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Rocket Pharmaceuticals Gains FDA Approval for First Gene Therapy in Children

3/28/2026

Summary

Story summary

Rocket Pharmaceuticals, Inc. received U.S. Food and Drug Administration (FDA) accelerated approval for KRESLADI, the first severe LAD-I gene therapy in children.
The therapy targets ITGB2 mutation patients lacking a matched donor for transplantation.
Shares rose nearly 9% in premarket trading after approval.
The FDA based its decision on data showing improved immune function markers.
Rocket plans to monetize a Rare Pediatric Disease Priority Review Voucher to fund other therapies.

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Story summary

Rocket Pharmaceuticals, Inc. received U.S. Food and Drug Administration (FDA) accelerated approval for KRESLADI, the first severe LAD-I gene therapy in children.
The therapy targets ITGB2 mutation patients lacking a matched donor for transplantation.
Shares rose nearly 9% in premarket trading after approval.
The FDA based its decision on data showing improved immune function markers.
Rocket plans to monetize a Rare Pediatric Disease Priority Review Voucher to fund other therapies.