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30-day roundup
The 10 most notable cure or vaccine studies from the last month.
A trial tests a one-time CRISPR gene-edited stem cell therapy for severe sickle cell disease, aiming to stop painful vaso-occlusive crises.
Read lead report →A first-in-human trial tests SGX-001, a gene therapy using a patient's own stem cells, to restore immune function in chronic granulomatous disease.
Read report →A trial tests a one-time gene therapy infusion for Sanfilippo syndrome (MPS IIIB), aiming to restore a missing enzyme and slow the disease's effects.
Read report →A trial tests different conditioning regimens before stem cell transplants in children with acute lymphoblastic leukemia to improve survival.
Read report →A trial tests CTX112, an engineered T-cell therapy, for severe neurologic autoimmune diseases like progressive MS and stiff-person syndrome.
Read report →An early trial tests a gene therapy for homozygous familial hypercholesterolemia, aiming to lower LDL cholesterol by restoring LDL receptor function.
Read report →A gene therapy trial tests whether modified stem cells can treat metachromatic leukodystrophy, a rare genetic disease.
Read report →A phase 1 trial tests combining focused ultrasound with T-cell therapy for diffuse midline glioma, a rare brain tumor.
Read report →A Phase 1 trial tests whether a single infusion of allogeneic CAR-T cells can reset the immune system in people with severe autoimmune diseases.
Read report →A trial tests gene-modified stem cells to correct the faulty gene behind beta thalassemia, aiming for a lasting fix without lifelong transfusions.
Read report →Trials whose completion date fell in this window and whose status is actually Completed. Results usually follow later.