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SR-Tiget at ESGCT 2026
Oct 27–30, 2026 Hamburg, Germany ~2.5k attendees Website
SR-Tiget brings 35 presentations to ESGCT 2026 in Hamburg. Its work centres on lentiviral blood stem cell gene therapy and genome editing for inherited blood and metabolic diseases. One strand tracks how engineered stem cell clones behave long after treatment, with further work on in vivo liver and macrophage engineering against cancer.
| Organization | ESGCT 2026 Attendance |
|---|---|
ST SR-Tiget Milan, Italy | |
San Raffaele Telethon Institute for Gene Therapy, which develops lentiviral stem cell gene therapies and gene editing for inherited diseases. | Chair Tue Oct 27 9:30am–11:00am CLINICAL TRIALS AND COMMERCIALISATION WORKSHOP 1 Manufacturing, CMC and accessClinical trials and translation Chair Tue Oct 27 11:15am–1:00pm CLINICAL TRIALS AND COMMERCIALISATION WORKSHOP 2 Manufacturing, CMC and access Talk Tue Oct 27 3:00pm–4:30pm Impact of HSC signature on gene therapy for beta-thalassemia Rare and inherited diseaseClinical trials and translation Talk Tue Oct 27 5:30pm–7:30pm SESSION 2b: (Epi)genome editing I Gene and epigenome editing Talk Tue Oct 27 6:30pm–7:30pm 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. Rare and inherited diseaseVectors and delivery (AAV, lentiviral, LNP) Talk Wed Oct 28 8:30am–10:30am Wiskott–Aldrich Syndrome: Translating Gene Therapy into Standard of Care Rare and inherited diseaseClinical trials and translation Talk Wed Oct 28 10:00am–10:30am Biological properties and clonality of engineered hematopoietic stem/progenitor cells persisting long-term after gene therapy Rare and inherited diseaseClinical trials and translation Chair Wed Oct 28 11:00am–12:30pm SESSION 4d: Metabolic diseases II In collaboration with ASGCT and AGCTS Rare and inherited disease Talk Wed Oct 28 12:15pm–12:30pm Liquid Biopsy Profiling Reveals CAR-T Cell Dynamics, Therapeutic Activity, and Toxicity in B-cell Malignancies Cell therapies and CAR-TCancer gene and cell therapy Poster Wed Oct 28 1:30pm–3:30pm Cell-Free DNA Methylome Deconvolution Reveals Tissue-Specific Injury Signals Across Inherited and Inflammatory Diseases Poster Wed Oct 28 1:30pm–3:30pm Decoding HSC identity: a single cell multiomics approach to dissect hematopoietic stem cell heterogeneity Cell therapies and CAR-T Poster Wed Oct 28 1:30pm–3:30pm Development of an epigenome editing strategy for the treatment of β-hemoglobinopathies Poster Wed Oct 28 1:30pm–3:30pm Dual-vector gene editing system for targeted integration of immune-active payloads and immune cell reprogramming Poster Wed Oct 28 1:30pm–3:30pm Folding-related adenosine mutations uncover a novel source lentiviral heterogeneity Poster Wed Oct 28 1:30pm–3:30pm Gamma-retroviral gene therapy for adenosine deaminase deficiency: long-term post-marketing outcome and hospital exemption experience with mobilized peripheral blood engineered CD34+ Poster Wed Oct 28 1:30pm–3:30pm Investigating the biological properties of circulating Hematopoietic Stem/Progenitor Cells in pediatric subjects as novel source for Gene Therapy. Poster Wed Oct 28 1:30pm–3:30pm MULTIPLEXED POLYFUNCTIONAL GENE-SIZED EDITING ENABLES SELECTION OF PRECISELY EDITED HEMATOPOIETIC STEM CELLS WITH ENHANCED ENGRAFTMENT POTENTIAL Poster Wed Oct 28 1:30pm–3:30pm Preclinical development of an optimized bicistronic lentiviral vector enabling effective HSC Gene therapy for GM2 gangliosidoses Poster Wed Oct 28 1:30pm–3:30pm Sonication linker-mediated PCR enables accurate and scalable quantification of vector integration sites Chair Thu Oct 29 8:30am–10:30am SESSION 7c: Accessibility of GT Manufacturing, CMC and access Talk Thu Oct 29 9:30am–10:30am Lentiviral vector transduction coupled to ex vivo expansion of circulating hematopoietic stem/progenitor cells to treat a bone disease with limited HSC availability Vectors and delivery (AAV, lentiviral, LNP)Rare and inherited disease Talk Thu Oct 29 10:15am–10:30am In vivo lentiviral engineering of the liver reveals and modulates systemic tumor immune escape Vectors and delivery (AAV, lentiviral, LNP)Cancer gene and cell therapy Talk Thu Oct 29 11:15am–1:00pm Poster Thu Oct 29 2:00pm–4:00pm Advancing Homology-Directed Repair (HDR)-mediated RAG1 gene correction in human hematopoietic stem and progenitor cells Poster Thu Oct 29 2:00pm–4:00pm An innovative platform approach for the parallel development of HSPC-GT for rare/ultra-rare lysosomal storage disorders with severe skeletal and neurological manifestations Poster Thu Oct 29 2:00pm–4:00pm CLEAN: a universal platform for seamless targeted gene addition by self-enforcing saturating selection in human hematopoietic stem cells Poster Thu Oct 29 2:00pm–4:00pm Comet LV: a lentiviral vector-based mRNA co-packaging technology for in vivo cancer immunotherapy Poster Thu Oct 29 2:00pm–4:00pm Dissecting hepatocyte heterogeneity in liver growth to improve in vivo gene therapy Vectors and delivery (AAV, lentiviral, LNP) Poster Thu Oct 29 2:00pm–4:00pm Hematopoietic Stem Progenitor Cell-Gene Therapy for GLB1-Related Disorders: restoration of β-Galactosidase activity and rescue of metabolic and functional defects in a Glb1-/- mouse model Poster Thu Oct 29 2:00pm–4:00pm In vivo macrophage engineering to deliver new immune-activating molecules to liver metastases Poster Thu Oct 29 2:00pm–4:00pm Introduction of a premature stop codon in apolipoprotein B gene reshapes low-density lipoproteins and results in reduction of cholesterol in hypercholesterolemic mice Poster Thu Oct 29 2:00pm–4:00pm LentiMac enables in vivo IFNa gene therapy in a novel hematochimeric model of liver metastases Poster Thu Oct 29 2:00pm–4:00pm Lentiviral In Vivo Gene Therapy Shows Restoration of ADA-SCID Phenotype and Effective Hematopoietic Stem Cell Gene Transfer in Hematochimeric Mice Poster Thu Oct 29 2:00pm–4:00pm Optimization of a platform transduction protocol for lentiviral HSPC gene therapy for lysosomal storage disorders. Poster Thu Oct 29 2:00pm–4:00pm Pre-clinical development of an ex-vivo Hematopoietic Stem/progenitor Cells-gene therapy for a-Mannosidosis Poster Thu Oct 29 2:00pm–4:00pm Senescence-driven inflammation and adaptive immunity influence the genotoxicity of ex vivo HSPCs gene therapy Poster Thu Oct 29 2:00pm–4:00pm Vector-associated clonal aging and somatic mutation burden after HSPC gene therapy Chair Fri Oct 30 9:00am–10:30am SESSION 11b: Bleeding disorders Rare and inherited disease Talk Fri Oct 30 9:30am–10:30am Charting human developmental hematopoiesis in vitro to open new routes for disease modeling Clinical trials and translation Talk Fri Oct 30 12:45pm–1:00pm Simultaneous orthogonal cell engineering by a single CRISPR-Cas9 polyfunctional editor Gene and epigenome editingCell therapies and CAR-T Chair Fri Oct 30 2:30pm–4:30pm SESSION 13 Cancer models & gene therapy Cancer gene and cell therapy |
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