Manufacturing Gene Therapy

Getty / Edited by BioPharma Dive

Note from the editor

When Bayer bought gene therapy developer AskBio for $2 billion late last year, the smaller company's in-house manufacturing capabilities were a major selling point. So much so the German drugmaker hinted the acquisition may be the foundation for future partnerships around AskBio's production base. 

Gene therapies are particularly complex and rely on specially engineered viruses to get into human cells. For diseases that affect large organs or muscles, trillions of virus copies may be needed. It's a daunting task and can't always be easily scaled up as clinical development progresses into larger trials. 

As a result, manufacturing has become a bottleneck in the field, a hurdle faced by larger pharmaceutical companies as well as small biotech startups. Companies like AskBio that have built up their manufacturing are in particular demand.

Those that haven't, or are progressing faster than they can expand, are forced to committ immense sums to constructing new factories. Earlier this year, for example, Biogen announced plans to spend $200 million on a plant in North Carolina. The planned investment follows similar decisions from Pfizer, Novartis, Bluebird bio and Audentes Therapeutics

In parallel, the contract firms that support biotech and pharmaceutical companies are ramping up, too. In recent years, Thermo Fisher spent $1.7 billion to buy gene therapy contract specialist Brammer Bio, while competitor Catalent bought Paragon Bioservices for $1.2 billion. Both companies have since announced further gene therapy-related investments. 

Even so, the industry's manufacturing footprint may not be growing fast enough to match an ever-widening pipeline of experimental therapies in development. According to a recent report from industry group Alliance for Regenerative Medicine, more than 400 gene therapy studies were in progress last year, as well as another 780 in related cell therapy fields. 

Many of those therapies won't succeed, or won't be advanced. But even a fraction could still tax companies' manufacturing capacities, particularly as diseases affecting larger numbers of patients are targeted. Meeting that challenge will be a key test for the gene therapy sector. 

Ned Pagliarulo Lead Editor

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Behind the scenes of gene therapy manufacturing

Gene therapies rely on specially engineered viruses to enter human cells; For diseases affecting large organs or muscles, trillions of virus copies may be needed. As a result, manufacturing has now become a bottleneck in the field.

included in this trendline
  • Thermo Fisher broadens gene therapy bet with new plant
  • Joining CDMOs, biotechs like Biogen build out manufacturing capacity
  • With rising demand, Catalent expands in gene therapy
Our Trendlines go deep on the biggest trends. These special reports, produced by our team of award-winning journalists, help business leaders understand how their industries are changing.
Davide Savenije Editor-in-Chief at Industry Dive.