Drug intelligence / Profile preview

AVC-201

Development stage
Phase 1
Lead developer
AvenCell Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intravenous
01

Overview

**AVC-201** is an investigational, off-the-shelf, CRISPR-engineered allogeneic switchable CAR-T therapy being developed by AvenCell for CD123-positive relapsed or refractory acute myeloid leukemia and minimal residual disease-positive acute myeloid leukemia. The treatment comprises a single infusion of multi-edited donor-derived Allo-RevCAR01-T cells plus repeated continuous intravenous administration of the CD123-directed targeting module R-TM123. The targeting module bridges the inert reverse CAR on the donor T cells to CD123 on leukemic cells, enabling controlled CAR-T activation; stopping the targeting-module infusion provides a pharmacologic on/off safety mechanism. AVC-201 is being evaluated in the Phase 1 RevSTAR-123 study. ([clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05949125))

Other names
Allo-RevCAR01-T-CD123Allo-RevCAR-01-T-CD123Allo-RevCAR 01-T-CD123
02

Targets

IL3RA (Interleukin 3 Receptor)

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