gene therapy – Page 2

Haematopoietic Gene Therapy Without Conditioning for Fanconi Anaemia-A: Results and Clinical Implications from the FANCOLEN-1 Trials

By MedXY 08/02/2025

Gene therapy using autologous, gene-corrected stem cells without conditioning offers sustained engraftment and reverses bone marrow failure in Fanconi anaemia-A, with a favorable safety profile.

Clinical Updates Hematology-Oncology news Specialties

Durable Efficacy and Long-term Safety of AAV Gene Therapy in Severe Hemophilia B: 13-Year Follow-up Results

By MedXY 08/01/2025

A single AAV gene therapy infusion for severe hemophilia B led to sustained factor IX expression, substantial reduction in bleeding and factor use, and no late safety concerns over 13 years.