The death of a six-year-old girl after receiving an experimental gene-editing therapy has been described by scientists as a tragedy that highlights the need to improve trial safeguards.
But some in the biotechnology industry fear Chinese policymakers may curtail the regulatory flexibility that scientists have long enjoyed in initiating clinical trials – a key mechanism behind China’s competitive edge in frontier treatments.
“I hope this tragedy does not have a lasting negative impact on China’s investigator-initiated clinical-research system, but there is a real risk of a regulatory backlash,” said Wang Haoran, chief executive of Neoland Biosciences, based in Shandong province.
“China has advanced rapidly in several frontier areas, partly because the mechanism has provided greater flexibility for carefully supervised early clinical exploration. That speed has increased the sense of urgency across the biotechnology industries in the United States and Europe.
“This case appears to reveal serious failures in risk assessment, transparency and institutional oversight. Stronger safeguards are clearly necessary, especially for first-in-human gene-editing treatments involving children.”
The girl, identified only as Mei, died days after receiving a bespoke therapy designed to correct a neurodevelopmental genetic mutation in March last year, according to a joint investigation by Retraction Watch and the journal Science published on July 23.