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

▎ WuXi AppTec content team editor September 9, 2021, Vor Biopharma announced that the US 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
.

Some large-scale genomic studies in the past have shown that many blood cells can survive healthy without expressing CD33
.

VOR33 is developed based on Vor's proprietary eHSC platform
.

On the eHSC platform, researchers use CRISPR/Cas9 gene editing technology to modify hematopoietic stem cells in vitro to knock out the gene encoding CD33
.

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
.

Targeted therapy can also let go of this, focus on dealing with tumor cells, while improving specificity, while reducing its potential side effects
.

VOR33 is designed to replace the standard transplantation treatment for AML patients who are at high risk of recurrence
.

▲The working mechanism of the eHSC platform (picture source: Vor's official website) "Obtaining fast track qualification is an important milestone for Vor, which marks the U.
S.
FDA's recognition of the potential of VOR33 to solve the major unmet medical needs of AML
.

" Chief Supervisor of Vor Quality Officer Mr.
Robert Pietrusko said, “We will continue to work closely with the FDA to accelerate the development of VOR33.
We are currently actively recruiting AML patients with limited treatment options to enter the Phase 1/2a clinical trial of VOR33
.

We expect to be in 2022.
The preliminary clinical data of VOR33 are reported half-yearly
.

"Reference: [1] VOR33 Granted US FDA Fast Track Designation for AML.
Retrieved September 9, 2021, from https://ir.
vorbio.
com/news-releases/news-release- details/vor33-granted-us-fda-fast-track-designation-aml Disclaimer: WuXi AppTec's content team focuses on introducing global biomedical health research progress
.

This article is for the purpose of information exchange only.
The opinions expressed in the article do not represent the position of WuXi AppTec, nor does it mean that WuXi AppTec supports or opposes the views in the article
.

This article is not a treatment recommendation either
.

If you need guidance on the treatment plan, please go to a regular hospital for treatment
.