Tigem at ESGCT 2026
Oct 27–30, 2026 Hamburg, Germany ~2.5k attendees Website
Tigem brings 16 presentations to ESGCT 2026 in Hamburg. Its work centres on AAV and mRNA therapies for metabolic and retinal diseases, including liver-directed approaches for lysosomal and bile flow disorders. Another strand designs synthetic gene circuits that hold a therapeutic dose steady whatever the number of gene copies delivered.
| Organization | ESGCT 2026 Attendance |
|---|---|
Tigem Pozzuoli, Italy | |
Telethon Institute of Genetics and Medicine, near Naples, researching rare genetic diseases and developing gene therapies for metabolic and eye disorders. | Chair Tue Oct 27 2:00pm–4:30pm SESSION 1: ESGCT 2026 Opening: Clinical applications of gene therapy Clinical trials and translation Chair Tue Oct 27 5:00pm–7:30pm SESSION 2d: Metabolic diseases I In collaboration with JSGCT and CSGCT Rare and inherited disease Chair Wed Oct 28 8:30am–10:30am SESSION 3a: AAVs/non integrative vectors II - Technology Vectors and delivery (AAV, lentiviral, LNP) Talk Wed Oct 28 9:30am–10:30am Advanced LAF-XP polyplexes for Non-Viral Delivery of mRNA to Retinal Photoreceptors Vectors and delivery (AAV, lentiviral, LNP)RNA and oligonucleotide therapies Chair Wed Oct 28 11:00am–12:30pm SESSION 4a: CNS & sensory I Rare and inherited disease Talk Wed Oct 28 12:15pm–12:30pm Development of mRNA replacement therapy in clinically relevant mouse models of Glycogen Storage Disease type 1b RNA and oligonucleotide therapiesRare and inherited disease Poster Wed Oct 28 1:30pm–3:30pm A Modular Synthetic Circuit for Copy-Number-Independent Gene Therapy Vectors and delivery (AAV, lentiviral, LNP) Poster Wed Oct 28 1:30pm–3:30pm Development of a multi-AAV platform for in vivo delivery of evolved type I-F CRISPR-associated transposase Poster Wed Oct 28 1:30pm–3:30pm Engineering a stable cell line for AAV production through synthetic biology Poster Wed Oct 28 1:30pm–3:30pm Evaluation of a new mutation-agnostic gene-editing approach for the rhodopsin-associated autosomal dominant Retinitis Pigmentosa in a humanized mouse model Poster Wed Oct 28 1:30pm–3:30pm Hepatocyte-directed AAV-mediated homology-independent targeted integration for the treatment of Progressive familial intrahepatic cholestasis type 3 Chair Wed Oct 28 1:30pm–2:30pm Poster Wed Oct 28 1:30pm–3:30pm Liver-Directed AAV-mediated Gene Therapy for Wolman Disease Talk Thu Oct 29 10:15am–10:30am Adenine base editing–mediated MECP2 gene correction as a novel gene therapy strategy for Rett syndrome Gene and epigenome editingRare and inherited disease Talk Thu Oct 29 11:00am–1:00pm Chair Thu Oct 29 11:00am–1:00pm Poster Thu Oct 29 2:00pm–4:00pm A Meta-analysis of Systemically Administered AAVs: Safety Outcomes and Risk Stratification Poster Thu Oct 29 2:00pm–4:00pm AAV integration, vector–host splicing and clonal selection shape the therapeutic effect of liver gene replacement and genome editing Poster Thu Oct 29 2:00pm–4:00pm Gene Dosage Compensation Circuits for Cis-Regulation Therapy. Poster Thu Oct 29 2:00pm–4:00pm Model-Guided Design of a Gene Dosage Compensation Circuit for Gene Supplementation Therapy Vectors and delivery (AAV, lentiviral, LNP) Poster Thu Oct 29 2:00pm–4:00pm Periportal expression of ornithine aminotransferase delivered by AAV vectors does not affect ureagenesis and supports the safety of gene therapy for gyrate atrophy of choroid and retina. Talk Thu Oct 29 5:00pm–6:00pm Evaluation of a new mutation-agnostic gene-editing approach for the rhodopsin-associated autosomal dominant Retinitis Pigmentosa in a humanized mouse model Gene and epigenome editingRare and inherited disease |
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