Ep154: Robert Ang on CRISPR to Protect Bone Marrow & Attack Cancer
Feb 9, 2024
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Robert Ang, CEO of Vor Bio, discusses using CRISPR gene editing to alter bone marrow transplants and improve treatment of acute myeloid leukemia. Topics include personal background, challenges of personalized medicine in cancer treatment, potential applications of CRISPR technology, and financial challenges in biotechnology.
VOR Bio is using CRISPR gene editing to delete the CD33 antigen gene from healthy bone marrow cells, protecting them from toxic therapies and exposing cancer cells for targeted treatments.
Early clinical trial results have shown successful engraftment of edited cells and reduced toxicity in patients treated with VOR Bio's CRISPR gene editing approach.
VOR Bio is also developing a CD33-directed CAR-T cell therapy to further enhance acute myeloid leukemia (AML) treatment.
Deep dives
The Promise of CRISPR Gene Editing in AML Treatment
VOR Bio, a biotech company in Cambridge, is developing a unique application of CRISPR gene editing for the treatment of acute myeloid leukemia (AML). By using CRISPR to delete the gene for the CD33 antigen found on both cancer cells and healthy blood-forming stem cells, VOR aims to make AML patients more responsive to targeted therapies like Pfizer's Myletarg. Preliminary clinical trial results have shown successful deletion of CD33 from stem cells, the engraftment of edited cells in the bone marrow, and promising safety profiles. VOR plans to expand its approach by developing a CD33-directed CAR-T cell therapy, leveraging CRISPR to protect bone marrow transplants and enable the T-cell therapy to kill remaining cancer cells. By harnessing the potential of CRISPR and cell therapies, VOR aims to revolutionize AML treatment and potentially achieve long-term remission and cure for patients.
A New Perspective on Cancer Treatment
VOR Bio is challenging the traditional approach to cancer treatment by targeting healthy cells instead of cancer cells. By using CRISPR gene editing, VOR can delete the CD33 antigen gene from healthy bone marrow cells, protecting them from toxic therapies while exposing cancer cells that bear the CD33 antigen. This unique strategy has shown promising results in early clinical trials, with patients successfully engrafting edited cells and receiving targeted therapies with reduced toxicity. VOR is also developing a CD33-directed CAR-T cell therapy for AML patients after bone marrow transplantation. With the potential for cures and improved patient outcomes, VOR is reshaping the future of cancer treatment.
Overcoming the Challenges of AML Treatment
VOR Bio's innovative approach to acute myeloid leukemia (AML) treatment addresses the challenge of toxic therapies that harm healthy bone marrow cells. By using CRISPR gene editing to delete the CD33 antigen gene from healthy cells, VOR protects them from targeted therapies like Pfizer's Myletarg, which attack CD33-bearing cancer cells. Early clinical trial results have shown successful engraftment of edited cells and reduced toxicity in patients. VOR is also developing a CD33-directed CAR-T cell therapy to further target AML cells. With a focus on changing the lives of AML patients, VOR is pushing the boundaries of traditional cancer treatment.
VOR Bio's Pursuit of Improved AML Treatment
VOR Bio is revolutionizing acute myeloid leukemia (AML) treatment by utilizing CRISPR gene editing to protect healthy bone marrow cells while targeting CD33-bearing cancer cells. By deleting the CD33 antigen gene, VOR aims to shield healthy cells from toxic therapies, enabling patients to receive more effective treatments without damaging their bone marrow. Preliminary clinical trial results have been promising, demonstrating successful engraftment of edited cells and reduced toxicity. VOR is also developing a CD33-directed CAR-T cell therapy to further enhance AML treatment. With its transformative approach and commitment to patient outcomes, VOR Bio is pushing the boundaries of AML treatment.
Harnessing CRISPR for Improved AML Treatment
VOR Bio is leveraging the power of CRISPR gene editing to improve treatment options for acute myeloid leukemia (AML) patients. By deleting the CD33 antigen gene, VOR aims to protect healthy bone marrow cells from toxic therapies while exposing CD33-bearing cancer cells to targeted treatments. Early clinical trial results have shown successful engraftment of edited cells and reduced toxicity in patients. VOR is also developing a CD33-directed CAR-T cell therapy as a potential follow-up treatment after bone marrow transplantation. By utilizing CRISPR to enhance current therapies, VOR Bio is paving the way for more effective and less toxic AML treatments.
Robert Ang, CEO of Cambridge, Mass.-based Vor Bio, on using CRISPR gene editing to alter bone marrow transplants and improve treatment of acute myeloid leukemia.
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