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BEAM-201 is being evaluated in a Phase I/II clinical study for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma (T-ALL/T-LL). The ongoing trial of BEAM-201 is a multicenter, open-label study evaluating safety and efficacy in patients with r/r T-ALL/T-LL.
Biotechnology company Beam Therapeutics has announced that in August, the first patient was treated with BEAM-201, a quadruplex-edited allogeneic CAR-T cell investigational therapy for r/r T-ALL/T-LL, a highly aggressive blood cancer arising from malignant transformation of T cell precursors, with few treatment options. Beam’s proprietary gene editing technologies are designed to enable precise, predictable, and efficient single-base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA.
John Evans, CEO of Beam commented on the potential of the base edited therapy of BEAM-201 representing a major milestone for the company, the scientists, and the patients they hope to serve:
“BEAM-201, to our knowledge the first quadruplex-edited cell therapy candidate in clinical development, is an allogeneic CAR-T cell investigational therapy with the potential to make a substantial impact for patients diagnosed with challenging T-cell cancers who have limited treatment options.”
The quadruplex base-edited therapy consists of an anti-CD7 allogeneic chimeric antigen receptor T cell (CAR-T). Multiplexed base editing is designed to eliminate the expression of the CD7, TRAC, PDCD1 and CD52 genes. This has the potential to reduce fratricide, graft-versus-host disease, and CAR-T exhaustion to enable BEAM-201 cells to evade anti-CD52 lymphodepleting agents and enable the use of an allogeneic cell source.
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Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities.
“Base editing is especially well-suited to this challenge, as it is designed to deliver highly efficient multiplex edits in cells without the double-stranded breaks that can lead to frequent chromosomal rearrangements and loss of cell viability.”
Enrolment for the trail is open, with multiple sites (NCT05885464).
Source: Beam Therapeutics Press Release
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