uniQure at ESGCT 2026
Oct 27–30, 2026 Hamburg, Germany ~2.5k attendees Website
uniQure brings 5 presentations to ESGCT 2026 in Hamburg. Its work centres on AAV gene therapy for Huntington's disease, including strategies that lower mutant HTT transcripts and address somatic instability. Other strands cover capsids that cross the blood-brain barrier and a knockdown-and-replace approach for alpha-1 antitrypsin deficiency.
| Organization | ESGCT 2026 Attendance |
|---|---|
uniQure Amsterdam, Netherlands | |
Develops AAV gene therapies for genetic and neurological diseases, including AMT-130 for Huntington's disease, and developed the hemophilia B therapy Hemgenix. | Poster Wed Oct 28 1:30pm–3:30pm BBB-Penetrant AAV Capsid Discovery via Transferrin Receptor Targeting for Preclinical CNS Gene Therapy Poster Wed Oct 28 1:30pm–3:30pm Direct targeting of the liver and lung with a knockdown and replace strategy for alpha-1 antitrypsin deficiency Poster Wed Oct 28 1:30pm–3:30pm Dual targeting of somatic instability and HTT in Huntington’s disease: an optimized AAV gene therapy strategy Poster Wed Oct 28 1:30pm–3:30pm Silencing at the source: reducing nuclear HTT transcripts and RNA foci in the R6/1 HD mouse model using AAV-delivered miRNA Poster Thu Oct 29 2:00pm–4:00pm Systemic delivery of AAV gene therapy with dual targeting of HTT and somatic instability using a blood–brain barrier-penetrant capsid rescues survival in R6/2 Huntington's disease model. |
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