BEAM-103 / Beam Therap - LARVOL DELTA

Beam Therapeutics Presents New Non-human Primate (NHP) Data Demonstrating Proof-of-concept for ESCAPE, a Non-genotoxic, Antibody-based Conditioning Approach to Treating Sickle Cell Disease, at American Society of Hematology (ASH) Annual Meeting (GlobeNewswire) - "ESCAPE is comprised of two investigational drug products: BEAM-103...and BEAM-104...The company intends to advance BEAM-103 and BEAM-104 for development in sickle cell disease (SCD) and beta-thalassemia...Long term engraftment of HSCs in the marrow was demonstrated by the presence of edited cells in the periphery beyond 6 months. Dosing with the BEAM-103 mAb led to rapid and near complete replacement of wild-type erythroid cells by edited cells, leading to early induction of therapeutically relevant levels of HbF. Levels of cells containing HbF reached >80% post-transplant. All NHPs achieved >40% γ-globin, a key constituent of HbF, post-transplant. Rapid and sustained reactivation of HbF post-transplant showed promise of therapeutic benefit in SCD patients. BEAM-103 dosing was well tolerated with no need for transfusions, antibiotics or additional supportive care...No changes to CD117 signaling, structure or expression were observed following editing."

Preclinical • Beta-Thalassemia • Sickle Cell Disease