Gene Therapy

Novartis’ star gene therapy faces potential safety issue following a recent infant death in study

Novartis got some more upbeat press earlier in the week when researchers on the Zolgensma gene therapy program outlined some updated biomarker data on their SMA drug and noted that 21 of 22 patients were alive and event free as of last fall. But it turns out there was a post script to the study that raises some safety concerns for the pharma giant’s therapy — recently acquired in an $8.7 billion acquisition of AveXis.


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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
Associate Director CMC
Elektroki Boston, MA
Director Process Development
Elektroki Boston, MA
Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT

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