Gene therapy firms are under pressure to get to market quickly according to an expert from MilliporeSigma, who says process development and tech transfer are significant hurdles.

The ability to treat – or cure – disease at the genetic level in a targeted manner is an attractive proposition for drug companies.

The challenge now is to make these therapies in a cost and time efficient manner according to Ratish Krishnan, associate director for cell and gene therapy bioprocessing at Milliporesigma.

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“Gene therapies present a significant opportunity to help people live better lives and are becoming mainstream. There are many challenges, however, in the journey to commercialization that could derail a programme.

“This is particularly true around tech transfer and manufacturing,” he said, adding that “Avoiding costly and time consuming mistakes is paramount as companies are competing for the same indication.”

Krishnan told BPI West attendees that many in the industry have responded by trying to accelerate development

“Time lines for gene therapy, commercialization are generally much faster than monoclonal antibodies, sometimes even up to 50% quicker, he said, explaining that to save time many firms carry out certain development phases in parallel.

“The agenda we see is to develop a process that works suitably, qualify it, validate it and then make changes to it later on. The catalyst is the fact that we have patients anxiously awaiting these therapies, and the demand is far greater than the available supply at the moment.”

And there are other options for gene therapy firms hoping to reduce time-to-market according to Krishnan.

“So there are other levers as well besides the products … such as raw materials and the manufacturing and the testing strategies you use, which can aid speed to commercialization.”

He added that, “We also got to think in terms of equipment, unit operations, things like software used in equipment and if possible, a long term vision of whether the technologies you use today will still be around in five year.”

Strategies for success

Ultimately the keys to cutting gene therapy commercialization timelines are preparation and collaboration according to Krishnan.

“A tight relationship between manufacturing and PD groups cannot be stressed enough, especially for these novel modalities when nothing is permanent to the process, but change. Balancing expectations from upper management taking time for process development and understanding the yield and purity trade off also becomes critical.

“And finally, you know, as we are building a parachute while diving from the sky, there is a need to focus on communication and escalation strategies, especially when something goes wrong. So the need for synergy across departments such as PD tech services, manufacturing, you know, operations, QA, QC, rec, CMC, and all those become paramount in this situation.”