Cellectis' UCART22 Granted Orphan Drug Status for Acute Lymphoblastic Leukemia

June 04, 2024-- Cellectis, a clinical-stage biotechnology company specializing in gene-editing innovations for cell and gene therapies, has announced that its candidate UCART22 has received Orphan Drug Designation (ODD) from the European Commission (EC) for the treatment of Acute Lymphoblastic Leukemia (ALL).

UCART22 is an allogeneic CAR T-cell product aimed at targeting CD22, currently under evaluation in the BALLI-01 trial. This Phase 1/2 study is designed to assess the safety, expansion, persistence, and clinical effectiveness of UCART22 in patients who have relapsed or are refractory to standard treatments for ALL.

ALL is a severe and fast-progressing cancer, accounting for 12% of all leukemia cases. If left untreated, it can be fatal within a few months. In 2024, the prevalence in the European Union (EU) is estimated to be 1.9 per 100,000 people. The European Medicines Agency (EMA) acknowledged the significant benefit of UCART22 based on preliminary data from highly pretreated patients.

Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis, highlighted the potential impact of UCART22, especially for patients with limited treatment options who have relapsed or are refractory to existing therapies, including those who failed previous CD19 directed CAR T-cell therapy and allogeneic stem cell transplants.

Cellectis presented promising data at the American Society of Hematology in December 2023, revealing that UCART22-P2, which is fully manufactured in-house, showed a 67% response rate at Dose Level 2, compared to a 50% response rate at Dose Level 3 with UCART22-P1, produced by an external CDMO. The company plans to provide updates on the BALLI-01 study by the end of 2024.

The Orphan Drug Designation in the EU is granted to medications intended for the treatment, diagnosis, or prevention of conditions that are seriously debilitating or life-threatening and affect fewer than five in 10,000 people in the EU. This designation provides regulatory, financial, and commercial incentives to encourage the development of treatments for rare diseases with unmet medical needs.

About Cellectis

Cellectis is a pioneering clinical-stage biotechnology company focused on life-saving cell and gene therapies through its innovative gene-editing platform. The company employs an allogeneic approach for CAR-T immunotherapies, aiming to make gene-edited CAR T-cells available off-the-shelf for cancer treatment. Utilizing TALEN® gene editing technology and PulseAgile electroporation system, Cellectis develops therapeutic solutions for various diseases that lack effective treatments. With over 24 years of expertise, Cellectis operates from its headquarters in Paris, France, and has offices in New York and Raleigh, North Carolina. Cellectis is publicly traded on the Nasdaq Global Market and Euronext Growth.

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