Terry Horgan, the 27-year-old patient who died eight days after receiving a CRISPR therapy custom-built for him, likely suffered a fatal innate immune response to the virus used to deliver the treatment, investigators concluded.
The findings, posted late Thursday to the preprint server Medrxiv, confirmed that CRISPR, the Nobel-Prize winning genome editing tool now being used to develop treatments for a wide range of diseases, played no role in Terry’s death.
The investigation offered the first detailed analysis of a case that had spurred excitement and trepidation in the rare disease world before ending in tragedy. CRISPR and other new genetic technologies had raised hopes that researchers might be able to design custom therapies for patients’ particular mutations, even if those mutations were too rare to convince industry to invest in development.
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