ASGCT: Why biopharma must face up to the ethical issues around prenatal gene therapies

It’s one of the most exciting ideas in medicine: prenatal cell and gene therapy, where pathological gene mutations and diseases are corrected in the fetus before it ever leaves the womb, protecting against a lifetime of illness or even early death.

Studies in animals are already homing in on candidate conditions and revealing new strategies for overcoming technical barriers. While success in humans has so far been limited to a handful of case studies involving cell therapies, even failures bring the science one step closer to translation.  

But, as University of California, San Francisco (UCSF) anthropologist Julia Brown, Ph.D., explained in a May 8 session at the 2024 American Society for Gene and Cell Therapy meeting in Baltimore, this progress comes with ethical concerns. 

“Ultimately, [prenatal gene and cell therapy] is ethical when there is evidence that scientific capabilities meet unmet medical need, which can be understood as a form…
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