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Cell and Gene Therapy: Agility in the Material Supply Chain

A robust risk assessment and raw material control prove labor-intensive but invaluable for a gene therapy company’s supply chain.

Pharmaceutical supply chains can be hard enough to manage for legacy drugs. But for new cell and gene therapies (CGTs), there are many additional complications.

At the 2019 PDA Cell and Gene Therapy Conference this week, Stefanie Brady, Director of Supply Chain at REGENXBIO, explained that with small sporadic manufacturing campaigns, it’s tough to source raw materials. “It’s not once a month, or even once a quarter… campaigns may only take place a few times throughout the year,” she said.

This makes it difficult for CMOs or in-house manufacturing to plan for this demand. “We don’t have a lot of manufacturing capacity, we have a very intricate process to manufacture and these processes aren’t automated. Plus, we need highly skilled workers,” so it’s not easy to transfer manufacturing, explained Brady.

Lead times present challenges

Raw materials for CGTs have long lead times, with plasmids requiring six months or even years. It’s hard for suppliers to know who needs what and when. Adding to the challenge is that these materials are often single-source and expensive.

REGENXBIO uses CMOs for its manufacturing processes. For the company and others like it, to prepare for a trial, they may reach out to CMOs and negotiate when they can get into their manufacturing suites. If one CMO has availability in four months and another cannot manufacture for 12 months, they may opt for the first to meet timelines. But that means they only have four months to get raw materials together.

Controlling raw materials in-house

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