AB-1001

Last updated May 28, 2025

AB-1001 is an experimental gene therapy developed for Huntington's disease. It is intended to increase the production of the CYP46A1 enzyme and cholesterol levels in the brain.^[1]^[2]

References

↑ Parkin, Georgia M.; Corey-Bloom, Jody (2023). "Considerations and Advances in Huntington's Disease Clinical Trial Design". Biomarkers for Huntington's Disease: Improving Clinical Outcomes. Springer International Publishing. pp. 405–431. doi:10.1007/978-3-031-32815-2_17. ISBN 978-3-031-32815-2.
↑ Schultz, Meredith; Durr, Alexandra; Karachi, Carine; O’Callaghan, Michael; Alves, Sandro; Lamontagne, Alain; Hurmic, Hortense; Cartier, Nathalie (25 April 2023). "Investigating the Effects of Brain Cholesterol Metabolism Using CYP46A1 Gene Therapy in Subjects with Huntington's Disease (P1-11.016)" . Neurology. 100 (17 Supplement 2). doi:10.1212/WNL.0000000000203493. ISSN 0028-3878. S2CID 258404880.

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[1] Parkin, Georgia M.; Corey-Bloom, Jody (2023). "Considerations and Advances in Huntington's Disease Clinical Trial Design". Biomarkers for Huntington's Disease: Improving Clinical Outcomes. Springer International Publishing. pp. 405–431. doi:10.1007/978-3-031-32815-2_17. ISBN 978-3-031-32815-2.

[2] Schultz, Meredith; Durr, Alexandra; Karachi, Carine; O’Callaghan, Michael; Alves, Sandro; Lamontagne, Alain; Hurmic, Hortense; Cartier, Nathalie (25 April 2023). "Investigating the Effects of Brain Cholesterol Metabolism Using CYP46A1 Gene Therapy in Subjects with Huntington's Disease (P1-11.016)" . Neurology. 100 (17 Supplement 2). doi:10.1212/WNL.0000000000203493. ISSN 0028-3878. S2CID 258404880.

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