P01
Emerging tools and cell engineering approaches
CLEAN: a universal platform for seamless targeted gene addition by self-enforcing saturating selection in human hematopoietic stem cells
Alessandra Weber, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
P02
Emerging tools and cell engineering approaches
Modular Radio-Cell Theranostic Platforms Integrating Molecular Imaging and Adoptive Cell Immunotherapy
Anna Gaimari, Istituto Romagnolo per lo Studio Dei Tumori "Dino Amadori" - IRST IRCCS
P03
Emerging tools and cell engineering approaches
A ntigen-specific dendritic cell immunotherapy to halt autoimmunity in Type 1 Diabetes
Aurora Forlani, Università Vita-Salute San Raffaele, Milan| San Raffaele Institute for Gene Therapy - SR-TIGET
P04
Emerging tools and cell engineering approaches
Baboon retrovirus envelope pseudotyped lentiviral vectors outperform VSVG pseudotyped vectors for macrophage transduction efficacy, cell survival and conservation of phenotype
Chiara Martinello, Université Côte d'Azur
P05
Emerging tools and cell engineering approaches
Developing CRISPRa-engineered human induced pluripotent stem cell-derived neural stem cells (hiPSC-NSCs) to empower treatment strategies for demyelinating diseases
Chiara Garsia, Gene and Neural stem cell therapy for lysosomal storage diseases, SR TIGET - The San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy | Vita-Salute San Raffaele University, Milan, Italy
P06
Emerging tools and cell engineering approaches
Targeted and saturating ABCD1 Gene Addition in HSPCs for the treatment of X-linked adrenoleukodistrophy
Damiano Pulito, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy
P07
Emerging tools and cell engineering approaches
Dual-vector gene editing approach for precision immunoengineering and targeted payload integration
Eleonora Carito, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, 20132, Italy | Vita-Salute San Raffaele University, Milan, 20132, Italy
P08
Emerging tools and cell engineering approaches
Hypoimmunogenic engineered lentiviral vectors to escape in vivo immune recognition
Giulia Buccarello, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Hospital, Milan, Italy
P09
Emerging tools and cell engineering approaches
Targeted gene addition in human HSPCs using TILV for protein replacement therapies
Giulia Scalisi, Genethon Inserm UMR951
P10
Emerging tools and cell engineering approaches
Antigen-specific IL-10–engineered dendritic cells modulate autoimmune neuroinflammation in neuromyelitis optica
Gloria Giacomello, 1Mechanisms of Peripheral Tolerance, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan; | 2 DIMET PhD Program, Università Milano Bicocca, Milan; | 3Clinical Neuroimmunology Unit, Institute of Experimental Neurology, Division of Neuroscience, IRCCS Ospedale San Raffaele, and Vita e Salute San Raffaele University, Milan; | 4Neurology Unit, Neurorehabilitation Unit, and Neurophysiology Service, and Neuroimaging Research Unit, Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan
P11
Emerging tools and cell engineering approaches
Generation of a comprehensive HSPC atlas across sources and ages
Guido Pacini, San Raffaele Telethon Institute for Gene Therapy
P12
Emerging tools and cell engineering approaches
Computational identification and experimental validation of targetable HSC–niche retention pathways to improve genotoxic-free hematopoietic stem cell gene therapy
Hussein Najib Antar, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, 20132 Milan (MI), Italy | Vita-Salute San Raffaele University, 20132 Milan (MI), Italy
P13
Emerging tools and cell engineering approaches
Novel Chimeric Antigen Receptor T cell therapy targeting mislocalised cell surface HSP90B1 for glioblastoma and other solid tumours
Krishneel Prasad, Walter and Eliza Hall Institute of Medical Research, Parkville 3052, Australia | Department of Medical Biology, The University of Melbourne, Parkville 3052, Australia
P14
Emerging tools and cell engineering approaches
Designing novel Versatile Proteolysis Chimeric Antigen Receptor (VIPER) T cell therapies for glioma and other solid tumours
Krishneel Prasad, Walter and Eliza Hall Institute of Medical Research, Parkville 3052, Australia | Department of Medical Biology, The University of Melbourne, Parkville 3052, Australia
P15
Emerging tools and cell engineering approaches
Designing Synthetic Intramembrane Proteolysis Receptors (SNIPRs) for parallel gene induction in T cells
Krishneel Prasad, Walter and Eliza Hall Institute of Medical Research, Parkville 3052, Australia | Department of Medical Biology, The University of Melbourne, Parkville 3052, Australia
P16
Emerging tools and cell engineering approaches
Lentiviral Vector–Mediated UBA1 Correction as a therapeutic approach for VEXAS syndrome
Laura Alessandrini, San Raffaele Telethon Institute for Gene Therapy| Vita-Salute San Raffaele University
P17
Emerging tools and cell engineering approaches
Investigating the biological properties of circulating Hematopoietic Stem|Progenitor Cells in paediatric subjects as novel source for Gene Therapy.
Luca Seffin, San Raffaele Telethon Institute for Gene Therapy
P18
Emerging tools and cell engineering approaches
Optimizing mRNA Manufacturing: A Comparative Study of the AMCAP™ One-Pot Synthesis and Capping Platform
Cristina Chiriaco, Anemocyte srl
P19
Emerging tools and cell engineering approaches
Lipid Nanoparticles Enable Efficient and Scalable Ex Vivo Gene Editing in HSPCs
Martina Fiumara, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.|Vita-Salute San Raffaele University, Milan, Italy.
P20
Emerging tools and cell engineering approaches
Metabolic rewiring of DC by IL-10: decoding the pathways governing immunoregulatory function
Michela Vuono, Mechanisms of Peripheral Tolerance Unit, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy | PhD Course in Molecular Medicine, Vita-Salute San Raffaele University, Milan, Italy
P21
Emerging tools and cell engineering approaches
HER2.CAR-T therapy eradicates drug-tolerant persisters in ALK-rearranged lung cancer organoids and xenograft models
Francesca picca, Department of Oncology, University of Torino, Orbassano, Italy|Department of Molecular Biotechnology and Health Sciences, University of Torino, Torino, Italy
P22
Emerging tools and cell engineering approaches
CRISPRa-mediated utrophin upregulation as a universal therapy for DMD
Paola Galbiati, Genethon, Evry, 91000, France.
P23
Emerging tools and cell engineering approaches
In vivo macrophage engineering to deliver new immune-activating molecules to liver metastases
Sara Breggion, Vector Engineering and In Vivo Tumor Targeting Unit, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy | Vita-Salute San Raffaele University, Milan 20132, Italy
P24
Emerging tools and cell engineering approaches
Computational and targeted CRISPR screening in HSPCs identifies haploinsufficient genes governing RNA processing, translation, and transcription as selection and conditioning candidates
Vigneshwaran Venkatesan, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
P25
Emerging tools and cell engineering approaches
A customizable extracellular vesicle platform for selective CRISPR|Cas9 delivery and gene editing
Alessio Ferraro, Istituto nazionale di genetica molecolare INGM
P26
Emerging tools and cell engineering approaches
Novel envelope glycoproteins for lentiviral vector pseudotyping enable efficient and selective target cell transduction
Daria Saibene, Vector Engineering and In Vivo Tumor Targeting Unit, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Hospital, Milan, Italy|Vita-Salute San Raffaele University, Milan, Italy
P27
Emerging tools and cell engineering approaches
Gene-sized editing with a multiplexable polyfunctional editor allows selection of precisely edited hematopoietic stem cells with enhanced engraftment potential
Dario Russo, San Raffaele Telethon Institute for Gene Therapy |University School of Advanced Studies IUSS
P28
Emerging tools and cell engineering approaches
Brain-targeted in vivo base editing for correction of Krabbe disease
Elena Pelloni, Center for Genomic Medicine and Rare Diseases, Department of Medical and Surgical Sciences, University of Modena and Reggio Emilia, Modena, Italy
P29
Emerging tools and cell engineering approaches
Naturally occurring AAV serotype enables efficient retinal transduction
Emanuela Pone, Gene Therapy Joint Lab
P30
Emerging tools and cell engineering approaches
A new generation of lentiviral vectors to enable precise temporal and spatial control of transgene expression
Matilde Bellandi, Vita-Salute San Raffaele University, Milan 20132, Italy
P31
Emerging tools and cell engineering approaches
Engineering of innovative tools for in vivo gene therapy to hematopoietic stem and progenitor cells
Sarah Giannone, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET)
P32
Emerging tools and cell engineering approaches
A vesicle-based platform for high-efficiency, high-viability CRISPR|Cas9 knockout in primary human myeloid cells
Silvia Fiori, Istituto Nazionale Genetica Molecolare (INGM) | Università degli Studi di Milano (UNIMI), Milan, Italy
P33
Emerging tools and cell engineering approaches
CRISPR-based functional genomics for rAAV production
Tommaso Ferrari, University of Modena and Reggio Emilia
P34
Gene correction strategies and clinical trials updates
Targeting the bone marrow niche by FGF23 inhibition improves the engraftment of transduced hematopoietic stem cells in a preclinical model of gene therapy for beta-thalassemia
Annamaria Aprile, Gene Transfer into Stem Cells Unit, San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy
P35
Gene correction strategies and clinical trials updates
Myeloid-mediated enzymatic correction of ARSA-deficient neural cells inhematopoietic stem cell gene therapy for metachromatic leukodystrophy
Filippo Casalini, 1. San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS Ospedale San Raffaele, 20132 Milan, Italy
P36
Gene correction strategies and clinical trials updates
Immune regulation of hematopoietic stem cell fitness and commitment in Beta -thalassemia
Giulia Chianella, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Italy
P37
Gene correction strategies and clinical trials updates
Tracking the Clonal Fate of HDR Edited T Cells for Hyper-IgM Syndrome Type 1
Ilenia Caracciolo, San Raffaele Telethon Institute for Gene Therapy, Milan, Italy| IUSS, Istituto Universitario di Studi Superiori di Pavia, Pavia, Italy
P38
Gene correction strategies and clinical trials updates
Optimized transduction of mPB CD34+ cells increase transduction efficiency and VCN while preserving stemness: towards a Phase IIb clinical trial of gene therapy for TDT beta-thalassemia
Samantha Scaramuzza, OSR TIGET
P39
Gene correction strategies and clinical trials updates
Targeting IL1 signaling improves HSC function and MSC support to HSC in Sickle Cell Disease
Silvia Sighinolfi, San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan Italy
P40
Gene correction strategies and clinical trials updates
Preclinical development of lentiviral-based hematopoietic stem|progenitor cells (HSPC)-gene therapy (GT) for Mucopolysaccharidosis type IVA within an innovative platform program for Lysosomal Storage Disorders.
Stefania Crippa, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy
P41
Gene correction strategies and clinical trials updates
An innovative platform approach for the parallel development of HSPC-GT for rare|ultra-rare lysosomal storage disorders with severe skeletal and neurological manifestations.
Stefania Crippa, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy
P42
Gene correction strategies and clinical trials updates
Toward base editing application for Hemophilia A precision therapy
Maurizio Cavallo, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan | Vita-Salute San Raffaele University, Milan
P43
In vivo pre-clinical gene therapy
Durable correction of PFIC3 by Alb -targeted HITI-mediated genome editing
Agnese Padula, TIGEM - Telethon Institute of Genetics and Medicine, Pozzuoli, Italy | GEM - Genomics and Experimental Medicine Program, Scuola Superiore Meridionale (SSM, School of Advanced Studies), Naples, Italy
P44
In vivo pre-clinical gene therapy
Lentiviral vector–mediated in vivo platelet FVIII gene therapy for Hemophilia A
Cecilia Iannì, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, 20132, Italy
P45
In vivo pre-clinical gene therapy
Gene therapy enables durable hematopoietic reconstitution in deficiency of adenosine deaminase 2
Chiara Rigamonti, Mechanisms of Inflammation in Health and Disease Unit, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy | Vita-Salute San Raffaele University, Milan, Italy
P46
In vivo pre-clinical gene therapy
AAV integration, vector–host splicing and clonal selection shape the therapeutic effect of liver gene replacement and genome editing
Elisa Bonomi, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy | Department of Electronics, Information and Bioengineering, Politecnico di Milano, Milan, Italy
P47
In vivo pre-clinical gene therapy
Targeted insertion of an optimized donor DNA enables simultaneous Knock-Out and correction in a humanized model of dominant retinitis pigmentosa
Federica Esposito, TIGEM
P48
In vivo pre-clinical gene therapy
Exploring a preclinical AAV-mediated gene therapy approach for the treatment of COASY Protein-Associated Neurodegeneration (CoPAN)
Floriana Cascone, Unit of Medical Genetics and Neurogenetics, The Fundation “Carlo Besta" Institute of Neurology, Milan, Italy
P49
In vivo pre-clinical gene therapy
LentiMac enables in vivo IFNa gene therapy in a novel hematochimeric model of liver metastases
Giovanna Giacca, SR-TIGET
P50
In vivo pre-clinical gene therapy
Safety of AAV-mediated, liver-directed gene therapy for gyrate atrophy of choroid and retina.
Iolanda Boffa, TIGEM, Telethon Institute of Genetics and Medicine, Pozzuoli, Naples
P51
In vivo pre-clinical gene therapy
Liver-Directed AAV-mediated Gene Therapy for Wolman Disease
Iolanda Boffa, TIGEM, Telethon Institute of Genetics and Medicine, Pozzuoli, Naples
P52
In vivo pre-clinical gene therapy
Challenges of liver-directed gene therapy for glycogen storage disease Type 3
Luca Bocchialini, Généthon, Évry, France | Paris-Saclay University, Evry University, Inserm, Généthon, Integrare Research Unit UMR_S951, Évry, France
P53
In vivo pre-clinical gene therapy
mRNA replacement therapy in clinically relevant mouse models of Glycogen Storage Disease type 1b
Lucia De Stefano, AORN Santobono Pausillipon, Medical Genetics Unit, Naples, Italy
P54
In vivo pre-clinical gene therapy
Modulation of TRPML1/TFEB pathway for the treatment of Wilson Disease
Maria Battipaglia, Telethon Institute of Genetics and Medicine, Pozzuoli, Italy
P55
In vivo pre-clinical gene therapy
Overcoming toxic conditioning in hematopoietic stem cell gene therapy leveraging mobilization-resistant CXCR4 variants
Ottavia Vitaloni, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, 20132 Milan (MI), Italy
P56
In vivo pre-clinical gene therapy
Pre-clinical development of an ex-vivo Hematopoietic Stem|progenitor Cells-gene therapy for a-Mannosidosis
Rachele D'Amore, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy|Università Vita-Salute San Raffaele, Milan, Italy
P57
In vivo pre-clinical gene therapy
Engineering thymic selection to promote acceptance of allogeneic transplantation.
Valentina Consoli, San Raffaele Telethon Institute for gene therapy (SR-Tiget), Milan, Italy
P58
In vivo pre-clinical gene therapy
Genome editing for therapy of Leber Congenital Amaurosis type 10
Daria Ponomariova, Tigem - Telethon Institute of Genetics and Medicine | University School of Advanced Studies IUSS, Pavia
P59
In vivo pre-clinical gene therapy
Genome-Wide in vivo CRISPRa screening identifies regulators of hematopoietic stem cell homing and engraftment
Davide Salati, universita Vita-Salute San Raffaele
P60
In vivo pre-clinical gene therapy
Targeting white matter astrocytes with a Gfap-disruptive Cas9 nuclease to treat Alexander disease
Irene Capuano, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Gene|Neural stem cell therapy for lysosomal storage diseases; Milan, Italy.
P61
In vivo pre-clinical gene therapy
Preclinical development of an optimized bicistronic lentiviral vector enabling effective HSC gene therapy for GM2 gangliosidoses
Irene Giustolisi, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS Ospedale San Raffaele, 20132 Milan, Italy | Universita' Vita Salute San Raffaele (UniSR), 20132 Milan, Italy
P62
Safety and responses to gene therapy
A Meta-analysis of Systemically Administered AAVs: Safety Outcomes and Risk Stratification
Alfonso Manuel D'Alessio, Telethon Institute of Genetics and Medicine (TIGEM)|Department of Translational Medicine, University of Naples "Federico II"
P63
Safety and responses to gene therapy
Vector-associated clonal aging and somatic mutation burden after HSPC gene therapy
Francesco Gazzo, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy | Department of Electronics, Information and Bioengineering, Politecnico di Milano, Milan, Italy
P64
Safety and responses to gene therapy
Novel mechanistic insights on lentiviral vector sequence heterogeneity
Kohei Shiroshita, San Raffaele Telethon Institute for Gene Therapy
P65
Safety and responses to gene therapy
Single-Cell Identification of Predictive HSC Biomarkers Defines Clinical Response to Lentiviral Gene Therapy in β-Thalassemia
Maria Rosa Lidonnici, San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute Milan, Italy
P66
Safety and responses to gene therapy
PANDORA: a multiparametric framework for sensitive detection of insertional mutagenesis in gene therapy
Riccardo Pizzichemi, Department of Electronics, Information and Bioengineering, Politecnico di Milano, Milan, Italy | San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) IRCCS San Raffaele Scientific Institute, Milan, Italy
P67
Safety and responses to gene therapy
Biological properties and clonality of engineered hematopoietic stem|progenitor cells persisting long-term after gene therapy
Serena Scala, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy
P68
Safety and responses to gene therapy
Long reading going short: Oxford Nanopore direct RNA sequencing of synthetic guide RNA
Francesca Ferrara, St. Jude Vector Laboratory, St. Jude Children’s Research Hospital, Memphis, TN, United States