7:55 PM
Dec 13, 2018
 |  BC Innovations  |  Product R&D

Gene therapy’s make or buy choice

How gene therapy companies are navigating manufacturing decisions

Editor's Note: This article was updated on Dec 14, 2018 at 6:47 AM PST

Whether or not to bring manufacturing in-house for gene therapy companies is becoming a critical choice, as contract manufacturers struggle to keep up with demand. The trade-off for the upfront investment risk is control over development timelines and the rapidly evolving science of process development.

A spate of gene therapies could hit the market in the next few years, following Spark Therapeutics Inc.’s Luxturna voretigene neparvovec, which last year became the first of the modality to gain FDA approval. At least one additional candidate is in registration -- Novartis AG’s Zolgensma onasemnogene abeparvovec -- and 14 others are in Phase III testing, according to BioCentury’s BCIQ database.

The surge is straining contract manufacturers, leading to long queues that threaten development timelines. Academic production centers are seeing similar backups (see “Academia’s Manufacturing Problem”).

At the same time, process development science for gene therapies is advancing swiftly, making it appealing for some drug developers to perform the work internally and secure manufacturing-related IP.

Because most gene therapy companies are targeting small patient populations, often on accelerated regulatory timelines, they need small quantities of product quickly, which the CMOs are not set up to accommodate.

While antibody or small molecule start-ups typically view the upfront costs of in-house production as prohibitive or too risky, for gene therapy developers the calculus may be different. But the field is divided on whether to make or buy.

“The CMOs are all capacity-constrained and the timelines to work with them were untenably long for a company like us.”

Natalie Holles, Audentes Therapeutics

At least three gene therapy companies -- Spark, Audentes Therapeutics Inc. and AveXis Inc. -- opted to build their own manufacturing processes from the start. All had pipelines of gene therapies, not a single asset, so they stood to get more mileage out of the investment.

Audentes President and COO Natalie Holles told BioCentury her company chose to manufacture in-house because available CMOs were limited and lacked expertise.

“We spent a lot of time looking at the external options and we just weren’t impressed,” said Holles. “The CMOs are all capacity-constrained and the timelines to work with them were untenably long for a company like us.”

RegenxBio Inc. and Adverum Biotechnologies Inc. opted to use CMOs, despite having pipelines of gene therapies.

RegenxBio SVP of Product Development and CTO Curran Simpson told BioCentury that using CMOs made the most sense on a cost basis, as setting up in-house manufacturing is “roughly a $30-$50 million investment.”

Nevertheless, RegenxBio and Adverum are both retaining a high degree of control over process...

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