AvenCell

AvenCell Therapeutics is a clinical-stage biotechnology company that emerged in 2021 from a strategic collaboration between Blackstone Life Sciences, Cellex Cell Professionals, and Intellia Therapeutics. The company, headquartered in Watertown, Massachusetts, with additional R&D and manufacturing in Dresden, Germany, was established with an initial capital commitment of $250 million from its sole founding investor, Blackstone. AvenCell's formation incorporated the German clinical-stage biopharmaceutical firm GEMoaB GmbH, now AvenCell Europe GmbH.

The company is focused on developing next-generation immunotherapies for hard-to-treat cancers, with a primary focus on hematological malignancies like acute myeloid leukemia (AML). AvenCell's business model is centered on advancing its proprietary cell therapy platforms through clinical trials to address significant unmet medical needs. Its core technology combines two distinct platforms: a switchable universal T-cell technology from the former GEMoaB and a license to Intellia's CRISPR/Cas9-based allogeneic platform. This integration aims to create reprogrammable T-cell products that can be turned "on" or "off" after administration to a patient, a mechanism designed to improve the therapeutic window and reduce the severe toxicities often associated with conventional CAR-T therapies.

AvenCell is developing both autologous (using a patient's own cells) and allogeneic ("off-the-shelf") therapies, which have the potential for greater scalability. Its leading product candidates, AVC-101 (autologous) and AVC-201 (allogeneic), both target the CD123 antigen found on most AML cells. The company has dosed patients in early-stage clinical studies for both candidates, reporting a 53% overall response rate in an initial study of 19 AML patients. Beyond AML, the company is exploring applications for its technology in other hematological malignancies and has expressed interest in expanding into the autoimmune disease space.

The company is led by CEO Andrew Schiermeier, a biotech executive with over two decades of experience, including leadership roles at Intellia Therapeutics and as Global Head of Merck KGaA's Oncology Business. In October 2024, AvenCell secured $112 million in a Series B financing round led by Novo Holdings, with participation from new and existing investors, to accelerate the clinical validation of its platform.

Keywords: cell therapy, CAR-T, immunotherapy, oncology, acute myeloid leukemia (AML), hematological malignancies, allogeneic therapy, autologous therapy, CRISPR/Cas9, gene editing, switchable CAR-T, CD123 target, biopharmaceutical, clinical-stage, Blackstone Life Sciences, Intellia Therapeutics, Novo Holdings, cancer treatment, T-cell therapy, autoimmune diseases, biotechnology