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-Multiplex deletion of myeloid antigens CD33 and CLL-1 or CD33 and CD123 in human hematopoietic stem cells resulted in long-term engraftment and persistence of enhancing
-Data introduced at European Society of Gene & Cell Therapy (ESGCT) Annual Congress
CAMBRIDGE, Mass., Oct. 11, 2022 (GLOBE NEWSWIRE) — Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering firm, right now introduced knowledge demonstrating the potential of the Company’s novel platform to make use of numerous base enhancing methods to efficiently delete a number of cell floor targets from hematopoietic stem cells (HSCs), probably enabling next-generation transplants for the remedy of acute myeloid leukemia (AML). The Company is presenting the information on the European Society of Gene & Cell Therapy (ESGCT) Annual Congress in Edinburgh, UK in two separate poster shows.
“Because most tumor antigens are also expressed on normal blood cells, traditional multi-targeted immunotherapy increases the risk of severe cytopenia,” defined Tirtha Chakraborty, Ph.D., Vor Bio’s Chief Scientific Officer. “These data show that we can potentially address this challenge by using our platform to efficiently remove one or multiple target antigens from HSCs. This approach to transplant, coupled with targeted therapies, may enable the delivery of more efficacious, safer and durable treatments for patients with blood cancers.”
Poster Presentation Summaries
Poster Title: Efficient knockout of each CD33 and CLL-1 by multiplex genome enhancing of human hematopoietic stem cells enhances the potential of next-generation transplants for acute myeloid leukemia (AML) remedy
Poster Summary: The preclinical knowledge show that multiplex deletion by CRISPR/Cas9 of CD33 and CLL-1 from human CD34+ hematopoietic stem and progenitor cells (HSPCs) maintained cell perform and endured long-term publish engraftment in vivo, with a high-level of enhancing and no counterselection when in comparison with unedited management cells. In addition, genetically modifying HSPCs to take away choose cell floor targets, to be able to enhance security and efficacy of next-gen AML therapies, doesn’t impair their perform and these twin edited cells confirmed important safety from focused immunotherapy in vitro.
Presenter: Michelle Lin, PhD, Head of Preclinical Sciences and HSC Biology, Vor Bio
Poster ID quantity: P150
Poster Session: Thursday, thirteenth October, 2022
Poster Title: Multiplex deletion of myeloid antigens by base enhancing in human hematopoietic stem and progenitor cells (HSPCs) allows potential for next-generation transplant for acute myeloid leukemia (AML) remedy
Poster Summary: The preclinical knowledge show that multiplex deletion utilizing a number of completely different base enhancing methods to edit twin targets (cytosine base editors to edit CD33 and CLL-1 and adenine base editors to edit CD33 and CD123) from hematopoietic stem and progenitor cells (HSPCs) maintained cell perform and endured long-term publish engraftment in vivo, with a high-level of enhancing, no counterselection, and no detectable genotoxic danger when in comparison with unedited management cells. In addition, genetically modifying HSPCs to take away these cell floor targets, to be able to enhance security and efficacy, doesn’t seem to impair perform. Dual-engineered cells endured long-term, and loss of a number of antigens was well-tolerated demonstrating that base enhancing supplies an environment friendly and protected technique for multi-gene disruptions in HSPCs and can allow next-generation AML therapies.
Presenter: John Lydeard, PhD, Head of Discovery and Molecular Engineering, Vor Bio
Poster ID quantity: P432
Poster Session: Thursday, thirteenth October, 2022
The posters shows can be found on the Vor company web site at: https://www.vorbio.com/publications/
AML is the most typical kind of acute leukemia in adults and is characterised by extreme proliferation of immature myeloid progenitor cells and their failure to correctly differentiate into mature blood cells. Healthy donor HSC transplantation is the usual of care and presently round 40% of sufferers with AML who obtain HSC transplantation undergo a relapse of their most cancers, with two-year survival charges of lower than 20%, highlighting the necessity for brand new therapeutic approaches for these sufferers.
Vor Bio is creating a [leading] remedy method consisting of gene-edited HSC transplants which can be designed to be immune to focused therapies, enabling post-transplant use of highly effective therapies akin to CAR-Ts or different focused immuno-therapies. This new method has the potential to guard wholesome cells from the damaging results of cancer-targeted therapies, leaving the cancerous cells uncovered and permitting these focused therapies to kill solely the most cancers cells whereas defending the wholesome cells.
About Vor Bio
Vor Bio is a clinical-stage cell and genome engineering firm that goals to vary the usual of look after sufferers with blood cancers by engineering hematopoietic stem cells to allow focused therapies post-transplant. For extra data, go to: www.vorbio.com.
Forward-Looking Statements
This press launch comprises forward-looking statements inside the that means of the Private Securities Litigation Reform Act of 1995. The phrases “could,” “demonstrate,” “design,” “develop,” “enable,” “enhance,” “expect,” “intend,” “may,” “plan,” “potential,” “project,” “should,” “target,” and related expressions are supposed to determine forward-looking statements, though not all forward-looking statements comprise these figuring out phrases. Forward-looking statements on this press launch embrace Vor Bio’s statements relating to the potential of Vor Bio’s multiplex enhancing method for the remedy of AML and of Vor Bio’s method to enhancing HSCs extra typically, the potential efficacy, security and sturdiness of these therapies, the progress of Vor Bio’s ongoing scientific trials, and the potential of Vor Bio’s novel platform. Vor Bio could not really obtain the plans, intentions, or expectations disclosed in these forward-looking statements, and you shouldn’t place undue reliance on these forward-looking statements. Actual outcomes or occasions might differ materially from the plans, intentions and expectations disclosed in these forward-looking statements consequently of numerous components, together with: uncertainties inherent within the initiation and completion of preclinical research and scientific trials and scientific improvement of Vor Bio’s product candidates, in addition to the outcomes of such research and trials; the success of Vor Bio’s method to transplant, coupled with focused therapies, to allow the supply of extra efficacious, safer and sturdy therapies for sufferers with blood cancers; and availability of funding adequate for its foreseeable and unforeseeable working bills and capital expenditure necessities. These and different dangers are described in higher element below the caption “Risk Factors” included in Vor Bio’s most up-to-date annual or quarterly report and in different studies it has filed or could file with the Securities and Exchange Commission. Any forward-looking statements contained on this press launch converse solely as of the date hereof, and Vor Bio expressly disclaims any obligation to replace any forward-looking statements, whether or not as a result of of new data, future occasions or in any other case, besides as could also be required by regulation.
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