Hematopoietic Stem Cell Transplant Treatment
The traditional tumor target paradigm aims to treat hematological malignancies such as acute myeloid leukemia (AML) by focusing on the specificity and potency of therapies that kill cancer cells expressing a target. However, this paradigm is limited by the expression of tumor targets on healthy cells, resulting in on-target toxicity. On-target toxicity has led to the development failure of many targeted therapies and is often a key mechanism limiting therapeutic use or dose.
Details
Our unique approach aims to help patients fight cancer by unlocking the full potential of targeted therapies with curative intent.
Our proprietary platform changes the traditional tumor target paradigm. Leveraging our expertise in hematopoietic stem cell (HSC) biology and genome engineering, we genetically modify HSCs to remove surface targets expressed by cancer cells and provide these cells as engineered hematopoietic stem cell (eHSC) transplants to patients. We believe that once these cells complete this process and engraft into the patient’s bone marrow, the HSCs and their blood cell progeny will be treatment resistant to targeted therapies and thereby unlock the potential of these therapies to selectively destroy cancerous cells while sparing healthy cells.
Our goal is to replace standard HSC transplants with next-generation, treatment-resistant eHSCs that unlock the potential of highly potent targeted therapies.
We are currently developing our lead eHSC product candidate, VOR33, and our companion therapeutic VCAR33, which together, we believe, have the potential to transform the treatment paradigm for AML and other myeloid malignancies.
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