22-23 May 2025
Sanofi Campus Gentilly
22-23 May 2025
Sanofi Campus Gentilly
17 rue Crépet
69007 Lyon
+33 (0)4 78 02 39 89
www.mabdesign.fr
17 rue Crépet
69007 Lyon
+33 (0)4 78 02 39 89
www.mabdesign.fr
ICGT2025
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Tuan Huy Nguyen, a statutory researcher at INSERM, is on full-time availability at GoLiver Therapeutics since its creation in 2017. As a scientist, he has over 30 years of expertise in the field of liver-directed cell and gene therapy. He has explored various biological tools (lentivirus, AAV, CRISPR/cas, ZFNs, liver-isolated hepatocytes and pluripotent stem cells) in small and large animal models. From 2012 to 2017, he led a research group at the INSERM UMR1064 – Center of Research in Transplantation and Immunology (Nantes). During this period, he initiated a new research program funded by an FP7-European grant to evaluate the therapeutic potential of pluripotent stem cells. This research has led to the creation of Goliver Therapeutics, which aims to develop innovative, highly effective medicines for liver diseases, and to meet to the major societal, industrial and economic challenges facing healthcare systems, at a cost that is affordable and accessible to all. He has received three research awards and served as deputy editor of the Current Gene Therapy journal from 2014 to 2017. He is the author of over 70 peer-reviewed publications.
Hematopoietic cell transplantation (HCT) is a crucial therapy for blood disorders like leukemia, sickle cell disease, and autoimmune diseases. Increasing demand for clinical-grade hematopoietic stem cells (HSCs) presents challenges in patient care.
To address this, we propose large-scale HSC production from human induced pluripotent stem cells (hiPSCs) at an Advanced Therapy Medicinal Production (ATMP) core facility and a new large animal model for human HCT. We developed a robust, transgene-free 17-day protocol to differentiate hiPSCs into transplantable HSCs using morphogens and cytokines. Collaborating with Atlantic Bio’s GMP ATMP facility, we scaled up HSC production, ensuring quality control through cytometric validation and transplantation into irradiated NSG mice.
In parallel, we adapted the Aachen mini-pig model for HCT, a suitable pediatric oncology model. Key developments included conditioning regimens using irradiation and immunosuppressive drugs, monitoring, and blood analysis. Overcoming porcine immunity, Cobra Venom Factor was used to protect human cells by inhibiting complement.
With scaled HSC production validated and conditioning protocols established, the large animal HCT study is in process. This marks a critical step towards hiPSC-derived transplants, advancing treatment for hematological and autoimmune diseases.
I am a PhD student under a CIFRE fellowship at the Etablissement Français du Sang (EFS). With a strong foundation in hematology and transfusion science, I’ve dedicated the past five years to research in this field, beginning with a Master’s internship at the Centre de Recherche de Saint Antoine in Paris, where I was inspired by the first laboratory-generated blood transfusions conducted on patients. Our team combines deep expertise in hematopoietic stem cells derived from iPSCs, translational medicine, and a commitment to advancing cell therapies.
Elena Gaia Banchi1, Nissrine Ballout 2, Rafael Alonso1, Johan Deniaud3, Sylvie Jacquot1, Kevin Fransquin1, Françoise Roux4, Marie Anne Colle3, Jérôme Ausseil2, Françoise Piguet1.
1TIDU GENOV, Paris Brain Institute, Paris, France,
2 Biochemistry, Toulouse University Hospital, Toulouse, France.
3 UMR 703 PAnTher INRAE/Oniris, Ecole Nationale Vétérinaire, Agroalimentaire et de l’Alimentation Nantes-Atlantique
4 ONIRIS Veterinary school of Nantes, Nantes France.
MPSIIIB is an autosomal recessive lysosomal storage disorder, caused by alpha-N-acetylglucosaminidase (NaGlu) enzymatic deficiency leading to accumulation of Heparan Sulfate Oligosaccharides (HSO) in tissues including the central nervous system (CNS). Patients manifest with early developmental delays followed by severe behavioral abnormalities, progressive neurodegeneration, and death before the age of 20 years. To date, there are no curative therapies for MPSIIIB. We have previously conducted a AAV-2/5 phase I/II intracerebral gene therapy trial that has shown promising results in four MPSIIIB patients with best results being obtained in the youngest patient (18 months-old). However, disease progression in tissues as important as meninges, brain capillary walls, and choroid plexus was presumably not stopped. Therefore, treatment of patients younger than 2 years and the delivery of NAGLU both within and outside the brain was concluded.
We recently described and presented last year a novel AAV gene therapy using AAVPHP.eB-CAG-NaGlu vector with intravenous delivery in mouse model and combine intracerebral and intravenous delivery in dog and non-human primates. We demonstrated a good efficacy but some local inflammation at the injection site in the brain.
We thus improved our therapeutic strategy by using last generation AAV with AAVMacPNS1, a serotype able of large Blood brain barrier crossing after intravenous delivery notably in large animals. We demonstrate a large CNS transduction of the vector in mouse and NHP after intravenous delivery as well as supra physiological NAGLU expression and activity and a perfect tolerance of the AAV on all tissues.
Efficacy and safety studies are currently ongoing in the dog model of the pathology and data will be presented.
Dr. Françoise PIGUET is heading the innovation unit GENOV in Paris Brain Institute focused on development of gene therapy approaches for neurodegenerative diseases. Since 2006, she contributed to the field of neurodegenerative diseases and development of AAV based- gene therapy approaches first on leukodystrophies, Huntington, Friedreich Ataxia, ALS and mucopolysaccharidosis. She previously developed a clinical trial on metachromatic leukodystrophy and generated 8 patents for notably a gene therapy approach for Friedreich Ataxia as well as for Rett syndrome, MLD, ALS and MPS as well as cell therapies. More recently, Françoise is working on development of new routes of delivery to efficiently target the central and peripheral nervous system. She is an expert of preclinical studies to fill IND and CTA applications.
Coave Therapeutics is revolutionizing gene therapy with its proprietary ALIGATER™ platform, designed to overcome challenges in tissue specificity, delivery efficiency, and manufacturability. ALIGATER™ enables a one-step chemical conjugation process to attach targeting ligands—such as peptides, small molecules, or antibody fragments—to AAV and non-viral vectors. This approach enhances therapeutic precision while maintaining compatibility with existing manufacturing processes. The platform’s first-generation conjugated AAVs (coAAVs), utilizing sugar-based ligands, have demonstrated superior performance in preclinical studies, achieving enhanced tissue diffusion and distribution in the CNS and retina of mice, rats, and non-human primates. Additionally, peptide-conjugated AAVs have been developed to precisely target human receptors, with targeting efficacy modulated by ligand density. These results highlight the versatility and robustness of the ALIGATER™ platform in enabling advanced gene delivery solutions. By addressing key limitations in gene therapy vector design, ALIGATER™ is paving the way for safer, more effective, and scalable genetic medicines with broad clinical applications.
Dr. Lolita Petit is the Chief Scientific Officer at Coave Therapeutics, a Paris-based biotechnology company developing next-generation gene therapies for CNS, neuromuscular, and ocular diseases. With extensive experience in gene therapy research and development, she has led programs from target discovery to clinical development. Dr. Petit has held key scientific leadership roles at Spark Therapeutics (a Roche company) and Johnson & Johnson, where she contributed to innovative gene delivery platform technologies and advanced therapeutic programs for CNS and ocular indications. She is recognized for building and leading high-performing, cross-functional teams that drive innovation and deliver transformative therapeutic solutions, with a focus on precision AAV engineering.
Targeted intracellular delivery of RNA still remains a key requirement. We focus on a bio-inspired chemical evolution strategy. By incorporation of artificial amino acids such as tetraethylene pentamino succinic acid or lipo amino fatty acids (LAF) into xenopeptides (XPs), double pH-responsive carriers have been designed for potent intracellular delivery of RNA in vitro and in vivo. Enhanced endosomal escape turned out to be a key factor for RNA delivery. A pH-dependent polarity of LAF was implemented by a central tertiary amine, which disrupts the hydrophobic character once protonated, resulting in drastic pH-dependent change in the distribution from lipid phase (physiological pH) to lipid/water interface (endosomal pH), as supported by molecular dynamics calculations and SAXS. Activity was maintained in full serum and at very low dosage of only ~2 nanoparticles/cell. Applications include mRNA expression in several organs upon systemic administration, in vivo gene silencing by siRNA-LNPs with superior activity in liver endothelial cells or, when including targeting ligand cRGDfk, in tumor endothelial cells. Potent carriers for CRISPER mediated genome editing, either via Cas9 mRNA/sgRNA or Cas9 protein/sgRNA RNPs, triggering therapeutic genome editing of immune check-point genes in cancer, or in vivo editing of dystrophin.
Prof. Ernst Wagner is Chair of Pharmaceutical Biotechnology, Department Pharmacy, LMU Munich (since 2001). He was Director Cancer Vaccines, Boehringer Ingelheim 1992-2001 (world-wide first polymer-based gene therapy in 1994), 1987-1995 Group Leader at IMP Vienna and Vienna University Biocenter, 1985-1987 postdoc at ETH Zurich, 1985 PhD (TU Vienna). He is Academician of European Academy of Sciences, Controlled Release Society (CRS) College of Fellows, Honorary Professor at U of Sichuan. He authored ≥ 524 publications, with ≥ 54 800 citations, h-index 117 (GS).
