Huntington's Disease Clinical Trials Corner: November 2022

Article Multidisciplinary Sciences

An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion

Caroline Gubser Keller et al.

Summary: Huntington's Disease is a neurodegenerative disorder caused by CAG repeat expansions in the HTT gene. The orally available and brain penetrant small molecule branaplam lowers HTT transcript by promoting inclusion of a poison exon or pseudoexon.

NATURE COMMUNICATIONS (2022)

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Article Neurosciences

Huntington's Disease Clinical Trials Corner: April 2022

Carlos Estevez-Fraga et al.

Summary: This edition of the Huntington's Disease Clinical Trials Corner provides detailed information on the GENERATION HD1, PRECISION-HD1 and PRECISION-HD2, SELECT-HD, and VIBRANT-HD trials, as well as a list of all currently registered and ongoing clinical trials in Huntington's disease.

JOURNAL OF HUNTINGTONS DISEASE (2022)

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Article Biochemistry & Molecular Biology

Pepinemab antibody blockade of SEMA4D in early Huntington's disease: a randomized, placebo-controlled, phase 2 trial

Andrew Feigin et al.

Summary: The SIGNAL Phase 2 study of pepinemab immunotherapy in early Huntington's disease (HD) did not meet its coprimary clinical efficacy endpoints, but had a favorable safety profile and showed a significant treatment-related reduction in caudate brain atrophy and reversal of the characteristic decline in brain metabolic activity that is typical of HD progression.

NATURE MEDICINE (2022)

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Article Multidisciplinary Sciences

Small molecule splicing modifiers with systemic HTT-lowering activity

Anuradha Bhattacharyya et al.

Summary: The authors describe the discovery of small molecule splicing modifiers that can be taken orally, cross the blood-brain barrier, and lower levels of huntingtin in a mouse model of Huntington's disease. These modifiers act by selectively modulating pre-messenger RNA splicing to reduce huntingtin expression throughout the brain and body.

NATURE COMMUNICATIONS (2021)

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Article Neurosciences