15.06.2020 12:16:28

Magenta Therapeutics, Beam Therapeutics Collaborate On Hematopoietic Stem Cells

(RTTNews) - Magenta Therapeutics (MGTA) and Beam Therapeutics (BEAM) reached a research and clinical collaboration agreement to evaluate the potential utility of MGTA-117, Magenta's novel targeted Antibody-Drug Conjugate for conditioning of patients with sickle cell disease and beta-thalassemia receiving Beam's base editing therapies.

Beam is pursuing two differentiated base editing approaches to treat hemoglobinopathies: its hereditary persistence of fetal hemoglobin or HPFH program to precisely and robustly elevate fetal hemoglobin, which could be used in treatments for both sickle cell disease and beta-thalassemia, as well as a novel approach to directly correct the sickle causing point mutation or Makassar.

The companies said that MGTA-117 precisely targets only hematopoietic stem and progenitor cells, sparing immune cells, and has shown high selectivity, potent efficacy, wide safety margins and broad tolerability in non-human primate models. MGTA-117 may be capable of clearing space in bone marrow to support long-term engraftment and rapid recovery in patients.

Beam will be responsible for clinical trial costs related to development of Beam's base editors when combined with MGTA-117, while Magenta will continue to be responsible for all other development costs of MGTA-117.

Magenta will also continue to develop MGTA-117 in other diseases, including blood cancers and genetic diseases. Each company will retain all commercial rights to their respective technologies.

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