Rocket Pharmaceuticals Inc.
Clinical trials sponsored by Rocket Pharmaceuticals Inc., explained in plain language.
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Doctors test One-Time gene fix for rare blood disorder in kids
Disease control OngoingThis trial is testing a one-time gene therapy for children with a rare, serious genetic disorder called Fanconi anemia type A. Doctors take a patient's own blood stem cells, fix the faulty gene in a lab, and then return the corrected cells to the patient. The goal is to prevent t…
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC
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Doctors track patients for 15 years after pioneering gene therapy for devastating immune disease
Disease control OngoingThis study is tracking 9 patients for up to 15 years after they received an experimental gene therapy for a rare and severe immune disorder called LAD-I. The therapy involved modifying a patient's own blood stem cells to correct the genetic defect. Researchers want to see if the …
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Mar 23, 2026 15:15 UTC