NEW YORK, Aug. 01, 2024 (GLOBE NEWSWIRE) — Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS) (the “Company”), a clinical-stage biotechnology company using its pioneering TALEN® genome editing technology to develop potential innovative therapies for serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Cellectis’ CLLS52 (alemtuzumab), an investigational medicinal product (IMP) used in the lymphodepletion regimen in combination with its UCART22 product candidate, being evaluated in the BALLI-01 clinical trial in relapsed/refractory B-cell acute lymphoblastic leukemia (ALL).
“We are pleased that the FDA has granted CLLS52 (alemtuzumab) ODD designation. As previously highlighted, the importance of alemtuzumab in the lymphodepletion regimen was demonstrated in our BALLI-01 clinical trial, where the addition of this lymphodepleting agent to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly greater UCART22 cell expansion, leading to greater clinical activity,” said Mark Frattini, MD, PhD, Chief Medical Officer of Cellectis.
Cellectis is the inventor of the UCART combination with a gene knockout CD52 with a lymphodepletion regimen including an anti-antibodyCD52 like alemtuzumab. The gene knockout CD52 renders the UCART product candidate resistant to alemtuzumab. Lymphodepletion of patients, in turn, reduces host immune cells and should improve the expansion and persistence of allogeneic CAR T cells. Inactivation of the gene CD52 in the UCART22 product candidate is achieved using TALEN® genome editing technology.
The FDA grants ODD status to drugs intended for the treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Obtaining ODD status can help expedite and reduce the cost of developing, approving, and commercializing a therapeutic agent.
About Cellectis
Cellectis is a clinical-stage biotechnology company using its pioneering TALEN® genome editing technology to develop innovative therapies for serious diseases. Cellectis is developing the first allogeneic CAR-T cell immunotherapy therapeutics, pioneering the concept of off-the-shelf, ready-to-use gene-edited CAR-T cells for the treatment of cancer patients, and a platform for therapeutic gene editing in hematopoietic stem cells for a variety of diseases. Leveraging its 25 years of genome engineering expertise, its TALEN® genome editing technology and pioneering PulseAgile electroporation technology, Cellectis is developing innovative product candidates that harness the power of the immune system to treat diseases with high unmet medical needs.
Cellectis is headquartered in Paris. Cellectis also has offices in New York and Raleigh, USA. Cellectis is listed on the Euronext Growth market (ticker: ALCLS) and on the Nasdaq Global Market (ticker: CLLS).
To learn more, visit our website: www.cellectis.com
Follow Cellectis on social media: @cellectis, LinkedIn and YouTube.
TALEN® is a registered trademark owned by Cellectis.
Warning :
This press release contains “forward-looking” statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as “may,” “should,” “target,” or “intend” or the negative of these terms and other similar expressions. These forward-looking statements are based on our management’s current expectations and assumptions and information currently available to us. Forward-looking statements include statements about the potential of CLLS52. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including the many risks associated with the development of biopharmaceutical product candidates, including the risk of losing orphan drug designation if it is determined that the product no longer meets all of the criteria for orphan drug designation prior to marketing approval, if any. In addition, many other important factors, including those described in our Annual Report on Form 20-F and Financial Report (including Management’s Discussion and Analysis) for the year ended December 31, 2023 and subsequent filings made by Cellectis with the Securities Exchange Commission from time to time, which are available on the SEC’s website at www.sec.gov, as well as other known and unknown risks and uncertainties, could adversely affect these forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we undertake no obligation to publicly update these forward-looking statements, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.
For further information about Cellectis, please contact:
Media contacts:
Pascalyne Wilson, Director, Communications, +33 (0)7 76 99 14 33, [email protected]
Patricia Sosa Navarro, Chief of Staff to the CEO, +33 (0)7 76 77 46 93
Investor Relations Contact:
Arthur Stril, Interim Chief Financial Officer, +1 (347) 809 5980, [email protected]
- ODD designation CLLS52_FRENCH.pdf
