GG

GeneT Gene Therapy Center of Excellence at ESGCT 2026

Oct 27–30, 2026 Hamburg, Germany ~2.5k attendees Website

GeneT Gene Therapy Center of Excellence brings 7 presentations to ESGCT 2026 in Hamburg. Its work centres on gene therapy for inherited ataxias, with several strands aimed at silencing the mutant ataxin-3 gene behind Machado-Joseph disease. Other work covers building gene therapy capacity in East Africa and communicating the field to the public.

OrganizationESGCT 2026 Attendance
GG
GeneT Gene Therapy Center of Excellence
Coimbra, Portugal
Universities & institutes
Portugal's gene therapy research centre at the University of Coimbra, covering research, clinical trials and production for rare and untreatable diseases.
Poster
Wed Oct 28
1:30pm–3:30pm
From Awareness to Readiness: CAPACITY Strengthening for Gene Therapy in East Africa
Regulatory (poster only)
Poster
Thu Oct 29
2:00pm–4:00pm
Adeno-associated virus-mediated miR-128 delivery reduces ataxin-3 aggregation, partially rescues the cerebellar transcriptomic profile, and improves motor function in transgenic Machado–Joseph disease mice
CNS & sensory
Poster
Thu Oct 29
2:00pm–4:00pm
Direct glial reprogramming in combination with mutant ataxin-3 silencing to treat Machado-Joseph Disease
CNS & sensory
Poster
Thu Oct 29
2:00pm–4:00pm
Early systemic CYP46A1 gene therapy enhances therapeutic benefit in a mouse model of Spinocerebellar Ataxia Type 3
AAVs / non integrative vectors
Poster
Thu Oct 29
2:00pm–4:00pm
GenExpress: a co-created, immersive, human-sized game for gene therapy communication
Other
Poster
Thu Oct 29
2:00pm–4:00pm
Multiplex CRISPRa-mediated activation of neuroprotective pathways rescues Machado–Joseph disease
CNS & sensory
Talk
Fri Oct 30
12:15pm–1:00pm
Cross-species validation of allele-specific silencing for Spinocerebellar Ataxia type 3
SESSION 12a: AAVs/non integrative vectors III - Translation
RNA and oligonucleotide therapiesRare and inherited disease

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