CD52

Aug. 01, 2024 -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, 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 as part of the lymphodepletion regimen associated with UCART22, evaluated in the BALLI-01 clinical trial in relapsed/refractory B-cell acute lymphoblastic leukemia (ALL). “We are excited that the FDA granted CLLS52 (alemtuzumab) ODD designation status. The importance of adding alemtuzumab to the lymphodepletion regimen has been demonstrated in Cellectis’ BALLI-01 study, where the addition of this lymphodepletion agent to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly higher UCART22 cell expansion allowing for greater clinical activity,” said Mark Frattini, M.D., Ph.D. Chief Medical Officer at Cellectis. Cellectis is the inventor of the combination of CD52 knockout UCART cells with a lymphodepleting regimen containing an anti-CD52 antibody such as alemtuzumab. The CD52 knockout aims to render the UCART product candidates resistant to alemtuzumab as part of the lymphodepleting regimen. Cellectis’ UCART22 product candidate has the CD52 gene inactivated by TALEN® gene editing technology. The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Receiving ODD may help to expedite and reduce the cost of development, approval, and commercialization of a therapeutic agent. Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. Cellectis utilizes an allogeneic approach for CAR-T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to make therapeutic gene editing in hemopoietic stem cells for various diseases. As a clinical-stage biopharmaceutical company with 25 years of experience and expertise in gene editing, Cellectis is developing life-changing product candidates utilizing TALEN®, its gene editing technology, and PulseAgile, its pioneering electroporation system to harness the power of the immune system in order to treat diseases with unmet medical needs. Cellectis’ headquarters are in Paris, France, with locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). The content above comes from the network. if any infringement, please contact us to modify.

Alemtuzumab is being tested in a Phase I trial along with Cellectis’ CAR T-cell therapy UCART22. Image credit: Shutterstock / Nemes Laszlo. The US Food and Drug Administration (FDA) has granted Cellectis ’s CLLS52 (alemtuzumab) an orphan drug designation for use in a type of leukaemia, as the company touts positive results from a Phase I trial. The designation is for the use of CLLS52 as part of the lymphodepletion regimen associated with a different CAR-T therapy by Cellectis , UCART22, in patients with relapsed/refractory B cell acute lymphoblastic leukaemia (ALL), as per a 1 August press release. Alemtuzumab is manufactured by Sanofi , under the brand names Campath and Lemtrada for chronic lymphocytic leukaemia (CLL) and multiple sclerosis (MS), respectively. Campath was withdrawn from markets in 2012 to make way for Lemtrada and prevent cheaper off-label use. Lemtrada is currently available only through a restricted programme due to its risks of autoimmunity, infusion reactions, and malignancies. Sanofi agreed to supply the drug to support Cellectis’s clinical trials as part of a May 2021 agreement. At the time, the companies said they would also enter discussions to carry out commercial supply of alemtuzumab under agreed-upon financial conditions. Cellectis used CLLS52 in a Phase I BALLI-01 trial (NCT04150497) as part of a lymphodepletion regimen, along with fludarabine and cyclophosphamide – named an FCA regimen. Cellectis was investigating its asset UCART22, which was also administered intravenously after a lymphodepletion regimen to determine the maximum tolerated dose and recommended Phase II dose. UCART22 has also been granted an orphan drug designation by the European Commission (EC). See Also: Otsuka snaps up Jnana with its phenylketonuria drug for $800m Molecure and Avicenna link to develop cancer drugs Manufactured from healthy donor cells, UCART22 expresses anti-CD22, a cell surface molecule often found on cancer cells. The UCART22 product has the CD52 gene inactivated to render it resistant to anti-CD52 antibodies such as alemtuzumab. Cellectis reported the trial showed positive preliminary efficacy and safety results in August 2023. Cellectis chief medical officer Mark Frattini said: “The addition of this lymphodepletion agent [alemtuzumab] to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly higher UCART22 cell expansion allowing for greater clinical activity.” Cellectis is busy working on up to ten new cell and gene therapy products as a result of an equity investment and research partnership with AstraZeneca in November 2023. The investment, which concluded in May this year , means the big pharma now has a total equity stake of 44% in Cellectis. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

-- Selective and complete eradication of leukemic cells with an ADC targeting the pan-blood marker CD45 -- -- Engineered hematopoietic Stem Cells (HSCs) shielded from the CD45-targeting ADC maintain full functionality -- -- Represents a novel, potentially universal approach to treating blood cancers -- BASEL, Switzerland & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Cimeio Therapeutics today announced a publication in Nature showing that its CD45 antibody-drug conjugate (ADC) eradicated aggressive leukemic cells in vivo, while hematopoiesis was fully preserved and protected from the ADC via shielded hematopoietic stem cells (HSCs). The findings point to a novel and potentially universal approach to treating blood cancers. CD45 is highly expressed on blood cancers but also on healthy blood cells. This pro now has prevented effective targeting for therapeutic purposes. Now, a group led by Dr. Lukas Jeker, and researchers at Cimeio have shown that acute myeloid leukemia (AML) cells can be eliminated using Cimeio’s proprietary CD45-targeting ADC. By transplanting engineered HSCs that are shielded from the CD45-directed therapy, the CD45 ADC was safely and effectively administered against the blood cancers. This work is foundational for the emerging field called epitope shielding, which enables the development of curative therapies for patients with hematologic diseases, severe autoimmune conditions and potentially infectious diseases such as HIV. The core patent for epitope shielding is exclusively licensed to Cimeio by University of Basel. “All AML cells express CD45 but since all healthy blood cells also express it, killing cells that express CD45 results in severe toxicity,” said Dr. Jeker, who is the co-founder of Cimeio and Professor of Experimental Transplantation Immunology & Nephrology at the Basel University Hospital, Switzerland. “We solved the problem by providing epitope-shielded HSCs, which enable the ADC to selectively deplete cancer cells while sparing the healthy ones.” “We believe that this therapy could change the treatment paradigm for patients with AML, which is a difficult-to-treat disease with a five-year survival rate of only 25%,” said Stefanie Urlinger, Ph.D., Chief Scientific Officer at Cimeio. “By protecting a patient’s heme system from toxicity, we are able to develop new targeted treatments previously not possible.” Cimeio Therapeutics is developing epitope engineered cells and paired targeted therapies for CD45 and CD117, and epitope engineered cells for CD33, CD52, and several other heme targets. About Cimeio Therapeutics Cimeio is an immunotherapy company developing Shielded-Cell & Immunotherapy Pairs™ (SCIP), novel immunotherapies which have the potential to transform treatment of hematologic diseases. Cimeio develops immunotherapies, along with paired, modified variants of naturally occurring cell surface proteins in HSCs. These novel epitopes edited variants maintain their function but are resistant to depletion when targeted by a paired immunotherapy which has high affinity for the wild-type version of these proteins. These immunotherapies have significant therapeutic potential, which Cimeio is using to develop curative treatments for patients with hematologic malignancies, autoimmune disorders, and genetic diseases. Shielded Cell and Immunotherapy Pairs and SCIP are trademarks of Cimeio Therapeutics, Inc. For more information, please visit .