US-based biotechnology company AvenCell Therapeutics has received approval from the European Medicines Agency (EMA) to begin a Phase I study of AVC-201, a CRISPR-engineered chimeric antigen receptor-T (CAR-T) candidate.

The single-ascending dose, open-label trial is designed to assess AVC-201's pharmacodynamics, pharmacokinetics, tolerability and safety in adults with relapsed or refractory acute myeloid leukaemia (AML) and other CD123-positive haematological malignancies.

The study intends to enrol up to 35 patients and will be carried out at several sites in Germany. 

Its primary objectives are to evaluate AVC-201's safety profile and determine the maximum tolerated dose of the drug.

Measuring the drug's CAR-T persistence, efficacy and safety are the secondary objectives.

Two dose levels of AVC-201 identified in the trial will be further assessed in a subsequent Phase II clinical trial.

AvenCell president and CEO Andrew Schiermeier said: “We are excited to build on the early success and promising activity and safety of our ongoing switchable autologous clinical programme in AML (AVC-101) by now including what we believe is the most scientifically-compelling allogeneic technology in the industry.

“With the application of both platform technologies, we are the first company in this space to completely separate the manufacture of cells from the ultimate patients and cancer indications they will be targeted to.

“This modular approach allows for unparalleled future flexibility and reduction in cycle times, massive scaling of supply, and meaningful reductions in the cost of goods, all of which will dramatically advance the field of cell therapy for patients.”

In addition to its AML clinical programme, AvenCell is engaged in other lead programmes for treating prostate cancer and other solid tumours.

The company was founded in 2021 by Blackstone Life Sciences, Cellex Cell Professionals and Intellia Therapeutics.

It is based in Cambridge, Massachusetts, US, and has research and development (R&D) and manufacturing operations in Dresden, Germany.