On November 15, 2016 Cellectis (Paris:ALCLS) (NASDAQ:CLLS) (Alternext: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene edited CAR T-cells (UCART), reported that a series of production runs of UCART123, a Company’s wholly-owned TALEN gene edited product candidate, was successfully performed in large scale, according to cGMP standards, for the purpose of conducting two Phase 1 clinical trials in patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).

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The cGMP manufacturing of UCART123 clinical batches has been operated with CELLforCURE, a LFB group company and the largest industrial facility for clinical and commercial production of innovative cell and gene therapies in Europe. CELLforCURE is in charge of implementing cGMP manufacturing processes that are designed and developed by Cellectis.

The manufacturing process for Cellectis’ allogeneic CAR T-cell product line, Universal CARTs or UCARTs, yields frozen, off-the-shelf, engineered CAR T-cells. UCARTs are meant to be readily available CAR T-cells for a large patient population. Their production can be industrialized and standardized with defined pharmaceutical release criteria.

UCART123 is an engineered T-cell product candidate that targets CD123, an antigen that is located on CD123-expressing leukemic cells in AML as well as leukemic and other tumoral cells in BPDCN. We are planning to file an Investigational New Drug application (IND) with the United States Food and Drug Administration (FDA) in order to initiate clinical studies.

AML is a devastating clonal hematopoietic stem cell neoplasm characterized by uncontrolled proliferation and accumulation of leukemic blasts in the bone marrow, peripheral blood, and occasionally in other tissues. These cells disrupt normal hematopoiesis and rapidly cause bone marrow failure and death. In the U.S. alone, there are an estimated 19,950 new AML cases per year, with 10,430 estimated deaths per year.

Preclinical and translational activities on UCART123 in AML are performed in collaboration with Dr. Monica Guzman, Associate Professor of Pharmacology in Medicine at Weill Cornell Medical College. The clinical research at Weill Cornell will be led by principal investigator Dr. Gail J. Roboz, Director of the Clinical and Translational Leukemia Programs and Professor of Medicine.

BPDCN is a very rare and aggressive hematological malignancy that is derived from plasmacytoid dendritic cell precursors. BPDCN is primarily a disease of the bone marrow and blood cells, but also often affects skin and lymph nodes.

Cellectis collaborates with the MD Anderson Cancer Center on the preclinical development of UCART123 in BPDCN preliminary to the Phase I clinical trial in BPDCN to be activated. The UCART123 clinical program at MD Anderson will be led by Professor Hagop Kantarjian, MD, Department Chair, Department of Leukemia, Division of Cancer Medicine.

"We are proud of achieving this important milestone for UCART123, our first wholly owned product candidate. The successful translation of an R&D concept into a cGMP clinical grade industrial product is one of the key gating factors for us to move into clinical trials," said Arjan Roozen, Vice President, GMP Solutions and Manufacturing.

"With this work and Cellectis’ breakthrough TALEN-based gene editing technology, we continue building upon the Company’s milestones as a pioneer in the field, strengthening our pipeline and bringing us closer and closer to finding efficient and cost-effective products for cancer patients across the globe," added David J.D. Sourdive, Executive Vice President, Corporate Development, Cellectis.