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- ||| tremtelectogene empogeditemcel (VOR33) / Vor Biopharma
Enrollment change, Trial completion date, Trial primary completion date: VBP101: Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS (clinicaltrials.gov) - Jan 25, 2024
P1/2, N=24, Recruiting, Sponsor: Vor Biopharma
N=18 --> 24 | Trial completion date: Sep 2025 --> Jan 2027 | Trial primary completion date: Dec 2023 --> Feb 2025
- Mylotarg (gemtuzumab ozogamicin) / UCB, PDL, Pfizer
Trem-Cel, a CRISPR/Cas9 Gene-Edited Allograft Lacking CD33, Shows Rapid Primary Engraftment with CD33-Negative Hematopoiesis in Patients with High-Risk Acute Myeloid Leukemia (AML) and Avoids Hematopoietic Toxicity during Gemtuzumab Ozogamicin (GO) Maintenance Post-Hematopoietic Cell Transplant (HCT). () - Dec 11, 2023 - Abstract #TCTASTCTCIBMTR2024TCT_ASTCT_CIBMTR_981;
P1/2
The exposure at 0.5mg/m2 GO dose corresponded to higher doses of GO in AML patients, possibly due to reduction of the CD33 hematopoietic antigen sink. Minimal cytopenias have been observed thus far after treatment with GO, supporting the hypothesis that CD33 deletion can protect donor cells from CD33-targeted therapies.
- tremtelectogene empogeditemcel (VOR33) / Vor Biopharma, Mylotarg (gemtuzumab ozogamicin) / UCB, PDL, Pfizer
Trem-Cel, a CRISPR/Cas9 Gene-Edited Allograft Lacking CD33, Shows Rapid Primary Engraftment with CD33-Negative Hematopoiesis in Patients with High-Risk Acute Myeloid Leukemia (AML) and Avoid... (SDCC - Ballroom 20CD) - Nov 3, 2023 - Abstract #ASH2023ASH_3106;
P1/2
Tremtelectogene empogeditemcel (trem-cel; formerly VOR33) is a hematopoietic stem and progenitor cell product, manufactured from CD34+ cells isolated from a patient-matched donor, that has been modified by CRISPR/Cas9 gene-editing to lack CD33...Donors undergo mobilization with G-CSF and plerixafor prior to apheresis...Patients undergo either a busulfan- or TBI-based myeloablative conditioning regimen prior to transplantation with trem-cel... To date, 6 patients between 32-68 y (median 63.5 y) have been treated with trem-cel at a median dose of 5.2 x 10 6 CD34+ cells/kg (2.6 - 7.6) and CD33 editing efficiency of 88% (80
- | tremtelectogene empogeditemcel (VOR33) / Vor Biopharma
Trial primary completion date: VBP101: Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS (clinicaltrials.gov) - May 19, 2023
P1/2, N=18, Recruiting, Sponsor: Vor Biopharma
To date, 6 patients between 32-68 y (median 63.5 y) have been treated with trem-cel at a median dose of 5.2 x 10 6 CD34+ cells/kg (2.6 - 7.6) and CD33 editing efficiency of 88% (80 Trial primary completion date: May 2023 --> Dec 2023
- Mylotarg (gemtuzumab ozogamicin) / UCB, PDL, Pfizer
CD33-DELETED HEMATOPOIETIC STEM AND PROGENITOR CELLS DISPLAY NORMAL ENGRAFTMENT AFTER HEMATOPOIETIC CELL TRANSPLANT (HCT) AND TOLERATE POST-HCT GEMTUZUMAB OZOGAMICIN (GO) WITHOUT CYTOPENIAS (Poster area) - May 12, 2023 - Abstract #EHA2023EHA_1708;
P1/2
Consistent with high CD33 editing efficiency in trem-cel, the majority of PB and BM myeloid cells lacked CD33 expression. The CD33 deletion was detected across donor cells of myeloid and lymphoid lineages, suggesting successful editing of a multipotent progenitor.
- Mylotarg (gemtuzumab ozogamicin) / UCB, PDL, Pfizer
INITIAL FIRST-IN-HUMAN RESULTS: CD33-DELETED HEMATOPOIETIC STEM AND PROGENITOR CELLS DISPLAY NORMAL ENGRAFTMENT AFTER HEMATOPOIETIC CELL TRANSPLANT (HCT) AND TOLERATE POST-HCT GEMTUZUMAB OZOGAMICIN (GO) WITHOUT CYTOPENIAS (ePoster Area) - Feb 11, 2023 - Abstract #EBMT2023EBMT_1281;
P1/2
Engraftment was comparable to similarly treated patients who received non-edited CD34-selected grafts (median 11 days) (Luznik et al 2022, JCO 40:356-368). Consistent with high CD33 editing efficiency in trem-cel, the majority of PB and BM myeloid cells lacked CD33 expression.
- Mylotarg (gemtuzumab ozogamicin) / UCB, PDL, Pfizer
Initial First-in-Human Results: CD33-Deleted Hematopoietic Stem and Progenitor Cells Display Normal Engraftment after Hematopoietic Cell Transplant (HCT) and Tolerate Post-HCT Gemtuzumab Ozogamicin (GO) without Cytopenias (World Center Marriott Magnolia 22-24; In-Person) - Jan 22, 2023 - Abstract #TCTASTCTCIBMTR2023TCT_ASTCT_CIBMTR_1435;
P1/2
Engraftment was comparable to similarly treated patients who received non-edited CD34-selected grafts (median 11 days) (Luznik et al 2022, JCO 40:356-368). Consistent with high CD33 editing efficiency in trem-cel, the majority of PB and BM myeloid cells lacked CD33 expression.
- VOR33 / Vor Biopharma, Mylotarg (gemtuzumab ozogamicin) / UCB, PDL, Pfizer
Trial in Progress: A First-in-Human Phase 1/2 Study of Hematopoietic Stem Cell Transplantation (HCT) with VOR33 (CD33-Deleted Allograft) Followed By Treatment with Gemtuzumab Ozogamicin (GO) in Patients with CD33+ Acute Myeloid Leukemia (AML) Who Are at High-Risk of Post-HCT Relapse () - Nov 29, 2022 - Abstract #ASH2022ASH_7923;
P=N/A, P1/
Key exclusion criteria include prior auto/allo-HCT or prior treatment with GO.Related and unrelated donors undergo mobilization with G-CSF and plerixafor prior to cell collection...Subjects undergo either a busulfan- or TBI-based myeloablative conditioning regimen prior to transplantation with VOR33...A Dose Escalation Committee and Data Safety Monitoring Committee will review GO dosing levels and monitor overall trial safety, respectively. VBP101 is currently open for enrollment and recruiting patients.
- ||| tremtelectogene empogeditemcel (VOR33) / Vor Biopharma
Enrollment open: A Long-term Follow-up Study of Patients Who Received VOR33 (clinicaltrials.gov) - Apr 11, 2022
P=N/A, N=36, Recruiting, Sponsor: Vor Biopharma
VBP101 is currently open for enrollment and recruiting patients. Not yet recruiting --> Recruiting
- |||||| tremtelectogene empogeditemcel (VOR33) / Vor Biopharma
New trial: A Long-term Follow-up Study of Patients Who Received VOR33 (clinicaltrials.gov) - Apr 3, 2022
P=N/A, N=36, Not yet recruiting, Sponsor: Vor Biopharma
- Mozobil (plerixafor) / Sanofi
G-CSF/Plerixafor Dual-Mobilized Donor Derived CD33CAR T-Cells As Potent and Effective AML Therapy in Pre-Clinical Models (GWCC - Hall B5, Level 1) - Nov 5, 2021 - Abstract #ASH2021ASH_3299;
Non-transduced T cells in the mob-CAR T-cell population showed limited ‘bystander’ activation, indicating a potentially favorable clinical toxicity profile. Additional in vivo assessment of mob-CAR T-cell function shows effective tumor clearance, which supports further efforts towards their clinical use in combination with engineered HSCs for the treatment of AML patients.
- VOR33 / Vor Biopharma
[VIRTUAL] In depth assessment of offtarget editing by CRISPR/Cas9 in VOR33, an engineered hematopoietic stem cell transplant for the treatment of acute myeloid leukemia () - Oct 2, 2021 - Abstract #ESGCT2021ESGCT_328;
This assessment of offtarget editing establishes a clinically translatable safety framework to evaluate genotoxicity in CD34+ HSPCbased cell therapies for AML treatment. equal contribution
- VOR33 / Vor Biopharma
[VIRTUAL] Preclinical evaluation, including genomic offtarget analysis, of VOR33: a clinicready CRISPR/Cas9 engineered hematopoietic stem cell transplant for the treatment of acute myeloid leukemia (Studio 3) - Sep 10, 2021 - Abstract #ESGCT2021ESGCT_115;
Offtarget editing assessment using unbiased cellbased GUIDEseq and sequence homologybased hybrid capture NGS analysis revealed no significant or reproducible offtarget editing events. These studies set the stage for a firstinhuman clinical trial of VOR33 to evaluate its safety and efficacy in patients with AML.
- | tremtelectogene empogeditemcel (VOR33) / Vor Biopharma
Enrollment status: VBP101: Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS (clinicaltrials.gov) - Sep 5, 2021
P1/2, N=18, Recruiting, Sponsor: Vor Biopharma
These studies set the stage for a firstinhuman clinical trial of VOR33 to evaluate its safety and efficacy in patients with AML. Enrolling by invitation --> Recruiting
- || tremtelectogene empogeditemcel (VOR33) / Vor Biopharma
Enrollment open, Trial initiation date: VBP101: Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS (clinicaltrials.gov) - Sep 2, 2021
P1/2, N=18, Enrolling by invitation, Sponsor: Vor Biopharma
Enrolling by invitation --> Recruiting Not yet recruiting --> Enrolling by invitation | Initiation date: Apr 2021 --> Aug 2021
- VOR33 / Vor Biopharma
[VIRTUAL] Rigorous Assessment of Off-Target Editing by CRISPR/Cas9 in VOR33, an Engineered Hematopoietic Stem Cell Transplant for the Treatment of Acute Myeloid Leukemia () - Apr 30, 2021 - Abstract #ASGCT2021ASGCT_858;
Through karyotyping, long-read on-target sequencing, and quantification of indel frequencies, we achieved an expansive appraisal of off-target events across multiple VOR33 products. This assessment of off-target editing by CRISPR/Cas9 establishes a rigorous and clinically applicable framework to evaluate off-target events in CD34+ HSPC-based cell therapies.
- VOR33 / Vor Biopharma
[VIRTUAL] VOR33: A Clinic-Ready CRISPR/Cas9 Engineered Hematopoietic Stem Cell Transplant for the Treatment of Acute Myeloid Leukemia () - Apr 30, 2021 - Abstract #ASGCT2021ASGCT_7;
CD34+ cells, isolated from G-CSF and plerixafor mobilized peripheral blood leukapheresis products, were edited using CRISPR/Cas9 to disrupt CD33 gene...Importantly, we found loss of CD33 protein conferred selective protection to VOR33-derived myeloid cells vs Mylotarg in vitro (>65-fold) and in vivo (>60-fold)... These studies set the stage for initiation of, as well as evaluation of safety and efficacy in, a multicenter first-in-human clinical trial of VOR33 in pts with AML.
- | tremtelectogene empogeditemcel (VOR33) / Vor Biopharma
New P1/2 trial: VBP101: Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS (clinicaltrials.gov) - Apr 18, 2021
P1/2, N=18, Not yet recruiting, Sponsor: Vor Biopharma