Drugs / tremtelectogene empogeditemcel
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04849910 | Dec 2021 → May 2025 | acute myeloid leukemia, myelodysplastic syndrome | Vor Biopharma | Terminated | Met primary Stop: Business |
News releases announcing trial results or a regulatory action · 3
| Date | Issuer | Release |
|---|---|---|
| 2022-12-07 | PureTech | Results PureTech Founded Entity Vor Bio Announces First AML Patient Successfully Transplanted with its Investigational Trem-cel (VOR33) and Tolerated Mylotarg™ puretechhealth.com ↗
The data observed from the first treated patient support the potential of a trem-cel transplant to be successfully manufactured, to engraft normally, and to maintain blood counts following treatment with the CD33-targeted therapy Mylotarg. |
| 2021-09-10 | PureTech | Regulatory PureTech Founded Entity Vor Biopharma Announces VOR33 Granted U.S. FDA Fast Track Designation for Acute Myeloid Leukemia puretechhealth.com ↗
the U.S. Food and Drug Administration ("FDA") has granted Fast Track designation to VOR33, Vor's lead engineered hematopoietic stem cell ("eHSC") therapeutic candidate for the treatment of acute myeloid leukemia ("AML"). |
| 2021-01-14 | PureTech | Regulatory PureTech Founded Entity Vor Announces FDA Clearance of IND Application for VOR33 puretechhealth.com ↗ |
All press releases naming this drug 3 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | tremtelectogene empogeditemcel | “creating tremtelectogene empogeditemcel (trem-cel).” PMID 38027064 ↗ Oct 2023 |
| Known as | trem-cel | “creating tremtelectogene empogeditemcel (trem-cel).” PMID 38027064 ↗ Oct 2023 |
| Known as | VOR33 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04849910 ↗ |
| Action | Inhibit | “we used CRISPR-Cas9 to genetically ablate the myeloid protein CD33 from healthy donor hematopoietic stem and progenitor cells (HSPCs)” PMID 38027064 ↗ Oct 2023 |
| Modality | Cell therapy | “an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell (HSPC) therapy product” NCT04849910 ↗ |
| Target | CD33 | “we used CRISPR-Cas9 to genetically ablate the myeloid protein CD33 from healthy donor hematopoietic stem and progenitor cells (HSPCs)” PMID 38027064 ↗ Oct 2023 |