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Mapping the path to gene therapy 2.0

Getting to gene therapy 2.0 will require improvements under way, plus an overhaul of manufacturing strategy

A series of incremental but important improvements on the existing gene therapies are starting to address some of the technology’s key limitations. But some argue it will take a major overhaul of manufacturing practices to produce the step change necessary for version 2.0.

While gene therapy 1.0 was over 20 years in the making, it left a to-do list centered on repeat dosing and manufacturing as the principal hurdles to becoming broadly used cures for multiple diseases.

In some cases, gene therapies can be one-and-done treatments. But often, there’s a need for redosing that can’t be met because the viral vectors that carry the gene elicit immune responses in which antibodies attack and eliminate the therapy before it reaches its target.

For safety reasons, drug developers have shied away from using vectors that integrate into the genome, opting instead for vectors that sit outside of chromosomes, either in the cytoplasm or nucleus. In those cases, the vector gets diluted through cell division.

Multiple doses are especially critical for pediatric patients, who are still growing, meaning that the therapy may be rapidly diluted even if slow-dividing cells are targeted.

“We have to switch from a candle to an electrical light; we need a strong paradigm shift in the field of production.”

Frederic Revah, Genethon

In addition, the inability to redose complicates clinical trial design and patient recruitment,

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