FAMILIAL HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS TYPE 3 (FHL 3)

Clinical trials for FAMILIAL HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS TYPE 3 (FHL 3) explained in plain language.

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Not yet recruiting 1

Gene therapy could replace bone marrow transplants for rare immune disease

Disease control Not yet recruiting

This trial tests a gene therapy for FHL3, a rare immune disorder caused by a gene mutation. Instead of a donor stem cell transplant, patients receive their own stem cells that have been corrected with a healthy gene. The goal is to restore immune function while avoiding transplan…

Matched conditions: FAMILIAL HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS TYPE 3 (FHL 3)

Phase: PHASE1, PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control

Last updated May 29, 2026 14:19 UTC

FAMILIAL HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS TYPE 3 (FHL 3)

Get alerted when new FAMILIAL HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS TYPE 3 (FHL 3) trials appear

Gene therapy could replace bone marrow transplants for rare immune disease