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The gene therapy market is experiencing rapid growth. From an estimated worth of $7.74 billion in 2023, the market is predicted to reach $38.7 billion by 2032 at a compound annual growth rate of 19.6%1. Currently, over 2,000 gene therapies are under development for treating a range of rare and chronic disease indications2. 

Despite their promise as innovative treatments, the cost of gene therapies remains a significant barrier to patients. With a one-time cost of $3.5 million, the AAV therapy Hemgenix is currently the most expensive drug on the market3. As a result, developers must find a way to introduce cost efficiency while maintaining high product quality. 

AAV developers are turning to platform approaches offered by contract development and manufacturing organisations (CDMOs) to help overcome complexities that could extend production timelines and increase costs. When using an AAV platform, the process can be seamlessly adapted and optimised for a wide range of AAV therapies. 

To deliver flexibility, AAV platforms must be able to adapt to the needs of the customer and the project. Developed starting from a small scale, the process involves: 

  • Screening different cell lines
  • Testing different reagents and methods
  • Determining the process parameters
  • Optimising conditions using design of experiment (DoE) approaches.


Aiming to achieve high product yield while minimising cost, an AAV platform development approach should consider scalability. The final optimised platform must be easily scalable, allowing a seamless transition from preclinical to clinical stages through to commercialisation. In addition to reducing production costs, platform approaches can also help to minimise risk. 

Read the recent Q&A with Xiaojun Liu, Director of AAV Process Development at ReciBioPharm, to discover more about how AAV platform approaches are helping to enhance gene therapy manufacturing and drive down costs. 

 

Read the full article

 

References:

  1. Gene Therapy Market Size, accessed January 25, 2024, from: https://www.precedenceresearch.com/gene-therapy-market#:~:text=The%20global%20gene%20therapy%20market,19.60%25%20from%202023%20to%202032.&text=Key%20Takeaway%3A,of%20around%2056%25%20in%202022

  2. Gene Therapies: A Wave Is Coming, accessed January 25, 2024, from: https://www.lse.ac.uk/business/consulting/news/gene-therapies#:~:text=The%20pace%20of%20research%20is,therapies%20currently%20undergoing%20clinical%20trials

  3. The Top 5 Most Expensive FDA-Approved Gene Therapies, accessed January 25, 2024, from: https://lifesciencesintelligence.com/features/the-top-5-most-expensive-fda-approved-gene-therapies#:~:text=Zolgensma%2C%20and%20Luxturna.-,Hemgenix,as%20congenital%20factor%20IX%20deficiency