Industry Insights

1. The 4 Trends Affecting Blockbuster Drug Status 1/25/2022

   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.

2. Proposed Solutions For Cell & Gene Therapy Validation Challenges 1/21/2022

   Cell and gene therapy validation requirements are still in development. As more experience and knowledge are gained on manufacturing processes, some challenges will be resolved and others will be identified. This article delves into challenges and proposed solutions for manufacturing process validation, designing PPQs for autologous cell therapy, and analytical method validation.

3. Aiming To Solve The 3 Key Challenges Of CAR T-Cell Manufacturing 1/18/2022

   These authors share their best practices for solving the three key challenges of CAR T-cell manufacturing (supply constraints, scalability issues, and storage and logistics challenges) using their experiences with Epstein Barr Virus (EBV) T cells.

4. Capital Raising For Cell & Gene Therapies: How To Craft Your Elevator Pitch 1/17/2022

   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.

5. Genome Sequencing Research For Rare Diseases & The Future Of The Field 1/11/2022

   Tomi Pastinen, M.D., Ph.D., is the director of the Genomic Medicine Center at Children’s Mercy Research Institute. In 2019, he and a team of CMRI researchers launched Genomic Answers for Kids, a first-of-its-kind pediatric data repository with the goal of collecting genomic data and health information from children and their families over the next seven years to create a database of 100,000 genomes. 

6. Regulatory Perspectives For CGT Development: Continued Need For Standards And Perseverance 1/7/2022

   The need for standards not only lies in the identification and development of standards, but also in implementation.

7. Are You Leveraging KOLs For Biopharma Market Access As Much As You Could Be? 1/7/2022

   Pharmaceutical companies spend a lot of time and effort engaging and collaborating with key opinion leaders (KOLs), an exercise typically owned by medical affairs. But few take advantage of network mapping tools and relationship-building principles from a market access lens to help inform and shape coverage strategies and identify access champions for their products.

8. Harnessing The Human Virome For The Next Generation Of Gene Therapy Vectors 1/5/2022

   Advances in metagenomic sequencing have revealed insights into the human virome with boundless value for gene therapy researchers. This includes exploring the diversity of a family of small commensal viruses called anelloviruses that have never been associated with disease. Anelloviruses are a promising new tool in our effort to overcome today’s limitations in genetic medicine.

9. RNA Takes The Spotlight In The Era Of Precision Medicine 1/4/2022

   RNA are the protein builders, and now that we have the technology to learn more about them, we have better tools for advancing precision medicine.

10. Collaborating With The FDA For Rare Disease Clinical Trial Success: Our Experiences 1/4/2022

    If you are familiar with rare diseases, you know there are unique challenges associated with conducting clinical trials to evaluate potential new treatments for small patient populations. When we set out to design our Phase 2b study for a treatment targeting congenital ichthyosis, we knew we would need a plan to tackle these challenges. Here's how we worked with the FDA throughout the process.