NEWS SK pharmteco Cell & Gene Europe, a global contract development, manufacturing, and analytical testing organization serving the pharmaceutical and cell & gene therapy industries, along with Assistance Publique – Hôpitaux de Paris (AP-HP), and Institut Imagine (Inserm, AP-HP, Université Paris Cité) announced the signing of a contract for the production of a 200L CGMP clinical batch of lentiviral vector (LVV) and associated regulatory support. AFMTéléthon is the main funder of the final preclinical phases and the technical production of the clinical batches that have enabled us to reach this new milestone. This collaboration focuses on developing a gene therapy against sickle cell disease using an ex vivo hematopoietic stem and progenitor cell (HSPC) gene modification approach with LVV. This innovative approach is based on a newly designed vector by the laboratories of Annarita Miccio and Marina Cavazzana, Research Directors at Institut Imagine, and also on previous collaborative work with Mario Amendola at Généthon laboratory. The project leverages SK pharmteco’s LentiSure™ platform and transgene plasmid, with analytical testing conducted at its facility in France. It is a key milestone towards the start of a clinical trial supported and sponsored by AP-HP and the translation of Institut Imagine’s research to clinical stage. “We are thrilled to partner with AP-HP and Institut Imagine on this critical project,” said Patrick Mahieux, President of SK pharmteco Cell & Gene Europe. “The successful production of both the technical and clinical batches will be yet another demonstration of the power of our LentiSure™ platform and our commitment to advancing gene therapies for serious diseases like sickle cell disease.” “We are proud to support this key validation phase for this gene therapy drug candidate for sickle cell disease, a rare and extremely serious blood disorder that affects thousands of people in France and millions worldwide, and to enable the researchers we have supported for many years, thanks in particular to the Telethon, to take the final step towards treating these patients” said Laurence Tiennot-Herment, President of AFM-Téléthon. “This collaboration represents a significant step forward in our efforts to develop a safe and effective gene therapy for sickle cell disease,” said Dr Jean-Roch Fabreguettes, clinical researcher and coordinator of AP-HP’s MEARY center for cell and gene therapy and manufacturing of advanced therapy medicinal products. “We are confident that this partnership with SK pharmteco and Institut Imagine will bring us closer to improving patients’ lives.” “Institut Imagine is dedicated to translating cutting-edge research into innovative therapies,” added Pr Marina Cavazzana, Head of Research. “This partnership with SK pharmteco and APHP enables us to move our pre-clinical findings into the clinic, offering hope for patients with sickle cell disease.” Sickle cell disease, a genetic disease that affects red blood cells, is most often diagnosed at birth. The most frequent symptoms are anemia, increased infection susceptibility, and painful attacks affecting various organs. According to the World Health Organization, approximately five million people are living with sickle cell disease. It is a chronic disease for which there is currently no cure; this clinical trial, therefore, represents a major hope for patients affected by this disease. SK pharmteco Cell & Gene Europe, AP-HP, AFM-Téléthon and Institut Imagine announce collaboration for lentiviral vector production for an innovative sickle cell disease clinical trial
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