Rocket Pharmaceuticals Inc.

This trial tests a one-time gene therapy for people with pyruvate kinase deficiency, a rare blood disorder causing severe anemia. Patients receive their own blood stem cells modified to produce a working enzyme. The goal is to raise hemoglobin levels and reduce or eliminate the n…

This study follows 9 people with Fanconi Anemia who received a one-time gene therapy to fix a faulty gene. Researchers will monitor their health and blood counts for 15 years to see if the treatment remains safe and effective. No new treatment is given during this follow-up perio…

This study follows 9 people with a rare immune disorder called LAD-I who previously received a one-time gene therapy. The goal is to see if the treatment remains safe and effective over time, and whether it helps them avoid needing a stem cell transplant. Researchers will track i…

This study tests a gene therapy for children with Fanconi anemia subtype A, a rare genetic condition that leads to bone marrow failure. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and put the corrected cells back into the body. The goal is to rest…

This study checks the long-term safety and effectiveness of a gene therapy called RP-L102 for people with Fanconi Anemia, a rare genetic condition that causes bone marrow failure and raises cancer risk. About 14 patients who already received RP-L102 in earlier studies will be fol…