Gene Therapy – An Industry Coming of Age

This article is the first in a series that was published in the eBook
Insights on Successful Gene Therapy Manufacturing and Commercialization.

You can download all the articles in the series, by downloading the eBook.

In 2018, Cell Culture Dish worked with a talented group of authors, all experts in the field of gene therapy, to create an Ebook that was meant to serve as a guide for identifying key considerations in gene therapy manufacturing and commercialization. The publication was very well received and earlier this year, we decided that it was time to publish a new guide based on the advancements that we had seen over the past two years. The process of writing this updated publication provided wonderful insight as we compared the state of the industry in 2018 to today. What we found is that significant strides have been made across manufacturing and commercialization, however there is still much to be done to reach gene therapy’s full potential.

In our previous Ebook, we referred to gene therapy as the future of medicine and while I still believe that term is accurate, as we have plenty more to do, the truth is that the future is here. We see this in the six gene therapy products approved in the US and in the hundreds of gene therapy products in the clinical pipeline. The enthusiasm for what this technology can do hasn’t waned; in fact we have compelling data on how these approved therapies are helping patients.

In a recent presentation, Alliance for Regenerative Medicine Gene Sector Overview July 2020, the patient impact of recently approved products was shared. The results are quite impressive. Zolgensma®, approved by the FDA in May 2019, is a gene therapy treatment for pediatric patients less than two years of age with spinal muscular atrophy (SMA). The Alliance for Regenerative Medicine (ARM) shared data that 93% of SMA Type 1 patients treated were alive without permanent ventilation at 24 months post-treatment. Luxturna®, a gene therapy treatment for certain inherited retinal diseases had 93% of patients who showed an improvement of at least one light level from baseline. In a recent press release, Kite shared data on its CAR T therapy for Mantle Cell Lymphoma, Tecartus™. In a single-arm, open-label study, 87% of patients responded to a single infusion of Tecartus, including 62% of patients achieving a complete response. These are just a few examples of the remarkable outcomes that these therapies can provide to patients.

However, these results did not come easy. The tremendous potential of gene therapy is the result of decades of research, implementation of select best practices from the field of biologics development, and impressive clinical results that collectively have led to significant industry investment and a pathway for regulatory approval. Gene therapies represent a new medical paradigm and that impacts regulatory approval, reimbursement methods, and manufacturing strategies. While there is much to be learned from following the example of almost 40 years of recombinant antibody manufacturing and commercialization, there are many differences that need to be addressed.

What makes gene therapies so different?

  • They offer the possibility, in many instances, of an actual cure instead of chronic treatment.
  • Frequently the treatment is a one-time-only treatment.
  • Gene therapies can be expensive to produce and administer which can be a challenge in terms of reimbursement.
  • Gene-modified cell therapies are highly customized, patient-specific therapies, which present unique challenges from a logistics and distribution perspective.

These differences represent what makes gene therapies so compelling, but also represent the challenges that the industry faces. In this new 2020 Ebook, Insights on Successful Gene Therapy Manufacturing and Commercialization, our aim was to share insights and lessons learned on how to manufacture and commercialize gene therapy products. We were lucky to bring together a talented group of authors from several different companies, who all saw the importance of coming together to create a piece that is educational and identifies the key challenges, new developments, recent successes, and areas for improvement. Our goal was to address some key areas important to the successful commercialization of gene therapies. This included discussion about gene therapy manufacturing, the latest in manufacturing methods, analytical analysis, key regulatory considerations and reimbursement strategies.

The key areas we identified include:

  • Planning for clinical and commercial manufacturing, including upstream and downstream bioprocess development, scale up and current best practices.
  • Advancements in gene therapy analytics and incorporating in-process analytics.
  • Ensuring scalability and efficient timelines in manufacturing while maintaining reasonable cost.
  • Reimbursement strategies for effective commercialization planning.
  • Regulatory considerations and outlook for the future.
  • Current state of the industry and areas for improvement.

The industry faces some formidable challenges ahead, but there are enabling technologies being developed, excellent resources and experts available, and importantly, a regulatory and manufacturing pathway that has been paved for other companies to follow. In this Ebook, with the help of experts sharing their perspectives on various topics, we examine the current state of the industry and how we can continue to advance these life-changing medicines for the benefit of patients. We sincerely hope this serves as a valuable resource for those wishing to learn more about gene therapy, its manufacture, and commercialization.

Brandy Sargent
Cell Culture Dish & Downstream Column

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