INSIGHTS ON CELL & GENE CLINICAL TRIALS

• The 4 Trends Affecting Blockbuster Drug Status

  Although the billion-dollar mark will remain one of the principal benchmarks to define the success of pharmaceutical products, new indicators are emerging. This article looks at the drugs identified in Clarivate's Drugs to Watch series from 2015 and 2016, analyzing the trends and challenges that occurred over the past five years. The analysis identified these 4 key trends.

• Capital Raising For Cell & Gene Therapies: How To Craft Your Elevator Pitch

  We often hear of the need for an “elevator pitch” in the context of raising capital – a brief 30-second persuasive speech to spark interest and engage with your audience. This article uses cytokine release syndrome (a disorder characterized by fever, tachypnea, and other symptoms) as an example to explain how to perfectly craft your elevator pitch within clinical drug development.

• How mRNA Technology Is Shaping The Future Of Medicine

  Discover how mRNA technologies open the doors to a whole range of new gene therapies and the role of lipid nanoparticles (LNPs) in current and future developments.

• What Are Extracellular Vesicles, Exosomes, & Microvesicles & Why Are They En Route To The Clinic?

  Researchers have recognized that therapeutic cargoes can exist naturally in the extracellular vesicles (EVs) or they can be introduced by a variety of other methods resulting in the therapeutic capabilities of EVs to be expanded to include several other diseases and modes of action.

• Quality By Design The Highest-Standard Approach To Process Validation

  The challenge was to develop and validate manufacturing processes for two novel molecules, one in phase 1 development and the other in phase 3.

• Robust, Scalable Manufacturing: N-Linked Glycan Analysis For Complex Glycoproteins

  An established cell line and a bench-scale manufacturing process for a complex glycoprotein produced high quality material but had a low yield and was not scalable.

• How To Overcome The 3 Major Challenges In Conducting Rare Disease Clinical Trials

  Designing and conducting clinical trials for rare diseases often requires drug developers to plan for and address many challenges that are often not associated with or are less severe in larger trials for more common diseases and conditions. If they are not adequately addressed, these challenges can result in trial delays or discontinuations. This article shares Cyclo Therapeutics' 3 lessons learned.

• The Benefits Of Streamlining And Standardizing Plasmid DNA For Cell And Gene Therapies

  The need for greater volumes of high-quality raw materials like plasmid DNA in the cell and gene therapy space has made pDNA standardization integral to streamlining the development process.

• Your Workbook To Create A Rare Disease Clinical Trial Strategy

  In the rare disease space, planning and creating a robust patient recruitment plan is essential. This will lay the foundation for the budget, tactics, and expectations that are needed. In this article, the author shares her three-step process that she implemented in her career and it can set you on a path toward success for clinical trial recruitment in rare disease.

• Rare Disease Clinical Trials: 3 Areas We Could Improve On

  Rare disease clinical trials pose unique and complex challenges. Small and geographically dispersed locations of treatment centers, disease heterogeneity, and incomplete disease natural history can complicate the process. However, there are actionable and tangible steps we can take to improve the process and enable development of innovative and meaningful therapies.