New Partnership Aims to Develop Treatment for Huntington’s Disease

According to a story from ncbiotech.org, a new partnership between Vertex Pharmaceuticals and RNA-based therapeutics company Ribometrix aims to develop potential treatments for serious diseases, including Huntington’s disease, which is a rare and fatal genetic disorder. The deal is estimated be valued at $700 million. The partnership aims to combine the two companies’ greatest advantages. For Vertex, this includes clinical, scientific, and regulatory experience; for Ribometrix, it is their proprietary platform which is tailored to identify and optimize new small-molecule therapies.

About Huntington’s Disease

Huntington’s disease is a heritable disorder that causes brain cells to die. This is a long term, progressive, and ultimately lethal disease that causes severe debilitation over time. The disease is caused by a genetic mutation that affects the HTT gene. It normally appears between 30 and 50 years, but in rare cases it can occur before age 20. Symptoms of Huntington’s may first appear as subtle mood and behavioral changes and loss of coordination. Other symptoms include random movements called chorea, abnormal posture, sleep issues, trouble chewing, swallowing, and speaking, dementia, anxiety, depression, and impulsivity. Nine percent of deaths are the result of suicide. Treatment for Huntington’s disease is symptomatic, with no cure or disease altering therapies available. Most patients die around 15 to 20 years after their diagnosis. To learn more about Huntington’s disease, click here.

New Partnerships, New Capabilities

The agreement between the two companies is expected to be valid for three drug development projects. The platform that Ribometrix has developed, which utilizes an RNA targeting mechanism, has the capability to greatly enhance Vertex’s research capabilities, expand the company’s project portfolio, and allow new potential therapies to be discovered more quickly. 

The news of the partnership follows an announcement of $7.8 million of new investment in Ribometrix, which will boost the company’s operations and momentum going forward.

There is a dire need for disease-modifying therapies for Huntington’s disease, which is one of many rare diseases in which no officially approved treatments have been developed anywhere in the world. Hopefully, this new partnership will break new ground and result in the eventual approval of the first ever treatment for this deadly rare illness.


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