the 1950s was the starting point for subsequent advances in molecular biology, ultimately leading to the cell and gene therapy products that are now reaching the market. These have allowed patients to be treated for, and sometimes cured of, a wide range of diseases, where previously, no therapeutic options were available. Without plasmids, many of the significant advances that have been made in genetic engineering in recent times would not have been possible.

Over the years, many different uses have emerged for plasmids. A good example is the production of the first recombinant protein treatment, insulin, which had its initial FDA approval in the early 1980s. This led to clinical trials of the first viral vectors in the 1990s, and in the subsequent decade, the first clinical trials on messenger RNA (mRNA) products.

Since then, plasmids have underpinned the development of numerous other therapeutic modalities. They have applications in both lentivirus (LV) and adeno-associated virus viral (AAV) vectors, as well as mRNA and plasmid-containing nanoparticles, in addition to immunotherapy and vaccine products, shaping the research of many modern, ground-breaking therapies.

BioPharm International’s September 2023 Emerging Therapies eBook

, director, Process and Analytical Development—pDNA, Catalent Cell, Gene & Protein Therapies, and 

, vice president, Quality, Catalent Cell, Gene & Protein Therapies.

When referring to this article, please cite it as Gomez Santos, N.; Oliver, B.J. Plasmid DNA—The Versatile Building Block. 

BioPharm International’s Emerging Therapies eBook 

Articles

Gene and Cell Therapies

Plasmid DNA (pDNA) is a critical raw material for the manufacture of cell and gene therapies. As this market continues to grow, so too does the demand for high-quality pDNA.

Plasmid DNA—The Versatile Building Block

Cell and gene therapies (CGTs) can potentially treat, and sometimes even cure, a wide range of genetic and acquired diseases ranging from immunodeficiencies to cancer. For this reason, the field has been growing at an astronomical rate. Having impeccable data about the makeup of a new therapeutic is vital to successful clinical trials that take a new drug candidate one step closer to FDA approval. But the myriad designs of CGTs in development today make that uniquely challenging. In this article, the author will explore some of the specific hurdles associated with lentiviral CGTs.

Marwan Alsarraj is biopharma segment manager, Digital Biology Group, Bio-Rad.

When referring to this article, please cite it as Alsarraj, M. Navigating the Intricacies of Lentiviral CGTs. 

Scientists are leveraging ddPCR technology to get an accurate read on viral titer and vector copy number.

Navigating the Intricacies of Lentiviral CGTs

Between COVID-19 vaccines launching messenger RNA (mRNA) therapies into the limelight and the rising potential for personalized cancer vaccines, biologics have captured the attention of scientists and investors alike. And for investors in particular, there is additional incentive to invest in biologics treatments due to the new exclusivity periods introduced in the Inflation Reduction Act, which give biologic treatments four more years of manufacturer exclusivity relative to small-molecule alternatives (4).

In tandem with this growing interest, market research firms are estimating the biologics market will grow to anywhere from $506 (5) to $719 billion (6) by the end of the decade. As a major market force, it is important to take stock of what biologics are being approved, as these decisions can provide insight into what the immediate future of the market might look like.

’s September 2023 Emerging Therapies eBook.

Grant Playter was previously associate editor with BioPharm International, and Daria G. Husni is assistant editor with BioPharm International.

When referring to this article, please cite it as Playter, G; Husni, D. G. A Growing Biologics Market. 

BioBusiness

As more novel therapy approvals trend towards biologics, it is important to take stock of what treatments are shaping the future of the market.

A Growing Biologics Market

Ongoing innovation in the biopharma pipeline continues to bring hope to patients and prescribers everywhere. Biologic molecules, however, are inherently fragile and thus are susceptible to deterioration in the face of excursions from the refrigerated, frozen, and ultra-frozen temperatures that are required throughout every step in the storage and transit process.

The universe of high-value biologics—which includes injectable specialty medications, cell and gene therapies (CGT), and other regenerative medicines and more—has come a long way over the past decade. Further complicating the picture, a growing number of innovative CGTs and other regenerative therapies call for the highly specialized collection and transport of not just extracted blood but extracted bone marrow, spinal fluid, and other tissue types, which are then used to produce engineered therapies that are used to repair, replace, or regenerate organs, cells, and metabolic processes in the body.

The cold chain infrastructure that is needed to safeguard these products as they move from manufacturer to patients for infusion in a clinical setting or self-injection at home has also continued to evolve in terms of improved shipping container designs and insulation materials and state-of-the-art tracking and monitoring devices. At the same time, stakeholders are growing more comfortable using advanced data analytics capabilities (including artificial intelligence [AI] and machine learning [ML] techniques) and are pursuing strategic partnerships and geographically advantageous acquisitions to safeguard these critical time- and temperature-sensitive healthcare interventions.

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 This article was previously published online July 12, 2023. 

Suzanne Shelley is a contributing editor to 


Emerging Therapies 2023 eBook
September 2023
Pages: 24-30

When referring to this article, please cite it as Shelley, S. Protecting High-Value Biologics at Every Turn. 

Supply Chain

Cold chain stakeholders continue to innovate with new technology development, the use of advanced modeling and analytics capabilities, and the pursuit of strategic partnerships.

Protecting Biologics at Every Turn

Cancer messenger RNA (mRNA) vaccines have made headlines multiple times in 2023. In February, an mRNA vaccine for cancer received breakthrough therapy designation after demonstrating efficacy in a Phase II trial (

). In May, the National Institutes of Health announced that in a small clinical trial, a personalized mRNA vaccine prevented pancreatic cancer from recurring for about half of trial participants (

). These developments were catalyzed in part by the success of the mRNA vaccine for COVID-19.

Unlike traditional vaccines, the manufacturing process for mRNA vaccines is cell-free and can be performed with synthetic enzymes. This allows for unprecedented speed and scale of mRNA vaccine development and manufacturing, which proved to be pivotal during the COVID-19 pandemic. In turn, the deployment of mRNA COVID-19 vaccines helped forge the robust supply chain for mRNA vaccine manufacturing that had not previously existed. Despite this success, clinical and logistical roadblocks to mRNA cancer vaccine development remain.

This article reviews the therapeutic prospects of mRNA vaccines and considerations for clinical development that could help address present issues with patient recruitment, regulatory compliance, manufacturing, and funding.

BioPharm International’s Emerging Therapies 2023

Andreas Dreps, PhD, is SVP, Drug Development Services at ICON; Martin Lachs, PhD, is VP, Project Management Oncology at ICON.


Emerging Therapies 2023 eBook
September 2023
Pages: 18-23

When referring to this article, please cite it as Dreps, A. and Lachs, M. Logistical Considerations in mRNA Vaccine Development. 

 Emerging Therapies eBook. September 2023.

Despite its success, clinical and logistical roadblocks to mRNA cancer vaccine development remain.

Emerging Therapies, September 2023 eBook

Plasmid DNA—The Versatile Building Block

Navigating the Intricacies of Lentiviral CGTs

A Growing Biologics Market

Protecting Biologics at Every Turn

Logistical Considerations in mRNA Vaccine Development