Vor Biopharma, Inc. is a clinical-stage biopharmaceutical company focused on developing allogeneic, or “off-the-shelf,” cell therapies for patients with blood cancers and other malignancies. Utilizing its proprietary ProCellics platform, Vor engineers healthy donor-derived hematopoietic stem cells (HSCs) with precise gene edits that confer resistance to targeted oncology agents. This approach aims to create a new class of programmable cell therapies that can both eradicate cancer cells and restore normal blood formation, offering the potential for improved safety and broader applicability compared with autologous modalities.
The company’s lead program, VOR33, is designed to protect healthy myeloid stem cells by knocking out the CD33 gene, enabling high-potency CD33-targeted treatments to eliminate acute myeloid leukemia (AML) cells without causing prolonged myelosuppression. Early-stage clinical trials are evaluating VOR33 in combination with antibody-drug conjugates and bispecific antibodies for relapsed or refractory AML. In parallel, Vor is advancing several preclinical programs that leverage its gene-editing expertise to address B-cell malignancies and other hematologic disorders, as well as exploring natural killer (NK) cell and T-cell platforms for solid tumor indications.
Founded in 2018 and headquartered in Cambridge, Massachusetts, Vor Biopharma brings together a multidisciplinary team of scientists, clinicians and industry veterans with extensive experience in gene editing, immuno-oncology and cell therapy manufacturing. The company maintains state-of-the-art research laboratories and scalable manufacturing capabilities designed to support both early-stage development and future commercial supply. Vor’s senior leadership team has successfully guided multiple cell therapy programs from discovery through clinical proof-of-concept.
Vor Biopharma collaborates with leading academic centers, research institutions and contract development organizations across North America and Europe to accelerate its pipeline. By integrating cutting-edge gene-editing technologies with strategic partnerships, the company aims to deliver transformative cell therapies that meet unmet needs in oncology and beyond. With ongoing trial enrollment and a robust preclinical portfolio, Vor remains committed to advancing novel treatments that could redefine standards of care for patients with life-threatening diseases.
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