Research & Development
Vor Biopharma wins FDA's FTD for VOR33 for AML
10 September 2021 -

Biotherapeutics company PureTech Health plc (Nasdaq:PRTC)(LSE:PRTC) reported on Friday the receipt of US Food and Drug Administration's Fast Track designation (FTD) for the VOR33 therapeutic candidate for the treatment of acute myeloid leukemia (AML).

The FDA's designation was awarded to the company's founded entity Vor Biopharma (Nasdaq:VOR) for the latter's lead engineered hematopoietic stem cell (eHSC) therapeutic candidate VOR33 for the treatment of acute myeloid leukemia (AML).

VOR's lead product candidate 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.

According to the company, 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.

Currently, Vor is enrolling AML patients with limited treatment options in its Phase 1/2a clinical trial, as well as exploring the use of its genome engineered hematopoietic stem cell platform in combination with multiple therapeutic modalities, with initial clinical data in the first half of 2022.



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