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CRISPR-edited stem cell therapy for the treatment of acute myeloid leukemia obtains FDA fast-track qualification

 Last Update: 2021-09-19
 Source: Internet
 Author: User

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On September 9, Vor Biopharma announced that the U.
S.
FDA has granted its engineered hematopoietic stem cell (eHSC) candidate drug VOR33 fast track qualification for the treatment of acute myeloid leukemia (AML)
.

In AML patients, CD33 protein is expressed on the surface of tumor cells in large quantities
.

Targeted therapies targeting CD33 may cause toxic side effects due to the combination of CD33 expressed on the surface of healthy cells and "injury" to healthy cells

Therefore, the hematopoietic stem cells infused back into the patient will produce healthy new cells that do not express CD33 protein
.

Healthy cells are like wearing a "invisibility cloak" and will not be accidentally injured by drugs

▲The working mechanism of the eHSC platform (picture source: Vor official website)

Note: The original text has been deleted

Reference materials:

[1] VOR33 Granted US FDA Fast Track Designation for AML.

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Express treatment of acute myeloid leukemia, CRISPR-edited stem cell therapy obtained FDA fast track qualification

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CRISPR-edited stem cell therapy for the treatment of acute myeloid leukemia obtains FDA fast-track qualification

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Express treatment of acute myeloid leukemia, CRISPR-edited stem cell therapy obtained FDA fast track qualification