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Rocket Pharmaceuticals Pauses Gene Therapy Trial After Death

5/27/2025

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Summary

Story summary

A participant in Rocket Pharmaceuticals' Phase 2 trial for RP-A501 gene therapy, aimed at treating Danon disease, tragically passed away due to an acute infection associated with capillary leak syndrome. In light of this incident, the trial is currently paused as the company investigates, particularly examining a new immune suppression agent. Rocket is dedicated to advancing treatments for this rare condition.

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Story summary

A participant in Rocket Pharmaceuticals' Phase 2 trial for RP-A501 gene therapy, aimed at treating Danon disease, tragically passed away due to an acute infection associated with capillary leak syndrome. In light of this incident, the trial is currently paused as the company investigates, particularly examining a new immune suppression agent. Rocket is dedicated to advancing treatments for this rare condition.