Rocket Pharmaceuticals Inc.
Clinical trials sponsored by Rocket Pharmaceuticals Inc., explained in plain language.
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Scientists track patients for 15 years after pioneering gene therapy for rare immune disease
Disease control OngoingThis study follows 9 patients for up to 15 years after they received an experimental gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I), a rare and serious immune system disorder. Researchers want to understand the long-term safety of the treatment and whether it continues …
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Apr 03, 2026 14:41 UTC
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Doctors test One-Time gene fix for rare blood disorder in kids
Disease control OngoingThis study is testing a one-time gene therapy for children with Fanconi Anemia type A, a rare genetic disorder that causes bone marrow failure. Doctors take the patient's own blood stem cells, correct the faulty gene in a lab, and then return the corrected cells to the patient. T…
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Apr 02, 2026 14:57 UTC