Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of he…

Sickle Cell DiseaseThalassemiaHemoglobinopathy

~Estimated

~$10 – $5,625

National Heart, Lung, and Blood Institute (NHLBI)NCT06313398

Phase 2

Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Non-Malignant Hematological Disorders in Children

This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is s…

Hemoglobinopathy (Disorder)Severe Aplastic AnemiaBone Marrow Failure Syndrome

~Estimated

~$10 – $6,250

Johns Hopkins All Children's HospitalNCT04356469

A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells

This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 w…

HemoglobinopathySickle Cell Disease

~Estimated

~$10 – $1,050

Beam Therapeutics Inc.NCT07373639