CD52

Abcuro is earmarking a portion of new series C funds to prepare for a potential commercial launch of ulviprubart.\n Sanofi-backed Abcuro has secured $200 million in series C funds to power its anti-KLRG1 antibody through a phase 2/3 muscle disease trial ahead of a planned push to regulators.Abcuro is targeting KLRG1 to drive the depletion of cytotoxic T cells in autoimmune diseases. While other players in the space are targeting CD25, CD2, CD52 and ICOS with the same goal in mind, Abcuro has been betting on KLRG1 because expression is restricted to highly cytotoxic late-differentiated effector memory and effector T cells.The idea is that its anti-KLRG1 antibody ulviprubart, also known as ABC008, will deplete cells that drive autoimmune disease without affecting regulatory T cells and central memory T cells.Ulviprubart is currently in a phase 2/3 trial for inclusion body myositis (IBM), a muscle wasting disease that affects about 50,000 people across the U.S. and Europe. Abcuro has a lot riding on the study, with plans to file an approval application with the FDA if the study is a success when it reads out in the first half of 2026.To that end, the biotech is also earmarking a portion of the series C funds to prepare for a potential commercial launch of ulviprubart. Some more of the cash will be spent on expanding the company’s manufacturing capabilities.  The financing round was led by New Enterprise Associates, with new investor Foresite Capital joining a list of previous backers like Sanofi Ventures, RA Capital, Bain Capital Life Sciences, Redmile Group, Samsara BioCapital, Pontifax, Mass General Brigham Ventures, New Leaf Ventures and funds and accounts managed by BlackRock, to name a few.“Continued support from all of our investors in this latest financing round validates our vision for the potential that ulviprubart may have as a novel treatment for progressive and devastating diseases mediated by highly cytotoxic T cells, including inclusion body myositis,” Abcuro CEO Alex Martin said in a Feb. 12 release.IBM looks like a good proving ground for ulviprubart’s potential. The disease is driven by T-cell attacks on muscle fibers that lead to loss of hand function and ability to walk. In 2019, Steven Greenberg, M.D., a neurologist at Brigham and Women’s Hospital and Abcuro founder, co-authored a paper that pointed to a link between KLRG1-expressing T cells and IBM.It led Mass General Brigham Ventures to team up with Sanofi Ventures to oversee a series A-1 round back in 2021 to build on Greenberg’s research. The biotech followed this up with a $155 million series B in 2023.As well as the later-stage study in IBM, the company is also overseeing a phase 1/2 clinical trial of ulviprubart in T-cell large granular lymphocytic leukemia.

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