$Beam Therapeutics (BEAM.US)$ Beam Therapeutics Presents New...

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

Beam Therapeutics (NASDAQ: BEAM) presented new data for its ESCAPE conditioning platform at the ASH Annual Meeting. The data demonstrated successful proof-of-concept in non-human primates (NHPs) for a non-genotoxic conditioning approach to treating sickle cell disease.

The platform consists of two investigational products: BEAM-103, an anti-CD117 monoclonal antibody, and BEAM-104, a cell therapy designed to elevate fetal hemoglobin (HbF). Key findings showed long-term engraftment of base-edited hematopoietic stem cells, with HbF-containing cells reaching >80% post-transplant and γ-globin levels exceeding 40%. The treatment was well-tolerated, requiring no supportive care.

The company plans to initiate Phase 1-enabling studies by the end of 2024 for both sickle cell disease and beta-thalassemia applications.

Positive
Successful proof-of-concept achieved in NHP studies with >80% HbF-containing cells post-transplant
Treatment well-tolerated with no need for transfusions or antibiotics
Long-term engraftment demonstrated beyond 6 months
Phase 1-enabling studies planned for end of 2024

Negative
None.