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FDA grants Fast Track Designation for Vor Biopharma’s AML drug

Published on 10/09/21 at 09:18am

Vor Biopharma has announced that the FDA has granted their acute myeloid leukemia (AML) engineered hematopoietic stem cell therapy (eHSC), VOR33, Fast Track Designation.

VOR33 consists of CRISPR genome-edited hematopoietic stem and progenitor cells that have been engineered to lack CD33. Once infused, VOR33 is designed to protect patients’ healthy cells from anti-CD33 therapies, such as VCAR33 or Mylotarg (gemtuzumab ozogamicin).

VOR33 is intended to replace standard of care transplants for AML patients who are at high risk of relapse and has the potential to seamlessly integrate into current transplant settings.

Robert Pietrusko, Vor’s Chief Regulatory and Quality Officer, said: “Receiving Fast Track Designation is an important milestone for Vor, which signals the FDA’s recognition of the serious and life-threatening medical condition of patients facing acute myeloid leukaemia and the potential of VOR33 to address this unmet medical need.

“We will continue to work closely with the FDA to expedite the development of VOR33, which is now actively enrolling in its Phase 1/2a clinical trial for AML patients who currently have limited treatment options. We continue to remain on-track to report VOR33’s initial clinical data in the first half of 2022.”

VOR33 was developed through Vor’s genome eHSC platform in combination with multiple therapeutic modalities, with the aim of creating treatment-resistant transplants that can enhance the potential of targeted cancer therapies through cell therapy and gene editing.

In a statement, Vor said it is actively enrolling in its Phase I/II clinical trial for AML patients who currently have limited treatment options.

Vor is also currently exploring the use of its genome engineered hematopoietic stem cell platform in combination with multiple therapeutic modalities.

Kat Jenkins

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