Top 10 Challenges: Producing commercial cell and gene products

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The international regenerative medicines market – which includes the likes of cell therapies, gene/gene-modified cell therapy and tissue engineering – is set to climb to around $67 billion by 2020.  Alongside, discovering therapies that will treat cancer and orphan diseases, many in the industry are hungry to land grab market share and produce the next blockbuster product.

However, there are many challenges that stand in the way of producing commercialised cell and gene products - whether they are allogenic or autologous. Autologous therapies will require separate manufacturing batches for each patient therapy. Those therapies can be hard to transfect. Whereas, allogenic therapies will take the altered cells, expand them as to create a large volume of them to be used by patient demographics. 

The rise of personalised medicines has led to the focus on small scale manufacture with a key challenge being that lots of the same element will run through a facility at once. Randomised trials with complex gene therapies- which target a small number of patients often in regards to orphan diseases can encounter various manufacturing hurdles. Also, evidence is needed to prove efficacy against historical controls with Phase II trials. This of course requires several years of data which is limited, incurring implications on cost and time to market.

Clinical results stand as a key challenge for product developers to meet. Dieter Hauwaerts, VP Operations, Celyad explained that in order to show really outstanding and reliable clinical results there needs to be truly outstanding manufacturing processes and technologies that are consistent and have a very high degree of quality built into them. Besides generating those breakthrough clinical results, pharma and biotech companies need to consider how they are going to manufacture, release and ship those products at a significantly larger scale. Dieter Hauwaerts believes that there always will be a place for cell and gene therapy products in smaller indications, but the true maturation of the field only will come when the industry can show results from a clinical and a commercial point of view in larger indications. Typically, that is where the CAR-T field is moving towards.


Ahead of the Cell and Gene Therapy Manufacturing conference Pharma IQ examines the various challenges that complicate the route to producing off the shelf cell and gene products. 

Challenges covered: 

  • Scientific Awareness Of Production
  • Limited Appropriate Manufacturing Platforms
  • Quality By Design
  • Quality Assurance
  • Cost Efficiency
  • Scalability ( Up And Out )
  • Quality Of Starting Materials And Influence On The Efficiency
  • Regulations
  • Closed System Processing
  • Supply Chain Collaboration 

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