News

Dosing Starts in SELECT-HD Trial That Seeks to Lower mHTT Levels

A Phase 1/2 trial evaluating Wave Life Science’s investigational therapy WVE-003 for Huntington’s disease has started dosing patients, the company has announced. The SELECT-HD trial (NCT05032196), underway in clinical sites in Australia, Germany, Poland, and the U.K., is currently looking to enroll 36 Huntington’s patients, ages…

SAGE-718 on FDA Fast Track as Potential Huntington’s Therapy

The U.S. Food and Drug Administration (FDA) has granted fast track designation to SAGE-718 as a potential therapy for Huntington’s disease. The FDA gives this designation to experimental medications with the potential to improve medical care for serious health conditions and fill unmet needs. The designation gives the therapy’s…

Abnormal DNA Repair Mechanism May Be Therapeutic Target

The interaction between FAN1 and MLH1 — two DNA repair proteins known to be genetic modifiers of Huntington’s disease — protects against further expansion of disease-causing CAG repeats, according to a study using human and mouse models of the disease. Specifically, this interaction prevents MLH1’s recruitment to a DNA…

AMT-130 Gene Therapy Appears Safe; More Data Forthcoming

No significant safety concerns were identified in the first eight Huntington’s disease patients treated with uniQure’s potential gene therapy AMT-130 in a U.S. Phase 1/2 clinical trial. Six of these patients received the low dose of the therapy — which is delivered directly into the brain — and two…

Presymptomatic Disease Changes One’s View of Time, Patients Say

Huntington’s disease patients who have not yet developed symptoms have a different relationship with time than otherwise-healthy individuals, according to a new study based on patient interviews. The findings highlight a need for long-term psychological support for people with presymptomatic Huntington’s to help them develop proactive coping strategies and…

Glia Brain Cells May Open Door to Treatment Strategy

Brain cells called glia work to protect the brain from damage in Huntington’s disease by reducing the expression of genes involved in maintaining connections between nerve cells, a new study indicates. The findings suggest that targeting glia could be a useful strategy for Huntington’s treatment, and also highlight that…

Hearing on Huntington’s Appreciated, But Parity Act Is Priority

While the Huntington’s Disease Society of America (HDSA) is pleased a U.S. House of Representatives subcommittee recently held a key hearing that featured Huntington’s disease, it continues to call for passage of bipartisan parity legislation. The House Energy and Commerce Subcommittee on Health hearing was focused at better…

VO659 Earns Orphan Drug Designation

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vico Therapeutics’ VO659, an investigational antisense oligonucleotide gene-silencing therapy for Huntington’s disease. “We are delighted that FDA has granted this orphan-drug designation,” Rupert Sandbrink, MD, PhD, chief medical officer of VICO, said in a…