Beam Therapeutics Inc., a firm in the biotechnology sector focusing on the creation of exact genetic treatments via base editing, declared that the first patient received BEAM-201 treatment. This new treatment is a quadruplex-edited allogeneic CAR-T cell therapy under investigation. The evaluation of BEAM-201 is underway in a clinical Phase 1/2 trial, with the aim to treat severe cases of relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma, which is a harmful condition in both children and adults.
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"With the first patient being treated with a therapeutic candidate from Beam and the first U.S. patient benefiting from a base editing therapy, we've reached a critical step forward for our organization, our dedicated scientists, and the patients we aim to assist," stated Beam's CEO, John Evans.
Beam-201 is being assessed in a multi-center, open-label Phase 1/2 trial related to safety and effectiveness in patients afflicted with relapsed/refractory T-ALL/T-LL. T-ALL/T-LL, a highly aggressive blood cancer resulting from malignant T cell precursor transformations, has limited therapeutic alternatives.
The first portion of the trial aims to evaluate safety, tolerability, and to identify the recommended Phase 2 dosage and lymphodepletion procedures. Important outcomes for the trial take into account treatment-induced and treatment-related side effects. The main efficacy objectives involve the proportion of patients that either yield complete or partial responses, those eligible for hematopoietic stem cell transplant, and those who reach a state of minimum residual disease negativity.
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According to the data provided by the Synapse Database, As of September 7, 2023, there are 35 investigational drugs for the CD7 target, including 29 applicable indications,28 R&D institutions involved, with related clinical trials reaching 65,and as many as 8048 patents.
BEAM-201, anti-CD7 allogeneic chimeric antigen receptor T cell is currently undergoing clinical trials for the treatment of CD7+ relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma. The intent behind multiplex base editing is to suppress the expression of CD7, TRAC, PDCD1, and CD52 genes. This strategy can potentially lessen fratricide, graft-versus-host disease, and exhaustion of CAR-T cells. Moreover, it allows BEAM-201 cells to avoid anti-CD52 lymphodepleting agents and to utilize an allogeneic cell resource.