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Huntington’s disease clinical trials enrolling in US

Huntington’s disease clinical trials enrolling in US

Clinical trials testing either tominersen, an experimental therapy designed to target the underlying cause of Huntington’s disease, or dalzanemdor, an oral candidate that aims to improve cognitive abilities, are currently enrolling Huntington’s patients in the U.S.

Updates on the therapies’ clinical programs were provided by representatives of their respective developers during a clinical trials lunch at the Huntington’s Disease Society of America annual convention, held May 30-June 1 in Spokane, Washington.

Huntington’s is caused by mutations in the HTT gene, which results in the production of an abnormal form of the huntingtin protein that is thought to drive nerve cell death and Huntington’s symptoms.

Roche’s tominersen, administered into the spinal canal to reach the brain, is an RNA-based medication that’s designed to reduce levels of both normal and mutated forms of the huntingtin protein.  Data from proof-of-concept trials have indicated that the therapy effectively lowers abnormal huntingtin levels as designed.

To this day, tominersen is the only drug that’s been able to do this.

Sage Therapeutics’ dalzanemdor (formerly SAGE-718) is an oral therapy that aims to help ease one of Huntington’s most troublesome symptoms: cognitive impairment. Dalzanemdor is designed to boost the sensitivity of receptor proteins for a brain signaling molecule called N-methyl-D-aspartate (NMDA). These receptors are known to be involved in nerve cell communication and cognitive function.

By helping to restore NMDA receptors’ activity, dalzanemdor aims to turn the metaphorical switch back up, increasing cognitive capacity.

June 10, 2024

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