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▎ WuXi AppTec content team editor July 8, 2021, Vor Biopharma announced that it has reached a cooperation agreement with Janssen Biotech, a subsidiary of Johnson & Johnson
.
It will combine Vor's proprietary engineered hematopoietic stem cell (eHSC) technology platform with Janssen's bispecific antibody therapy for the treatment of acute myeloid leukemia (AML) to develop an innovative combination for the treatment of AML
.
In cell therapy, the eHSC platform developed by Vor has an innovative mode of action, and is often used in combination with other targeted therapies for the treatment of tumors
.
In some cases, both tumor cells and healthy cells express targets that the drugs are designed to attack
.
Therefore, although these targeted therapies correctly bind to the target when performing the "killing enemy" task, they may also "injury" healthy cells (on-target off tumor effect), causing treatment-related adverse reactions
.
▲Anti-cancer targeted therapy may accidentally injure healthy cells (picture source: Vor's official website).
On the eHSC platform, researchers use CRISPR/Cas9 gene editing technology to modify stem cells and knock out genes encoding target proteins
.
When these genetically engineered stem cells are infused back into the patient's body, new cells will continue to be produced, and these healthy new cells no longer express the target protein of targeted therapy
.
Therefore, healthy cells are like wearing a "invisibility cloak" and will not be accidentally injured by drugs
.
Targeted therapy can also let go of this and concentrate on dealing with tumor cells
.
▲The working mechanism of the eHSC platform (picture source: Vor's official website) Take the research therapy VOR33 in the Vor R&D pipeline as an example.
In AML patients, the surface of tumor cells will express a large amount of CD33 protein
.
In the past, some large-scale genomic studies have shown that many blood cells can survive healthy without expressing CD33
.
Therefore, by knocking out the CD33 gene of hematopoietic stem cells in vitro and returning it to the patient's body to reduce the healthy cells expressing CD33, therapies targeting CD33 can improve specificity while reducing its potential side effects
.
Dr.
Tirtha Chakraborty, Chief Scientific Officer of Vor, said: “As we continue to explore the potential of the eHSC platform combined with broad-spectrum blood cancer targeted therapies, we are very pleased to reach this collaboration with Janssen
.
We believe that this unique combination will make full use of each The advantages of this technology, while reducing the side effects of these powerful immunotherapy
.
"Reference: [1] Vor Biopharma Announces Collaboration with Janssen to Develop Engineered Hematopoietic Stem Cell Transplants Combined With a Bi-Specific Antibody Therapy for Acute Myeloid Leukemia (AML).
Retrieved July 8, 2021, from https:// .
com/news-release/2021/07/08/2259858/0/en/Vor-Biopharma-Announces-Collaboration-with-Janssen-to-Develop-Engineered-Hematopoietic-Stem-Cell-Transplants-Combined-With-a -Bi-Specific-Antibody-Therapy-for-Acute-Myeloid-Leukemia.
html Note: This article aims to introduce the progress of medical and health research, not a treatment plan recommendation
.
If you need treatment plan guidance, please go to a regular hospital
.
.
It will combine Vor's proprietary engineered hematopoietic stem cell (eHSC) technology platform with Janssen's bispecific antibody therapy for the treatment of acute myeloid leukemia (AML) to develop an innovative combination for the treatment of AML
.
In cell therapy, the eHSC platform developed by Vor has an innovative mode of action, and is often used in combination with other targeted therapies for the treatment of tumors
.
In some cases, both tumor cells and healthy cells express targets that the drugs are designed to attack
.
Therefore, although these targeted therapies correctly bind to the target when performing the "killing enemy" task, they may also "injury" healthy cells (on-target off tumor effect), causing treatment-related adverse reactions
.
▲Anti-cancer targeted therapy may accidentally injure healthy cells (picture source: Vor's official website).
On the eHSC platform, researchers use CRISPR/Cas9 gene editing technology to modify stem cells and knock out genes encoding target proteins
.
When these genetically engineered stem cells are infused back into the patient's body, new cells will continue to be produced, and these healthy new cells no longer express the target protein of targeted therapy
.
Therefore, healthy cells are like wearing a "invisibility cloak" and will not be accidentally injured by drugs
.
Targeted therapy can also let go of this and concentrate on dealing with tumor cells
.
▲The working mechanism of the eHSC platform (picture source: Vor's official website) Take the research therapy VOR33 in the Vor R&D pipeline as an example.
In AML patients, the surface of tumor cells will express a large amount of CD33 protein
.
In the past, some large-scale genomic studies have shown that many blood cells can survive healthy without expressing CD33
.
Therefore, by knocking out the CD33 gene of hematopoietic stem cells in vitro and returning it to the patient's body to reduce the healthy cells expressing CD33, therapies targeting CD33 can improve specificity while reducing its potential side effects
.
Dr.
Tirtha Chakraborty, Chief Scientific Officer of Vor, said: “As we continue to explore the potential of the eHSC platform combined with broad-spectrum blood cancer targeted therapies, we are very pleased to reach this collaboration with Janssen
.
We believe that this unique combination will make full use of each The advantages of this technology, while reducing the side effects of these powerful immunotherapy
.
"Reference: [1] Vor Biopharma Announces Collaboration with Janssen to Develop Engineered Hematopoietic Stem Cell Transplants Combined With a Bi-Specific Antibody Therapy for Acute Myeloid Leukemia (AML).
Retrieved July 8, 2021, from https:// .
com/news-release/2021/07/08/2259858/0/en/Vor-Biopharma-Announces-Collaboration-with-Janssen-to-Develop-Engineered-Hematopoietic-Stem-Cell-Transplants-Combined-With-a -Bi-Specific-Antibody-Therapy-for-Acute-Myeloid-Leukemia.
html Note: This article aims to introduce the progress of medical and health research, not a treatment plan recommendation
.
If you need treatment plan guidance, please go to a regular hospital
.
