Decades-Long Search for Huntington’s Disease Treatment Nears a Breakthrough

Decades after the identification of the genetic marker for Huntington’s disease, the biopharma industry continues its relentless pursuit of an effective treatment for this devastating condition.

Recent developments within this space include setbacks for Roche and Wave Life Sciences, the closure of Triplet Therapeutics, and the halting of the Huntington’s program by NeuBase Therapeutics.

The discovery of the huntingtin gene, associated with Huntington’s disease, was a landmark achievement attributed to Rudy Tanzi and his team in 1983.

The identification of the CAG repeat in the huntingtin gene as the cause of the disease marked a crucial milestone a decade later, shedding light on the mechanism behind the debilitating symptoms.

Prilenia Therapeutics, under the leadership of Michael Hayden, is actively involved in the development of treatments for Huntington’s and other neurodegenerative disorders.

Paul Bolno, from Wave Life Sciences, highlights the dual nature of Huntington’s disease as both a toxic gain and loss of function, complicating therapeutic approaches.

Wave’s focus on allele-specific knockdown with WVE-003 aims to selectively reduce mutant huntingtin protein levels while preserving the essential functions of the wildtype protein.

Delivery challenges, such as targeting the central nervous system effectively, have been a significant hurdle in the development of therapies for Huntington’s disease.

Prilenia’s pridopidine, although showing promise, recently missed key endpoints in Phase III trials, hinting at the complexities involved in treating this condition.

Despite setbacks, European regulators have urged Prilenia to pursue approval for pridopidine, signaling a potential shift towards a breakthrough in Huntington’s disease treatment.

The pioneering work of scientists like Rudy Tanzi and ongoing research efforts by companies like Roche, Wave Life Sciences, and Prilenia Therapeutics underscore the industry’s dedication to finding a solution for this debilitating genetic disorder.