30-day roundup
The 10 most notable cure or vaccine studies from the last month.
A phase 1 trial tests RP-A501, a gene therapy for Danon Disease, in males. It aims to assess safety and potential to correct heart muscle damage.
Read lead report →A first-in-human trial tests CTX460, a gene-editing treatment, in adults with emphysema caused by severe alpha-1 antitrypsin deficiency.
Read report →A phase I/II trial tests donor-derived CARCIK CD123/33 cells in adults and children with relapsed or refractory AML and BPDCN before a stem cell transplant.
Read report →A trial tests GNT0004, a one-time gene therapy that delivers a working dystrophin gene to muscles in boys with Duchenne muscular dystrophy.
Read report →Researchers test whether allogeneic hematopoietic cell transplantation can reset the immune system and achieve lasting remission in children with refractory sys
Read report →A Phase 2 trial tests whether combining checkpoint inhibitors with interferon and antivirals can help people with chronic hepatitis B clear the virus.
Read report →Researchers test whether peginterferon alfa-2b plus tenofovir alafenamide can clear hepatitis B surface antigen in adults with HBeAg-negative chronic hepatitis
Read report →A single-patient study tests CRD-TMH-001, an intravenous gene-editing therapy, for a rare mutation of Duchenne muscular dystrophy, focusing on safety.
Read report →A registry follows adults with colorectal cancer that has spread only to the liver who receive a liver transplant, tracking survival, recurrence, and quality of
Read report →A phase 1 trial tests engineered donor T cells plus interleukin-2 delivered into recurrent malignant glioma tumors to see if the approach is safe and feasible.
Read report →Trials whose completion date fell in this window and whose status is actually Completed. Results usually follow later.