Another COVID-19 casualty: ‘Major delays’ for gene therapy trials for rare diseases

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Credit: Batten Hope

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases.

That’s the consensus of industry experts who spoke in a May 26 webinar hosted by the events organizer Terrapinn, producer of the annual World Orphan Drug Congress.

COVID-19 raises specific operational challenges to rare disease clinical trials that can force delays, [uniQure’s Sander] van Deventer said, such as slow enrollment, limited access to hospitals, and missing data.


Particularly vulnerable are trials targeting patients with a higher-risk profile; these will require specific strategies to be assessed on a case-by-case basis.

“This has certainly been true for gene therapy clinical trials,” van Deventer said, without giving examples. “Transplant trials have been almost halted because of the pandemic. And I already know of some planned clinical trials that have run into major delays.”

Related article:  Viewpoint: Human challenge trials – volunteers intentionally infected with COVID-19 – are ‘uninformative, unnecessary and unethical’

In fact, [PTC Therapeutics’ Thomas] Bols said, the COVID-19 crisis is actually an opportunity for the biopharma industry, and especially for companies pursuing cell and gene therapies.


“If there’s one thing that makes this crisis clear, it’s the importance of innovation. A lot of companies, small and big, are frantically working to come up with a vaccine or a therapy to counter COVID-19. This really underlines the need for innovation, and the role our industry is playing to come up with a solution.”

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